Particles gene therapy viral vector mediated rna interference rna viral. Medical search. Frequent questions

Recombinant adeno-associated viruses (AAV) are one of the leading vectors for gene therapy applications that deliver gene-editing enzymes, antibodies, and RNA interference molecules to eliminate viral reservoirs that fuel persistent infections. (biomedcentral.com)
Here we describe the identification of bottlenecks limiting adeno-associated virus (AAV)-mediated prime editing in vivo and the development of AAV-PE vectors with increased PE expression, prime editing guide RNA stability and modulation of DNA repair. (nature.com)
Hirata, R. K. and Russell, D. W. (2000) Design and packaging of adeno-associated virus gene targeting vectors. (thermofisher.com)
In this review article, we outline key characteristics of major gene therapy viruses-adenovirus, adeno-associated virus (AAV), and lentivirus-and summarize the mechanisms regulating important steps in the virus journey from binding at peripheral nerve terminals to nuclear delivery. (frontiersin.org)
Shedding is the dissemination of the Adeno-associated virus (AAV) vector through secretions or excreta from the animal model or patient. (absorption.com)
Accordingly, this present study describes the generation of hepatic tissue analogues by pneumatic extrusion printing for the investigation of adenoviral infections and adeno-associated virus vector-mediated gene therapy. (tu-berlin.de)
Recently, viral vector gene therapy modalities using adeno-associated viruses (AAVs) have been shown to be an effective therapeutic agent for both rare and common diseases in humans. (jhu.edu)
Adeno-associated virus (AAV) vectors are a leading non-pathogenic platform used for gene therapy delivery. (selectscience.net)
Lentiviruses (LV) and adeno-associated viruses (AAV) are both commonly used viral vectors with different infection characteristics. (cyagen.com)
These lentiviral vectors (LVV) and adeno-associated viral vectors are widely used in the construction of various cell models, in vivo injection of living animals, gene therapy, and other research fields. (cyagen.com)

LncRNAs also contain promoter - or enhancer-associated RNAs that are gene proximal and can be either in the sense or antisense orientation. (wikipedia.org)
More interestingly, a large number of noncoding fragments were found in peripheral blood cells of patients with asthma, including natural antisense chains, pseudogenes, and differential expression of ncRNA between genes [ 14 ]. (hindawi.com)
They include coding mRNAs and non-coding (nc) RNAs among them antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), micro-RNAs (miRNAs), small activating RNAs (saRNAs), RNA aptamers and RNA guides. (frontiersin.org)
There are currently several products containing RNA on the market, and many are under development, among them mRNA, antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), small activating RNAs (saRNAs), micro-RNAs (mi-RNAs), RNA aptamers and RNA guides. (frontiersin.org)
Antisense therapies, in which oligonucleotides inhibit viral replication, have shown promising results in non-human primates following post-exposure treatment. (biomedcentral.com)
Antisense oligonucleotide gapmers containing phosphoryl guanidine groups reverse MDR1-mediated multiple drug resistance of tumor cells. (yale.edu)
Inactivating Gene Expression with Antisense Modified Oligonucleotides. (yale.edu)

Herein, we present the first evaluation of cationic dendrimers based on 2,2-bis(methylol)propionic acid (bis-MPA) as nonviral vectors for transfection of short interfering RNA (siRNA) in cell cultures. (mdpi.com)
The study encompassed dendrimers of generation one to four (G1-G4), modified to bear 6-48 amino end-groups, where the G2-G4 proved to be capable of siRNA complexation and protection against RNase-mediated degradation. (mdpi.com)
ZMapp), anti-sense phosphorodiamidate morpholino oligomers (PMO AVI-6002), lipid nanoparticle small interfering RNA (LNP-siRNA: TKM-Ebola), and an EBOV glycoprotein-based vaccine using live-attenuated recombinant vesicular stomatitis virus (rVSV-EBOGP) or a chimpanzee adenovirus (rChAd-EBOGP)-based vector. (biomedcentral.com)
Gene expression can be attenuated by several approaches, including nucleic acid-based therapies with anti-sense oligonucleotides (ASOs) or RNA interference ( RNAi ), via small interfering RNA ( siRNA ) or micro-RNA ( miRNA ). (scientificarchives.com)
The discovery of RNA interference (RNAi) and the roles that short interfering RNA (siRNA) and micro-RNA (miRNA) play in directing the reversible silencing of genes has enabled the creation of a whole new class of RNA therapeutics to target disease-causing genes that may be otherwise undruggable. (precisionnanosystems.com)
Particle size and PDI determined by dynamic light scattering of siRNA LNPs produced by 3 independent users on the NanoAssemblr® Benchtop demonstrating process consistency. (precisionnanosystems.com)
Here we show, using both small interfering RNA (siRNA) particles and the plasmid based RNAI delivery system, pSUPER-RNAI, that decreasing endogenous hypothalamic AGRP levels in the central nervous system leads to significant physiological responses including an increase in metabolic rate and a decrease in body weight. (biomedcentral.com)
SiRNA particles against both AGRP and GFP were designed based on reports by Elabashir et al. (biomedcentral.com)
Injection of these siRNA particles into the arcuate nucleus of the hypothalamus reduced AGRP mRNA about 50% 24 hours after injection, compared to the control group injected with siRNA particles targeted to GFP (Fig. 1b and 1c ). (biomedcentral.com)
Small interfering RNA (siRNA) particles decreases AGRP mRNA. (biomedcentral.com)
(a) Synthetic siRNA particle designed against AGRP mRNA. (biomedcentral.com)
(b) Representative bands from the Northern blot 24 hours after injection shows decreased levels of AGRP mRNA in the group injected with AGRP-siRNA particle compared to the control GFPi-siRNA injections. (biomedcentral.com)
(c) Quantification of the Northern blot results yields a statistically significant decrease in expression of 50% with the AGRP-siRNA particle. (biomedcentral.com)

Burkholder, J. K., Decker, J., and Yang, N. S. (1993) Rapid transgene expression in lymphocyte and macrophage primary cultures after particle bombardment-mediated gene transfer. (thermofisher.com)
AAV-derived vectors are among the most promising gene therapy tools because of their replication incompetence and ability to mediate long-term transgene expression. (absorption.com)
Strategies such as modifying viral capsids or using immunosuppressive agents may help mitigate this immune response and prolong transgene expression. (aliyuncs.com)
In ovo electroporation offers an easier and more efficient way to manipulate gene expression during embryonic development, and this technique will be useful for neuron-targeted transgene expression. (biomedcentral.com)
It was also correlated with a concomitant decrease in transgene expression, which can limit or reduce the curative effect of the gene therapy. (selectscience.net)

Cell and gene therapy development requires a comprehensive understanding of the unique aspects of these products in relation to their in vivo safety profiles. (absorption.com)
Analysis of the persistence of the vector/virus (gene therapy product) to target and nontarget tissues following direct in vivo administration in animals can be useful for the identiﬁcation of potential target organs of toxicity. (absorption.com)
According to recent reports AAV vector can persist for several years as intact particles in the subretinal space of dogs and nonhuman primates raises the possibility of low levels of replication, which has never been formally excluded in vivo. (absorption.com)
In vitro and in vivo experiments have shown that Ebolavirus pathogenicity is multifactorial, including viral and host determinants. (biomedcentral.com)
The chicken in ovo model is an attractive system to explore underlying mechanisms of neural and brain development, and it is important to develop effective genetic modification techniques that permit analyses of gene functions in vivo. (biomedcentral.com)
The present study demonstrates that RNAI protocols can be used to assess physiological function of neuronal genes in vivo . (biomedcentral.com)
In contrast, RNAI can be used to study long-term phenotypes (including body weight and memory functions) due to the development of a DNA plasmid-based system to deliver short double-stranded RNA sequences in vivo [ 8 ]. (biomedcentral.com)
In the context of AAV gene therapy, we study immune responses in a preclinical setting in vivo , and in vitro using human blood, or samples from different animal species. (selectscience.net)
Our additional in vivo and in vitro model development and phenotypic evaluation services enable us to serve as a comprehensive research solution provider for cell and gene therapy studies worldwide. (cyagen.com)
We also offer In vivo and in vitro model development and CAR-T cell pharmacodynamic evaluation services as a comprehensive research solution provider for cell and gene therapy studies. (cyagen.com)
it is especially suitable for in vivo research and has become one of the most promising viral vectors in the field of gene therapy. (cyagen.com)

In this review, we summarized the interactions of noncoding RNAs and viruses in the occurrence and development of AR, along with the treatments focusing on the noncoding RNAs in the past five years. (hindawi.com)
Gene delivery of antiviral therapeutics to anatomical sites where viruses accumulate and persist is a promising approach for the next generation of antiviral therapies. (biomedcentral.com)
Administration of gene therapy viruses into skeletal muscle, where distal terminals of motor and sensory neurons reside, has been shown to result in extensive transduction of cells within the spinal cord, brainstem, and sensory ganglia. (frontiersin.org)
Gene therapy viruses are non-replicating, but still hijack host cell machinery to express transgenes of interest in the nucleus. (frontiersin.org)
Crucially, some viral vectors (i.e., viruses specifically used to deliver genetic material into cells) have the potential to circumvent the blood-brain- (BBB) and blood-spinal cord barriers (BSCB) when intravenously injected. (frontiersin.org)
Adenoviruses are DNA viruses that can be genetically modified to deliver therapeutic genes into target cells, offering a means to correct or replace defective genes responsible for disease pathogenesis. (aliyuncs.com)
Host lipid metabolism, including lipid droplet (LD) biogenesis, has been associated with viral replication and pathogenesis of different viruses. (biomedcentral.com)
During many stages of the viral replication cycle in the cytoplasm, + RNA viruses interact with host proteins and alter cell homeostasis to benefit viral replication and assembly [ 12 , 13 ]. (biomedcentral.com)
In this context, multiple + RNA viruses use the host lipid machinery to facilitate their replication and assembly [ 23 ]. (biomedcentral.com)
Pharmacological interference in lipid metabolism and LD inhibition affects viral replication of different viruses. (biomedcentral.com)

Recombinant lentiviral vector LV-CMV-RABV G-eGFP was packaged, titered, and then transduced into HEK 293T cells. (bvsalud.org)
We used a replication defective recombinant retrovirus vector GINaTK (bearing HSV-TK gene) to make packaging cell PA317 produce progeny virions. (wjgnet.com)
A recombinant vesicular stomatitis vaccine vector protected non-human primates in pre- and post-exposure challenge studies. (biomedcentral.com)

Messenger RNA (mRNA) vaccines represent a relatively new vaccine class showing great promise for the future. (researchgate.net)
While the first mRNA vaccines against an infectious disease have reached the market ( 1 , 2 ), many other medicinal products with RNA as an active substance (see Table 1 ), either vaccines against non-infectious diseases or non-vaccine drugs, are under development ( 3 - 18 ) or even approved ( 19 - 25 ). (frontiersin.org)
Purification is crucial in manufacturing therapeutics such as monoclonal antibodies (mAbs), mRNA, viral vectors, and non-viral vectors. (jhu.edu)
Since specific pharmacological antagonists of AGRP do not presently exist, we assessed if reduction of hypothalamic AGRP mRNA by RNA interference (RNAI) would influence metabolic function, an outcome suggesting that pharmacological antagonists might constitute useful reagents to treat obesity. (biomedcentral.com)

microRNA (miRNA) plays an important role in regulating gene expression. (wikipedia.org)

c production of replication incompetent AAV vectors via transient plasmid transfection into 293 producer cells that express adenovirus type 5 E1 genes. (biomedcentral.com)
This is a gene that confers resistance to a certain antibiotic or toxin, which helps in identifying and selecting the cells that contain the plasmid. (stemcelldaily.com)

However, the interaction between viral infection and allergy is complex and the mechanism is still unclear. (hindawi.com)
Although, the interaction between viral infection and allergy is complex and the mechanisms remain unclear. (hindawi.com)
HIV infection is efficiently controlled but not cured by Anti-Retroviral Therapy (ART) (Deeks et al. (europa.eu)
2012). This is due to the establishment, early after primary infection, of a viral reservoir that is responsible for the persistence of low levels of plasma viremia in patients under suppressive ART (Chun et al. (europa.eu)
[ 33 ] These findings suggest that early postexposure interferon-beta therapy may be a promising adjunct in the treatment of Ebola virus infection. (medscape.com)
Apart from supportive care, neither a licensed vaccine nor a specific therapy is available for the treatment of the human EBOV infection [ 2 ]. (biomedcentral.com)
Recent infection with yellow fever virus was confirmed in all four organ recipients by identification of yellow fever virus RNA consistent with the 17D vaccine strain in brain tissue from one recipient and seroconversion after transplantation in three recipients. (cdc.gov)
The major barrier to eradication of HIV infection is the latent viral reservoir that persists despite long-term highly active antiretroviral therapy (HAART). (biomedcentral.com)
However, lentiviral vectors (LVV) are the most commonly used for cell therapy applications, such as CAR-T cell construction, and have been widely applied in scientific and clinical research due to their wide host range, stable expression of target gene, and high infection efficiency of mammalian cells. (cyagen.com)
Cyagen uses the 3rd generation Lentiviral Vector (LVV) system, which has high titer, low cytotoxicity, and high infection efficiency. (cyagen.com)
89, No. 21: 10537, 1992), a relatively non-toxic peptide that disrupts viral fusion thereby protecting CD4+ lymphocytes from de novo infection. (justia.com)
Additionally, such agents, by blocking de novo infection, may prevent the expansion of viral reservoirs. (justia.com)
The identification and elucidation of host pathways for viral infection are critical for understanding the viral infection processes and novel therapeutics development. (mdpi.com)

Currently, nucleic acid therapies may be delivered via intermittent administration intrathecally or via intracerebroventricular injection or may be introduced by intraparenchymal injection of viral vectors encoding these therapies. (scientificarchives.com)

A review of retroviral pathogenesis and its relevance to retroviral vector-mediated gene delivery. (thermofisher.com)

We then transferred the HSV-TK gene to target cells SW1990 using these progeny virions, and treated these gene-modified tumor cells with GCV to study the sensitivity of the cells to GCV and their bystander effects by routine MTT-method. (wjgnet.com)
The mutational inactivation of the TP53 gene in tumor cells has been reported to affect not only tumor cells but also the surrounding cells in the tumor microenvironment and to promote tumor-stromal activation and subsequent tumor growth ( 11 , 12 ). (spandidos-publications.com)

Current methodologies for reprogramming, however, face a number of translational hurdles, including heavy reliance on viral vectors, and the highly stochastic nature of current transfection methodologies. (osu.edu)

A prime editing guide RNA (pegRNA) contains a spacer that guides the PE to the target DNA site as well as a 3′ extension that encodes the desired edit. (nature.com)
The mutated HTT gene encodes a protein, mutant huntingtin (mHtt), characterized by a long polyglutamine tract. (scientificarchives.com)
The MCS is usually located within a lacZ gene, which encodes for β-galactosidase enzyme. (stemcelldaily.com)

Occasional RNA molecules act catalytically, as RNA enzymes (ribozymes) or take part in protein export. (wikipedia.org)
Both snRNA and snoRNA are categorized into a class of small RNA molecules that are present in the nucleus. (wikipedia.org)
Viral regulation of B7 family inhibitory molecules in epithelial cells leads to suppression or termination of immune responses [ 19 ]. (hindawi.com)
In some embodiments, methods and compositions are provided for creating and using virion-derived protein nanosphere particles (NSPs) that exhibit surprising selectivity for delivering molecules to tumors without targeting healthy tissue and without producing a serotype-specific immunogenic response in the subject. (justia.com)

By harnessing these viral vectors' ability to efficiently infect both dividing and non-dividing cells, scientists can introduce specific genes into diseased tissues and initiate targeted cellular responses aimed at restoring normal physiological functions. (aliyuncs.com)
Plasmids have been widely used in biotechnology and genetic engineering, as they can be manipulated to introduce, modify, or delete specific genes in the host cell. (stemcelldaily.com)

Many RNA-based drugs, both vaccines and non-vaccines, are under development or even approved. (frontiersin.org)
The EBOV is classified as a biosafety level 4 pathogen and is considered a category A agent of bioterrorism by Centers for Disease Control and Prevention, with no approved therapies and vaccines available for its treatment apart from supportive care. (biomedcentral.com)
Despite the promising possibility that CRISPR/Cas9 could be used to inactivate or even delete proviral DNA from HIV-1 infected cells, an important unanswered question is whether and how HIV-1 might escape from the programmed CRISPR/Cas9 attack, a topic that is fundamental to attempts aimed at HIV treatment and prevention, including the use of small molecule-based antiretroviral therapy and HIV vaccines. (biomedcentral.com)

The term "intergenic" refers to the identification of these transcripts from regions of the genome that do not contain protein-encoding genes. (wikipedia.org)
Moreover, the minimal genome requirements of AAV to replicate permit replacement of most of the genome with foreign DNA, resulting in a packaging capacity of up to 4.7 kb in standard AAV vectors, or about half of that in self-complementary AAV vectors (Fig. 1 ). (biomedcentral.com)
a genome organization of wild type AAV2 depicting its ssDNA genome and the 7 viral RNAs expressed from 2 genes Rep (Black boxes) and Cap (Grey boxes) and via the p5, p19 or p40 promoters. (biomedcentral.com)
The patient tested negative for hantavirus RNA, but next-generation sequencing of a serum sample yielded a complete genome for Rio Negro virus. (cdc.gov)
Genetic studies of patients with autoimmune diseases have shown that one of the most important roles in the developing of these diseases is played by a cluster of genes of the major histocompatibility complex (MHC), as compared with other genome areas. (actanaturae.ru)
In participating UK research institutions, investigators can publish open access in Genome Research, Genes & Development, RNA, and Learning & Memory without article publication charges and all staff can read the entire renowned Cold Spring Harbor journal collection. (cshlpress.com)

Long intergenic Non-coding RNA (LincRNA) is defined as RNA transcripts that are longer than 200 nucleotides. (wikipedia.org)
Cas9 associates with a guide RNA of which the first 20 nucleotides pair with the target DNA. (biomedcentral.com)

Several antiviral therapies are currently under investigation, but only a few of these have been tested in non-human primate models. (biomedcentral.com)

Presence of vector sequences is most commonly tested by Q-PCR, although immunohistochemistry (IHC) has also been applied largely in the assessment of the BD and safety of vectors in gene therapy. (absorption.com)
Rapid escape was observed when non-conserved HIV-1 sequences were attacked, but it did take longer for HIV-1 to escape from Cas9/sgRNAs that targeted the more conserved viral DNA sequences [ 12 ]. (biomedcentral.com)
Another striking feature was the frequent occurrence of insertions and deletions (indels), at least for the less conserved viral target sequences. (biomedcentral.com)
Abstract The candida Ipi3p is required for DNA replication and cell viability in Ipi1-3p have been found to be required for cell viability and for processing of the ITS2 sequences from 35S pre-rRNA in pre-60S ribosomal particles [8, 9]. (irjs.info)

Plasmids can also be used to study gene expression and regulation, gene function and interaction, and gene therapy. (stemcelldaily.com)
Plasmids often carry genes that confer some advantage to the host cell, such as antibiotic resistance, virulence factors, or metabolic capabilities. (stemcelldaily.com)

Small RNA requires a delivery vehicle to protect against nucleases and to facilitate cellular uptake and release into the cytoplasm. (precisionnanosystems.com)

This limitation distinguishes lncRNA from small non-coding RNAs which encompasses microRNAs (miRNAs), small interfering RNAs (siRNAs), Piwi-interacting RNAs (piRNAs), small nucleolar RNAs (snoRNAs), and other short RNAs. (wikipedia.org)
This class includes but is not limited to microRNAs (miRNAs), small interfering RNAs (siRNAs), Piwi-interacting RNAs (piRNAs), small nucleolar RNAs (snoRNAs), and other short RNAs. (wikipedia.org)

Ribosomal RNA (rRNA) includes non-coding RNAs that play essential roles in rRNA regulation. (wikipedia.org)
Using gene manipulation strategies, we reveal for the first time that Cav-1 plays an essential role in CSC regulation and aggressiveness of SWCNT-transformed cells partly through p53 dysregulation, consistent with their suggested role by microarray and gene ontology analysis. (oncotarget.com)
Several lines of evidence strongly suggest that agouti-related peptide (AGRP) plays a key role in the regulation of metabolic function but ablation of the AGRP gene has no apparent effect on metabolic function. (biomedcentral.com)

Microwell Array-Mediated Delivery of Lipoplexes Containing Nucleic Acids for Enhanced Therapeutic Efficacy. (osu.edu)
While gene therapy - and delivery methodologies - have become more effective over the years, human immune responses to the vector remain a significant challenge for the safety, efficacy, predictability, and durability of AAV-mediated gene therapy today. (selectscience.net)
Objective:To explore the effect of different HER2 expression levels and gene amplification on the efficacy of immunotherapy in metastatic urothelial carcinoma (UC).Methods:The clinical data of 77 patients with metastatic UC who received immunotherapy from June 2017 to April 2021 after failure to the previous chemotherapy were analyzed retrospectively, including 49 males and 28 females with the median age of 62 years. (bvsalud.org)

Ribosomal RNA (rRNA) takes part in protein synthesis. (wikipedia.org)
Transfer RNA (tRNA) helps decode a messenger RNA sequence into a protein. (wikipedia.org)
The BD and persistence of a specific viral coat can be analyzed by engineering a fluorescent protein (e.g. (absorption.com)
Besides their function in the virus replication cycle, the viral glycoprotein, nucleoprotein, minor matrix protein and polymerase cofactor are viral determinants of pathogenicity, with evasion of the host innate and adaptive immune responses as the main mechanism. (biomedcentral.com)
APJ‑suppressed fibroblasts treated with an antagonist or small interfering RNA showed myofibroblast‑like properties, including increased proliferation and migratory abilities, via accelerated phosphorylation of Sma‑ and Mad‑related protein 2/3 (Smad2/3). (spandidos-publications.com)

Chesnoy, S. and Huang, L. (2000) Structure and function of lipid-DNA complexes for gene delivery. (thermofisher.com)
Therefore, these data demonstrate the essential role of lipid metabolism and LDs during + RNA viral replication. (biomedcentral.com)

Identification of natural isolates from various tissues in humans and non-human primates has generated an extensive catalog of AAV vectors with diverse tropisms and transduction efficiencies, which has been further expanded through molecular genetic approaches. (biomedcentral.com)
Virus-mediated gene therapy has the potential to deliver exogenous genetic material into specific cell types to promote survival and counteract disease. (frontiersin.org)
AAV vectors mostly remain episomal, and can result in efficient and long-term transduction of genetic material into non-dividing cells. (cyagen.com)

Monoclonal antibodies are the classical basis for targeted therapy, but the development of alternative binding proteins has made it possible to use non-immunoglobulin proteins as targeting modules. (actanaturae.ru)

Among these, AAVs are currently the most widely used delivery vectors in gene transduction and gene therapy due to their advantages, which include good tissue specificity, low immunogenicity, and high safety. (cyagen.com)

In addition, Th2 cells are important mediators of type I allergy, and it has been reported that ncRNA promotes Th2 cell migration to target organs by regulating chemokine gene expression [ 17 ]. (hindawi.com)
As stated by the USFDA, " The biodistribution data, coupled with other preclinical safety endpoints such as clinical pathology and histopathology, help determine whether vector presence or gene expression correlates with any tissue-specific detrimental effects in animals . (absorption.com)
The control of gene expression by therapeutic agents has great potential for the field of neurological diseases. (scientificarchives.com)
Such therapeutic approaches include the suppression of expression of toxic gain-of-function mutations, or the introduction of genes expressing constituents that are beneficial to a particular condition. (scientificarchives.com)
Since the discovery of the pathologic HTT mutation in 1993 and the creation of animal models of the disease, novel therapies have been developed to reduce expression of the HTT gene - so-called huntingtin-lowering therapies [3,5-8]. (scientificarchives.com)
Going forward, non-invasive gene therapies will need to be developed that both lower m HTT expression and suppress neuro- inflammatory processes. (scientificarchives.com)
We tested various specimens (blood, cerebrospinal fluid, intraocular fluid, serum, and tissues) from the organ donor and recipients by serology, RT-PCR, immunohistochemistry, metagenomic next-generation sequencing, and host gene expression, and conducted a traceback of blood transfusions received by the organ donor. (cdc.gov)
In the present study, we performed a comparative study of the ubiquitous CMV promoter and three neuron-specific promoters, chicken Ca2+/calmodulin-dependent kinase (cCaMKII), chicken Nestin (cNestin), and human synapsin I. We explored the possibility of manipulating gene expression in chicken embryonic brain cells using in ovo electroporation with the selected promoters. (biomedcentral.com)
Overexpression of the FOXP2 gene driven by the cNestin promoter in brain cells significantly affected expression levels of target genes, CNTNAP2 and ELAVL4 . (biomedcentral.com)
Together with the observation that transgenic over-expression of the AGRP gene leads to hyperphagia and obesity [ 1 ], these data suggest that antagonism of AGRP may reduce food intake and body weight, thus potentially serving as a therapy for obesity. (biomedcentral.com)
Finally, the gene expression of Atf4, Ire1α, Xbp1s, Xbp1t, Grp78 and Chop was measured from these cDNA samples by qPCR (quantitative polymerase chain reaction). (helsinki.fi)
The qPCR data describes the expression of exact gene. (helsinki.fi)
By statistical analysis, it was possible to compare the expression of these genes between 6-OHDA group and vehicle group. (helsinki.fi)
In other genes there were no statistical differences, unlike in several other studies where the expression was found to be increased. (helsinki.fi)

To overcome these issues and still improve the efficiency of chemotherapeutic agents there is a demand for less toxic and more target specific therapies towards cancer cells, i.e. novel drugs, drug delivery systems (DDSs) and also gene delivery systems. (nanomaterialpowder.com)
The success of AAV as a gene delivery vector is due to several characteristics, including its nonpathogenic nature, its good safety profile, and its ease of production to clinical grade. (biomedcentral.com)
So far, the approved AAV-based drugs and most clinical trials utilizing AAV vectors aim to supplement a defective gene with a new, working copy [ 3 ], but many studies have investigated AAV for the delivery of non-self therapeutic genes. (biomedcentral.com)
Lab-on-Chip devices, gene delivery chips, tissue engineering scaffolds) for applications in fundamental and/or translational medical research. (osu.edu)
Additionally, we describe how neuropathology can negatively influence these pathways, and conclude by discussing opportunities to optimize the intramuscular administration route to maximize gene delivery and thus therapeutic potential. (frontiersin.org)
One potential application is the delivery of genes encoding growth factors or cytokines that promote angiogenesis, the formation of new blood vessels. (aliyuncs.com)
Furthermore, achieving targeted delivery of adenoviral vectors remains a challenge. (aliyuncs.com)
Ongoing research and technological advancements will continue to refine the use of these vectors, addressing challenges related to immunogenicity, targeted delivery, and manufacturing processes. (aliyuncs.com)
A major obstacle to fulfilling the therapeutic promise of gene therapies for hereditary brain diseases, such as Huntington' Disease (HD), is the requirement for viral vectors and/or an invasive delivery system (stereotaxic injection into brain or infusion into the intrathecal space). (scientificarchives.com)
These technical limitations have given impetus to the development of alternative non-invasive delivery systems for gene therapy of brain diseases. (scientificarchives.com)
Design and development of nanocarriers packaged with gene-lowering agents represents a significant advance towards non-invasive nose-to-brain delivery of gene therapy for HD and other hereditary brain disorders. (scientificarchives.com)
Although electroporation and viral vector-mediated gene delivery techniques have been used to introduce exogenous DNA into chicken embryonic cells, transducing neurons efficiently and specifically remains challenging. (biomedcentral.com)

Recently, the AAV-based drugs Luxturna, a therapy to treat inherited blindness, and Zolgensma, a treatment for spinal muscular dystrophy, were approved by the FDA for use in the US, signifying important milestones for the establishment of AAV-based therapeutics in the clinic. (biomedcentral.com)
The Immunology team at Spark Therapeutics, led by Dr. Klaudia Kuranda, Head of Immunology works to identify and characterize immune responses that might occur after infusion of AAV-based gene therapies. (selectscience.net)

Here, we aimed to establish a platform for rRABV G production and purification, and determine the immunogenicity and antigenicity of rRABV G. The cDNA fragment encoding the soluble form of RABV G was synthesized and cloned into a lentiviral expressing vector. (bvsalud.org)

These may include monoclonal antibody (mAbs)-based therapies (e.g. (biomedcentral.com)

This suggests that these mutations are not the result of mistakes by the error-prone viral reverse transcriptase (RT) enzyme but rather represent mutations that are generated by the cellular non-homologous end joining (NHEJ) machinery that repairs broken DNA (Fig. 1 ). (biomedcentral.com)

Long non-coding RNA (LncRNA) are a type of RNA which is usually defined as transcripts which are greater than 200 base-pairs in length and not translated into proteins. (wikipedia.org)
These RNAs must not have open reading frames that encode proteins. (wikipedia.org)
In some embodiments, the nanosphere particle comprises wild-type HPV L2 capsid proteins. (justia.com)
For example, adenoviral vectors can be used to introduce genes encoding calcium-handling proteins involved in excitation-contraction coupling, which is essential for proper contraction and relaxation of cardiac muscle cells. (aliyuncs.com)

Interactions of miRNAs with other regions, including the 5' UTR, coding sequence, and gene promoters have also been reported. (wikipedia.org)

This review will summarize the most recent strategies to identify AAV vectors with enhanced tropism and transduction in cell types that harbor viral reservoirs. (biomedcentral.com)

By enhancing blood supply to the damaged heart tissue, these therapeutic genes can potentially improve cardiac function and reduce symptoms such as shortness of breath and fatigue. (aliyuncs.com)

One particularly influential approach in this regard is the use of adenoviral vectors, which hold great potential for unlocking the therapeutic benefits of gene therapy. (aliyuncs.com)
Gene therapy using adenoviral vectors offers an innovative solution for individuals like John who face debilitating conditions that have traditionally been difficult to manage effectively. (aliyuncs.com)
This article aims to explore the current understanding and advancements in utilizing adenoviral vectors for regenerative biomedicine. (aliyuncs.com)
Adenoviral vectors have shown promise in gene therapy for chronic heart failure by targeting key cellular mechanisms involved in the progression of this condition. (aliyuncs.com)
However, several challenges must be overcome to ensure the safe and effective use of adenoviral vectors in regenerative biomedicine. (aliyuncs.com)
Additionally, issues related to vector stability, scalability, and manufacturing processes need careful consideration when developing adenoviral vector-based therapies. (aliyuncs.com)
In conclusion, adenoviral vectors hold immense potential for revolutionizing regenerative biomedicine and offering new treatment options for chronic conditions such as heart failure. (aliyuncs.com)
With further progress in this field, gene therapy using adenoviral vectors may become a transformative approach in improving patient outcomes and quality of life for individuals like John. (aliyuncs.com)

With thousands of clinical trials to date, gene therapy is a flourishing strategy with great promise for the treatment of diseases impacting the nervous system. (frontiersin.org)
HD is an autosomal dominant neurodegenerative disease for which several clinical trials have demonstrated gene-lowering effects following intrathecal administration. (scientificarchives.com)
A number of systemic AAV-based gene therapy clinical trials have reported asymptomatic elevation in serum levels of liver transaminases, which is an indication of liver inflammation and hepatotoxicity, mentions Dr. Majowicz. (selectscience.net)
In clinical trials, T-20 has been shown to reduce plasma viral load by up to two logs (Kilby et al. (justia.com)

Ebolaviruses are known for their ability to cause devastating viral hemorrhagic fever outbreaks in humans and great apes, but the natural reservoir for EBOV has remained elusive for many years. (biomedcentral.com)
Through viral load suppression, we may be able to prolong a patient's survival in order to provide a better chance for the patient to develop natural immune defense against the EBOV. (biomedcentral.com)
The EBOV is an enveloped filamentous RNA virus belonging to the family Filoviridae . (biomedcentral.com)

Thus, understanding HIV persistence and developing drugs able to flush out HIV, in order to achieve viral eradication or "sterilizing cure" remain outstanding challenges. (europa.eu)
In: RNA Interference - Challenges and Therapeutic Opportunities. (osu.edu)

At RNA doses of 1 mg/kg over 90% knockdown was achieved for LNPs made on the NanoAssemblr® Benchtop , Blaze , and GMP System . (precisionnanosystems.com)
Additionally, in some gene therapy trials in which high doses of AAV vectors were administered systemically, toxicities related to the activation of the complement system have been reported. (selectscience.net)

RNA sequencing, shRNA knockdown, and chromatin Immunoprecipitation (ChIP) sequencing were performed to detect the target genes of ginsenoside Rg1-CNV that regulate angiogenesis. (cbinsights.com)
TRIM28 knockdown increased responsiveness to anti-PD-1 therapy in immunocompetent mice, characterized by increased CD8 + T tumor-infiltrating lymphocytes and decreased MDSCs. (biomedcentral.com)

The five-year survival rate of colorectal cancer patients is ~60%, but it decreases to 14% when distant metastases are present, regardless of the availability of several combination therapies as systemic treatments ( 1 ). (spandidos-publications.com)
These insights may inform the development of combination therapies to enhance the effectiveness of immune checkpoint blockade therapy in NSCLC. (biomedcentral.com)

Then, bioinformatic analysis was performed to investigate the mechanism of action of epigenetic modifying enzymes in regulating target genes. (cbinsights.com)

Meanwhile, the intervention of ginsenoside Rg1-CNV promoted the demethylation of H3K27me3 within the promoter region of VEGF-A and Jagged1 genes and reduced the H3K27me3 modification within this region. (cbinsights.com)
The present study identified TRIM28 as a promoter of chemokine-driven recruitment of MDSCs through RIPK1-mediated NF-κB activation, leading to the suppression of infiltrating activated CD8 + T cells and the development of anti-PD-1 resistance. (biomedcentral.com)

Infrastructural ncRNAs seem to have a housekeeping role in translation and splicing and include species such as rRNA, tRNA, snRNA.Regulatory ncRNAs are involved in the modification of other RNAs. (wikipedia.org)
These results suggest that the noncoding RNA regulatory network may play a potential role in the development of allergic rhinitis (Figure 1 ). (hindawi.com)
Few regulatory studies are available to assist in the regulatory categorization of RNA-based medicinal products. (frontiersin.org)

The persistence of HIV in treated patients results from the establishment of a viral reservoir insensitive to ART and poorly visible to the immune system. (europa.eu)

Long non-coding RNAs include lincRNAs, intronic ncRNAs, circular and linear ncRNA. (wikipedia.org)

Thus, over the past few decades, extensive efforts to optimize AAV vectors for gene therapy applications have focused on capsid engineering with approaches such as directed evolution and rational design. (biomedcentral.com)
New technologies capable of mediating nuclear reprogramming through non-viral deterministic approaches are needed in order to facilitate the transition from lab bench to bedside. (osu.edu)
However, things are becoming more and more complicated as the current evolution of technology in this field makes it possible to produce similar RNA-based drugs using different approaches. (frontiersin.org)
The group is studying targeted approaches to circumvent or treat such responses, with the objective of advancing the safe and efficacious use of gene therapies in patients. (selectscience.net)

Prime editors (PEs) are precision gene editing agents that can perform virtually any substitution, small deletion and small insertion at target DNA sites in living cells 2 . (nature.com)
While these vectors have a broad tropism for infecting various cell types, non-specific uptake by off-target tissues can lead to unintended side effects. (aliyuncs.com)
In addition to this single guide RNA (sgRNA), Cas9 also needs to recognize a multi-nucleotide region that is adjacent to the 3′ end of the target DNA, which is termed PAM (protospacer adjacent motif). (biomedcentral.com)
Some mutations abrogate the function of viral DNA and will not be selected, while others will be selected because they are not deleterious to the virus, yet generate resistance to Cas9/sgRNA attack because the target DNA sequence is changed. (biomedcentral.com)
Ipi3p also functions as a component of the Five Friends of Methylated CHTOP (5FMC) complex which is definitely recruited to ZNF148 by methylated CHTOP, leading to desumoylation of ZNF148 and subsequent transactivation of ZNF148 target genes [10]. (irjs.info)

As long-lived viral DNA within specific cellular reservoirs is responsible for persistent hepatitis B virus, Herpes simplex virus, and human immunodeficiency virus infections, the discovery of AAV vectors with strong tropism for hepatocytes, sensory neurons and T cells, respectively, is of particular interest. (biomedcentral.com)

In cell coculture assays, ICOVIR-15K-cBiTE-mediated oncolysis resulted in robust T-cell activation, proliferation, and bystander cell-mediated cytotoxicity. (aacrjournals.org)

Biodistribution and viral shedding assays are two critical means by which such safety can be assessed. (absorption.com)
Biodistribution (BD) assays determine the amount of viral DNA/RNA in different cells and organs in an organism. (absorption.com)

Small noncoding RNA is usually categorized as being under 200 bp in length, whereas long noncoding RNA is greater than 200bp. (wikipedia.org)
Small non-coding RNA (sncRNA) are a type of RNA. (wikipedia.org)
Small nuclear RNA (snRNA) and small nucleolar RNA (snoRNA) are widely known to guide the nucleotide modifications and processing of rRNA. (wikipedia.org)
Small nuclear and small nucleolar RNAs are critical components of snRNPs and snoRNPs and play an essential role in the maturation of, respectively, mRNAs and rRNAs within the nucleus of eukaryotic cells. (wikipedia.org)

Knowledge about species infectivity, tissue tropism, and cellular infectivity of different vectors is very important for assessing the BD of a cell and gene therapy product. (absorption.com)
Methods: We constructed nanoparticles to fully encapsulate the therapeutic drug (ginsenoside Rg1), which can be transferred into brain tissue via the receptor-mediated transfer of drug-encapsulated nanoparticles. (cbinsights.com)
2 Currently, intravenous thrombolysis with tissue-type plasminogen activator (trPA) and endovascular thrombectomy (EVT) remains the mainstay of acute stroke therapy within the initial 4.5 h after stroke onset. (cbinsights.com)

TP53 is a significant tumor suppressor gene, and its somatic mutations are one of the most frequent alterations in ~50% of all human cancers, including colorectal cancer ( 10 ). (spandidos-publications.com)
Indeed, when the targeted viral DNA regions were sequenced, mutations were identified that interfered with sgRNA recognition. (biomedcentral.com)
The majority of the resistance mutations appeared to cluster at the site at which Cas9 was designed to cleave the viral DNA, even though the sgRNA binding site is much bigger. (biomedcentral.com)
This possibility was confirmed by deep sequencing analysis that showed that a number of the resistance-conferring mutations in the viral escape variants indeed matched the mutations that were introduced into the viral DNA in CD4+ T cells that had been infected by HIV-1 for only 36 h [ 13 ]. (biomedcentral.com)

The invention also provides nanosphere particle preparations that are useful for selectively delivering therapeutic, diagnostic, and/or other agents to cancer cells of subjects without eliciting a serotype-specific immunogenic response in the subjects. (justia.com)
Specifically, we infected cells and birds with a molecularly barcoded WNV and sequenced viral RNA from single cells to quantify the number of WNV barcodes in each. (cdc.gov)
Further, electroporation circumvents the caveat of receptor interference encountered in viral systems, and nondividing cells can be transduced. (biomedcentral.com)

If a foreign DNA is inserted into the MCS, it disrupts the lacZ gene and prevents the production of β-galactosidase enzyme. (stemcelldaily.com)

Recent advances in nuclear reprogramming have opened up the possibility for the development of highly effective, patient-specific cell therapies. (osu.edu)
Examples of RNA-based drugs currently or formerly on the market or under development. (frontiersin.org)
Although there is currently no approved treatment available, the past 10 years has seen remarkable progress in our understanding of the pathogenicity of Ebolavirus and the development of prophylactic and post-exposure therapies against it. (biomedcentral.com)
However, toxicities associated with long term HAART have put a high priority on the design and development of less toxic therapies. (justia.com)

They are not part of the main chromosome of the cell, but they can replicate independently and carry genes that may confer some advantages to the host organism. (stemcelldaily.com)

Anatomy20

Organisms38

Diseases6

Chemicals and Drugs92

Analytical, Diagnostic and Therapeutic Techniques and Equipment38

Phenomena and Processes61

Disciplines and Occupations1

Technology, Industry, Agriculture3

Information Science3

Health Care2

Adeno-associated10

Antisense7

SiRNA13

Transgene expression5

Vivo11

Viruses10

Recombinant3

MRNA4

MiRNA1

Plasmid2

Infection13

Nucleic acid1

Pathogenesis1

Tumor cells2

Transfection1

Encodes3

Molecules4

Specific genes2

Vaccines3

Genome6

Nucleotides2

Antiviral therapies1

Sequences4

Plasmids2

Cytoplasm1

SiRNAs2

Regulation3

Efficacy3

Protein5

Lipid2

Genetic3

Antibodies1

AAVs1

Expression14

Delivery12

Therapeutics2

Immunogenicity1

MAbs1

Reverse transcriptase1

Proteins4

MiRNAs1

Transduction1

Therapeutic genes1

Adenoviral8

Clinical trials4

EBOV3

Challenges2

Doses2

Knockdown2

Combination therapies2

Enzymes1

Promoter2

Regulatory3

Persistence1

NcRNA1

Approaches4

Target5

Reservoirs1

Cytotoxicity1

Assays2

Small4

Tissue3

Mutations4

Cells3

Disrupts1

Development4

Organism1