Gene delivery efficiency. Medical search. Frequent questions

Reduced delivery efficiency has left the potential of non-viral vectors relatively untapped. (frontiersin.org)
This Research Topic will focus on materials as non-viral vectors for cellular uptake and delivery of genes and nucleic acids in order to achieve potential benefits to human health and advance gene therapy prospects. (frontiersin.org)
A common method of transferring new genes or gene-editing components into cells, to provide novel functions, is by transduction of engineered lentiviral or other vectors. (seattlechildrens.org)
This approach is widely applicable to a variety of gene carriers, including viral-based vectors and virus-like nanoparticles. (seattlechildrens.org)
In this scenario, vectors would deliver genes for immunoadhesives - proteins that B cells would produce from introduced genes-to bind and neutralize specific pathogens. (seattlechildrens.org)
The virus-driven delivery tools are called "viral vectors. (health.am)
Ultimately, introducing GRPE elements into viral vectors could enhance the ease and effectiveness of gene therapy, which typically uses transplanted human stem cells. (health.am)
Lentiviral (LV) vectors of the Retroviridae family show interesting properties for monogenic gene therapy, since they integrate into the host genome and allow long-lasting gene expression 22 . (nature.com)
These mutant vectors were first tested for their transduction efficiency in HeLa and HEK293T cells. (nih.gov)
2 h for wild type vectors) in vitro , which further translates to modest increase in hepatic gene transfer efficiency in vivo . (nih.gov)
Recombinant adeno-associated viral (AAV) vectors and integration-deficient lentiviral vectors are promising therapeutic delivery systems for gene therapy to the central nervous system (CNS). (nih.gov)
However, challenges in efficient and targeted delivery to specific cell populations make strategies employing these vectors in the CNS particularly challenging. (universityofcalifornia.edu)
The global viral vectors and plasmid DNA manufacturing market was valued at $319.01 million in 2019 and is expected to reach over $1.3 billion by 2027, according to a report from Precedence Research, which points out that viral vectors have become ideal for gene transfer due to their efficient gene delivery, high transfection efficiency, and stable gene expression. (genengnews.com)
Further, an upsurge in the registration of clinical trials on viral vectormediated gene therapy is stimulating demand for viral vectors in gene transfer. (genengnews.com)
The growing pervasiveness of target disorders and diseases, the accessibility of funding for gene therapy development, current research into viral vector-based cell and gene therapies, and efficacy of viral vectors in gene therapy delivery are together supporting the market growth," notes the Precedence Research study. (genengnews.com)
Non-viral vectors are preferred for gene therapy, despite lower transfection efficiencies, and possess many customizable attributes that are desirable for tissue engineering applications. (ku.edu)
Professor Alexander said the grant will also develop gene viral vectors to target blinding eye disease and treat childhood kidney disease. (edu.au)
All four of the funded projects will develop manufacturing capacity for clinical-grade gene transfer vectors in NSW. (edu.au)
Non-viral vectors based on magnetoplexes, lipoplexes and polyplexes for VEGF gene delivery into central nervous system cells. (ehu.eus)
Nanotechnology based non-viral vectors hold great promise to deliver therapeutic genes into the central nervous system (CNS) in a safe and controlled way. (ehu.eus)
Overall, these results provide new insights for VEGF gene delivery into CNS cells using non-viral vectors. (ehu.eus)
We show that knock-down of each single gene from multiple target vectors is achieved at an efficiency comparable to that obtained after transduction using single target viral vectors. (ejbiotechnology.info)
Introduction: Gene therapy holds great potential for treating a wide range of diseases, and adeno-associated virus (AAV) vectors have emerged as promising vehicles for delivering therapeutic genes. (lu.se)
The objective of this Master thesis project is to develop a comprehensive bioinformatic pipeline that enables the identification of DNA barcodes associated with AAV vectors, thereby facilitating the evaluation of AAV variant efficiency and tropism in mammalian species. (lu.se)
The findings will be summarized, highlighting the efficiency, accuracy, and scalability of the pipeline in identifying DNA barcodes associated with AAV vectors. (lu.se)

Further, for each payload, we identified the structural drivers for nucleic acid internalization, cell viability and transfection efficiency and mined structure-function correlations and predictive relationships that can aid experimentalists engaged in polymer design and synthesis for diverse gene therapy applications. (aiche.org)
Influence of Polymer Architecture and Molecular Weight of Poly(2-(dimethylamino)ethyl methacrylate) Polycations on Transfection Efficiency and Cell Viability in Gene Delivery. (mpg.de)
This NIH funded R00 project has three specific aims which target improved delivery efficiency, throughput, and cell viability in intracellular delivery with an emphasis on stem cell gene editing. (uh.edu)
Ultimately, electroporation exhibits a high transfection efficiency in many cell types, which is highly desirable for tissue engineering applications, but electroporation and other physical non-viral gene delivery methods are still limited by poor cell viability. (ku.edu)
Overcoming the challenge of poor cell viability in highly efficient physical non-viral techniques is the key to using gene delivery to enhance tissue engineering applications. (ku.edu)

A cationic cyclodextrin derivative-lipid hybrid nanoparticles for gene delivery effectively promotes stability and transfection efficiency. (bvsalud.org)

Recharging cationic DNA complexes with highly charged polyanions for in vitro and in vivo gene delivery. (wikipedia.org)
The researchers synthesized a series of RGD peptide-conjugated ionizable lipids The lipids were formulated with DOPE, cholesterol, and lipid-PEG to form lipid nanoparticles which were used to deliver mRNA in vitro and in vivo , as well as co-deliver Cas9 mRNA and sgRNA for evaluation as a platform for gene editing applications. (avantilipids.com)
The researchers used a luciferase reporter system to demonstrate the efficacy of the lipids in vitro , showing that they were able to deliver mRNA to HeLa cells with high specificity and efficiency. (avantilipids.com)
Gene therapy has been utilized successfully to repair or inactivate mutations in animal models of monogenic human diseases 19 as well as human in vitro cells 12 . (nature.com)
To enhance the osteogenic potential of stem cell sheet, we fabricated bone morphogenetic protein 2 ( BMP-2 ) gene-engineered cell sheet using a complex of polyethylenimine-alginate (PEI-al) nanocomposites plus human BMP-2 complementary(c)DNA plasmid, and studied its osteogenesis in vitro and in vivo. (dovepress.com)
The expression of osteogenesis-related gene was increased, which demonstrated that released BMP-2 could effectively induce the cell sheet osteogenic differentiation in vitro. (dovepress.com)
siRNA conjugated chitosan complexes were fabricated with and without an antibody (Ab) targeting moiety and screened for efficiency to reduce iNOS expression in vitro. (syngene.com)
Both in vitro and in vivo results demonstrate that the nanoemulsions improve mucus penetration and facilitate effective cellular delivery of siRNA. (cdc.gov)

The aim of this Research Topic is to report current and future advancements in the use of materials, chemicals and particles as non-viral alternatives for nucleic acid delivery. (frontiersin.org)
Developing polymers for gene delivery is one such multi-dimensional design challenge that requires an intricate balance between intermolecular forces implicated in the packaging, cellular uptake and release of genetic payloads from polymer-nucleic acid complexes. (aiche.org)
The T4 Gene 32 Protein is a single-stranded nucleic acid binding protein that has the function of stabilizing single-stranded regions of DNA. (qiagen.com)
however, successful delivery of nucleic acids into primary cells, progenitor cells, and stem cells has proven exceptionally challenging. (ku.edu)
In this seminar, I will discuss our research efforts to develop "intelligent" pH-sensitive, membrane-destabilizing, and degradable polymeric carriers that can effectively deliver therapeutic nucleic acids past the endosomal membrane and into the cytoplasm of cancer cells to successfully suppress the expression of targeted genes. (usc.edu)
Accurate quantification of cytosolic siRNA, and the establishment of the intracellular dose-response relationships, will aid the development and characterization of novel delivery strategies for nucleic acid therapeutics. (lu.se)

The lipids were also able to deliver mRNA encoding the CRISPR-Cas9 gene editing system and base editor BE3 to HeLa cells, resulting in efficient gene editing. (avantilipids.com)
The researchers found that modified RNA improves the efficiency of CRISPR-Cas9 delivery. (eurekalert.org)
A Penn State-led team of interdisciplinary researchers has developed techniques to improve the efficiency of CRISPR-Cas9, the genome editing technique that earned the Nobel Prize in 2020. (eurekalert.org)
While CRISPR-Cas9 is faster, less expensive and more accurate than other gene-editing methods, according to project leader Xiaojun "Lance" Lian, associate professor of biomedical engineering and biology at Penn State, the technology has limitations - especially in applications to improve human health. (eurekalert.org)
The human genome is enormous, and CRISPR-Cas9 makes it possible for scientists to find and target a mutated gene for the purpose of studying it," Lian said. (eurekalert.org)
In this talk, I will describe how combinatorial polymer libraries, high-throughput experimentation and statistical models have been combined to yield a powerful predictive platform for the discovery and optimization of polymeric vehicles for CRISPR/Cas9 delivery. (aiche.org)

We welcome Original Research papers, Reviews, Mini-reviews or papers providing descriptions of novel methods for significantly advancing gene therapy. (frontiersin.org)

A facile nonviral method for delivering genes and siRNAs to skeletal muscle of mammalian limbs. (wikipedia.org)
A novel picolitre incubator based microfluidic system for consistent nonviral gene carrier formulation is presented. (rsc.org)
DNA Melting Temperature Assay for Assessing the Stability of DNA Polyplexes Intended for Nonviral Gene Delivery. (mpg.de)

They destroy the RNA before it can make proteins - say, in a matter of a few hours - and, in doing so, destroy the gene editing attempt. (eurekalert.org)
Since viruses have unique ability infect cells and deliver the contents in the cytoplasm with almost 100 per cent efficiency, two novel technologies have been developed to deliver proteins and RNAs, respectively, based on virus-like particles. (businessweekly.co.uk)
The inventors have shown that the crystallinity, flexibility, and chemical tunability of PIX can be exploited to encapsulate guest proteins with high loading efficiencies. (universityofcalifornia.edu)
And, the electrostatic host-guest interactions enable reversible, pH-controlled uptake/release of guest proteins as well as the mutual stabilization of the host and the guest, thus creating a uniquely synergistic platform toward the development of functional biomaterials and the controlled delivery of biological macromolecules. (universityofcalifornia.edu)

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)
Dr. Torbett and collaborators are improving transduction of hematopoietic stem cells to increase delivery efficiency. (seattlechildrens.org)
For example, Dr. Torbett's group is studying polyphenols as transduction enhancers based on their results showing that the resveratrol-like compound caraphenol A dose-dependently increases gene delivery and genomic integration into human cells. (seattlechildrens.org)
These multiple possibilities support future use of combinations of compounds to synergistically increase transduction efficiency. (seattlechildrens.org)
In addition to improving gene therapy and delivery of gene-editing tools into cells, increasing transduction efficiency using small molecule agents could facilitate using of hemopoietic stem cells for protective functions. (seattlechildrens.org)
Of the 12 common serotypes used for gene delivery applications, Adeno-associated virus (AAV)rh.10 serotype has shown sustained hepatic transduction and has the lowest seropositivity in humans. (nih.gov)
In the present study the cellular tropism and transduction efficiency of seven AAV vector serotypes (AAV1, 2, 3, 4, 5, 6, 8) and an integration-deficient lentiviral vector were assessed for their ability to transduce corticospinal neurons (CSNs) following intracortical injection. (nih.gov)
Generated through Sangamo's AAV capsid engineering platform, SIFTER (Selecting In vivo For Transduction and Expression of RNA), these capsids have demonstrated a potential for high efficiency delivery of investigatory gene therapy constructs to the central nervous system in pre-clinical animal models following administration into the cerebrospinal fluid and seek to optimize delivery of therapeutics to a range of previously inaccessible areas. (businesswire.com)

The therapeutic gene is loaded onto the vector which then crosses the cell barrier to deliver the cargo gene to target cells, tissues or entire organs. (frontiersin.org)
The critical step in successful gene therapy is vector selection. (frontiersin.org)
After identifying the problem, researchers introduced the element into a lentiviral vector, successfully and significantly improving the quality and quantity of the gene therapy. (health.am)
Delivery and success of gene therapy for human cells has the potential of increasing five to ten times with the introduction of the GRPE into the lentiviral vector. (health.am)
The development of an AAV vector system that can escape the circulating neutralizing antibodies in the host will substantially widen the scope of gene therapy applications in humans. (nih.gov)
AAVs hold tremendous promise as a gene delivery vector to treat such conditions given their reasonable starting efficiency and safety profile. (universityofcalifornia.edu)
The adeno associated virus (AAV) vector is the platform of choice for delivering gene therapeutics. (genengnews.com)
The ability to inhibit two or more genes simultaneously from one single expression vector further widens the application spectrum of RNAi, both in functional studies and therapeutic strategies. (ejbiotechnology.info)
Special attention will be given to avoiding non-specific tissue infection, particularly in the liver, as it is a common site for unwanted AAV vector delivery. (lu.se)
Existing vector control tools have several limitations in terms of cost, delivery and long-term sustainability. (who.int)

Bates MK, Zhang G, Sebestyen MG, Neal ZC, Wolff JA, Herweijer H. Genetic immunization for antibody generation in research animals by intravenous delivery of plasmid DNA. (wikipedia.org)
Wooddell C, Reppen T, Wolff, JA, Herweijer H. Sustained liver-specific transgene expression from the albumin promoter in mice following hydrodynamic plasmid DNA delivery. (wikipedia.org)
CRISPR uses a disc of genetic material, known as plasmid DNA, to deliver guided ribonucleic acid (RNA) that positions the Cas9 enzyme at the precise location of the target gene. (eurekalert.org)
Gene therapy involves viral, plasmid, and oligonucleotide-based approaches. (medscape.com)

Perfluorocarbon nanoemulsions enhance therapeutic siRNA delivery in the treatment of pulmonary fibrosis. (cdc.gov)

The commonly used gene carrier liposomes and cationic polymers have limited their clinical application due to their respective disadvantages. (bvsalud.org)
CDG2-LHNPs with stability and high- transfection efficiency overcome the shortcomings of liposomes and polymers applied separately, and have great potential for gene drug delivery. (bvsalud.org)
Furthermore, PEI polymers illustrate high delivery efficiency but induce cytotoxicity. (tdl.org)
Methods: Successful gene expression by PLGA nanoparticles alone or in combination with arginine-modified PEI polymers (A5P10) was accessed by a luciferase reporter gene encapsulated in PLGA nanoparticles. (tdl.org)

Diseases with a genetic cause could be treated by supplying a correct version of the faulty gene. (sciencedaily.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)
Improvements in the technology of gene delivery identified by Arts and his colleagues could lead to many more effective studies that help patients with many different diseases, including cancer. (health.am)
This agreement highlights the value of our AAV capsid engineering program, not only for the discovery of novel capsids for the advancement of our wholly owned programs, but also as an opportunity to work with partners to broaden the scope of diseases addressable with AAV gene therapy. (businesswire.com)
The genetic information in a cell is stored in exactly the same way in every person, but certain diseases, like cancer, can cause some genes to move. (businessweekly.co.uk)
It will identify new avenues to broadly enhance gene and cell therapies in patients with unmet needs in many genetic diseases and cancer," he said. (edu.au)
Increased knowledge of genomics over the past two decades has made it apparent that the traditional category of genetic diseases represents only those conditions in which the genetic contribution is particularly marked, whereas in fact diseases can be arrayed along a spectrum representing the varied contribution of genes and the environment. (who.int)
These conditions are described as genetic diseases because a defect in one or more genes or chromosomes leads to a pathological condition. (who.int)
Others can arise from the presence of an abnormal gene in any autosome: if the gene is dominant, it results always in what is called a dominant condition, whereas if it is recessive many of these diseases appear only when the gene is inherited from both parents (and are thus called recessive conditions). (who.int)
First, we briefly present the ana- include both the delivery of health- municable diseases (NCDs) includ- lytical framework used in this chap- care services and broader individu- ing cancer (UN, 2015). (who.int)

This is particularly important for gene therapies, as off-target effects can lead to unwanted side effects and reduced efficacy. (avantilipids.com)
This can improve the therapeutic index of gene therapies and increase their efficacy. (avantilipids.com)
One of the key challenges of gene therapies is the risk of triggering an immune response in the patient. (avantilipids.com)
This versatility allows for the development of lipids that can deliver mRNA to different types of cells and tissues, increasing the potential for the development of effective gene therapies. (avantilipids.com)
This approach has the potential to improve the efficacy of gene therapies and address the challenges of treating complex genetic disorders. (avantilipids.com)
Four University of Sydney researchers have received grants worth $5 million from the NSW government to support trials for cell and gene therapies to treat rare genetic conditions and cancer. (edu.au)

However, the efficacy of gene therapy is limited by the challenge of delivering therapeutic agents to target cells. (avantilipids.com)
Recent advancements in messenger RNA (mRNA) technology have made it possible to directly introduce therapeutic genes into cells, but the efficacy of this approach is still limited by the delivery method. (avantilipids.com)
Gene therapy relies mainly on viruses-which transport genomes inside the cells they infect-to deliver genetic material into a patient's cells. (health.am)
Enhanced gene therapy and other improvements in targeted cell delivery might eliminate the need for stem cells and allow cells to be administered directly into patients. (health.am)
Only 20% to 30% of the targeted cells will receive gene-editing DNA when using CRISPR. (eurekalert.org)
When modRNA Cas9 is successfully delivered to the target gene, it creates a double-stranded break in the genome, which some cells will try to fix. (eurekalert.org)
The goal is to develop plasmon-induced intracellular delivery and imaging platforms capable of facilitating efficient and precise delivery of a variety of cargos into living cells, while maximizing their survivability. (uh.edu)
As another example, the Schaffer lab has engineered new gene delivery vehicles that can augment and edit the genome of hESCs and neural stem cells. (ca.gov)
A major bottleneck in nanocarrier and macromolecule development for therapeutic delivery is our limited understanding of the processes involved in their uptake into target cells. (universityofcalifornia.edu)
PEI-al nanocomposites carrying BMP-2 gene could efficiently transfect bone marrow mesenchymal stem cells. (dovepress.com)
Our results suggested that PEI-al nanocomposites efficiently deliver the BMP-2 gene to bone marrow mesenchymal stem cells and that BMP-2 gene-engineered cell sheet is an effective way for promoting bone regeneration. (dovepress.com)
To that end, we have developed a novel approach that for the first time enables high-efficiency gene editing and gene delivery to in primary human B cells. (selectbiosciences.com)
This grant will allow us to determine the specific mechanisms that improve gene transfer efficiencies into cells. (edu.au)
Gene therapy involves the delivery of DNA into cells. (edu.au)
Transfection efficiencies of nanoplexes were measured in terms of percentage of GFP expressing cells, mean fluorescent intensity (MFI) and VEGF (ng/ml) production in HEK293, C6 and primary neuronal culture cells. (ehu.eus)
Magnetoplexes showed the highest transfection efficiencies in C6, followed by lipoplexes, and in primary neuronal culture cells, followed by polyplexes. (ehu.eus)
Sustained expression of short hairpin RNA (shRNA) can be accomplished in mammalian cells by viral delivery systems. (ejbiotechnology.info)
Using lentiviral constructs, stable gene silencing is established both in dividing and non-dividing cells. (ejbiotechnology.info)
Refer to the following optimal transfection conditions for maximal transfection efficiency on PC12 cells. (signagen.com)
Studies on particle characteristics, their cellular uptake into aforementioned cell lines and on subcellular localisation, and transfection efficiency in the cerebellar cells proved the feasibility of nanoparticle-based gene delivery. (uni-frankfurt.de)

Gene editing has emerged as a promising strategy for treating a wide range of genetic disorders. (avantilipids.com)
Researchers at the University of Pennsylvania have recently investigated the potential use of RGD peptide-conjugated lipids for targeted mRNA delivery and gene editing applications. (avantilipids.com)
The study found that the RGD peptide-conjugated lipids were able to efficiently deliver mRNA to specific cell types and achieve high levels of gene editing efficiency. (avantilipids.com)
But there are delivery and editing efficiency problems with current DNA-based CRISPR methods, according to Lian. (eurekalert.org)
The researchers also found that the modRNA could improve other gene editing techniques, such as base editing. (eurekalert.org)
Base editing can knock out genes or correct mutations in the genome by using a protein to change a single nucleotide instead of cutting both strands, like CRISPR does. (eurekalert.org)
According to Lian, as more gene-editing labs improve gene editing efficiency and effectiveness, researchers will be able to better understand genes and their functions more quickly. (eurekalert.org)
Though gene editing has made rapid progress, the paucity of chemically defined, safe and efficient delivery vehicles has hindered its clinical translation. (aiche.org)
To accelerate polymer discovery for vital clinical applications such as gene editing, we need to replace traditional iterative processes with alternative experimental strategies that leverage advances in parallel synthesis and data analytics. (aiche.org)
The hit polymer identified through this study outperformed commercial transfection reagents by achieving nearly 60% editing efficiency via non-homologous end-joining. (aiche.org)
Genome Editing- Altering a genome with unparalleled efficiency and precision. (cdc.gov)

Gene therapy involves the introduction of therapeutic genetic materials in order to rectify aberrant or mutated host gene products responsible for a disease. (frontiersin.org)
Virus-mediated gene therapy has the potential to deliver exogenous genetic material into specific cell types to promote survival and counteract disease. (frontiersin.org)
Advances in gene therapy offer promising treatment for genetic abnormalities, tumors and resistance to toxic chemotherapies. (health.am)
But a failure to distribute enough modified genetic information to the patient's body has prohibited gene therapy from being more widely used. (health.am)
During virus production, GRPE coordinates the production and filling of the container with the genetic material of HIV-1, or the desired human gene. (health.am)

For example, the lipid component of the delivery system can be modified to improve stability, increase circulation time, or enhance cellular uptake. (avantilipids.com)

Nanobubbles increase the efficiency of drug and gene delivery. (worldoil.com)
Another field of interest is the biological functionalisation of nanoparticles for drug and gene delivery and to use these innovative methods as delivery platform for implants, in particular for stents and prostheses. (epfl.ch)

A promising method for the delivery of such genes involves the use of DNA/lipid complexes (lipoplexes). (sciencedaily.com)

Lewis DL, Hagstrom JE, Loomis AG, Wolff JA, Herweijer H. Efficient delivery of siRNA for inhibition of gene expression in postnatal mice. (wikipedia.org)
However, interfering with Sprouty expression by gene therapy or siRNA treatment provides a realistic approach to evaluate the therapeutic potential of indirectly stimulating ERK activities in neurological disease. (springer.com)
To evaluate their potential during hepatic gene therapy, C57BL/6 mice were administered with wild-type or optimal mutant AAVrh.10 and the luciferase transgene expression was documented by serial bioluminescence imaging at 14, 30, 45, and 72 days post-gene transfer. (nih.gov)
DNA methylation and histone modifications play a central role in the epigenetic regulation of gene expression and cell differentiation. (cipsm.de)
Morpholino oligos (Morpholinos) are widely used tools for knocking down gene expression and are currently in a clinical trial for treatment of Duchene muscular dystrophy. (oregonstate.edu)
While PLGA nanoparticles are FDA approved and well established for their biocompatibility, they fail to facilitate exogenous gene expression in primary human astrocytes. (tdl.org)
The addition of A5P10 improved nuclear localization and successfully achieved gene expression in primary human astrocytes. (tdl.org)
Conclusion: By integrating two polymeric nanoparticles, we developed an improved system for gene delivery and expression in primary human astrocytes. (tdl.org)

Their project is about creating a versatile delivery method for cancer therapeutics. (businessweekly.co.uk)
Antisense oligonucleotide (AO)-mediated exon-skipping therapeutics shows great promise in correcting frame-disrupting mutations in the DMD gene for Duchenne muscular dystrophy. (oregonstate.edu)
Endosomal escape and subsequent cytosolic delivery of small interfering RNA (siRNA) therapeutics is believed to be highly inefficient. (lu.se)

Recently, stem cell- and gene-based therapy has been applied in BTE and achieved some good clinical results. (dovepress.com)
He received grant funding to build on his team's recent clinical success using gene therapy to cure haemophilia, published in the New England Journal of Medicine . (edu.au)
However, insufficient systemic delivery limits clinical adoption. (oregonstate.edu)

In this account, we developed the α- cyclodextrin -conjugated generation-2 polyamidoamine dendrimers - lipids hybrid nanoparticles (CDG2-LHNPs) for gene delivery. (bvsalud.org)

Dr. Torbett is interested in partnerships that translate his extensive expertise in basic viral processes to applications that either inhibit viral pathogens or enhance use of viruses or virus-like particles for gene delivery to the nucleus. (seattlechildrens.org)

The mechanism of high transfection efficiency of pDNA/CDG2-LHNPs was further explored using pharmacological inhibitors chlorpromazine , filipin , and cytochalasion D. The result demonstrated that cell uptake of pDNA/CDG2-LHNPs was mediated by clathrin -mediated endocytosis (CME), caveolae -mediated endocytosis (CvME), and macropinocytosis together. (bvsalud.org)
Quantification of gene delivery efficiency and gene targeting has been greatly aided with the flow cytometry and high throughput imaging capabilities of the CIRM facility. (ca.gov)
Results show high transfection efficiency with low cytotoxicity. (syngene.com)

Virus mediated gene delivery (or gene therapy) has been widely used as a delivery method for the past two decades. (businessweekly.co.uk)
As President of the International Society for Cell & Gene Therapy his leadership role in Australian cell and gene therapy is widely acknowledged. (edu.au)

The development of commercially available transfection reagents for gene transfer applications has revolutionized the field of molecular biology and scientific research. (mdpi.com)
166.00 : SignaGen Laboratories, A Gene Delivery Company Providing Custom AAV Adenovirus Lentivirus Production Services & Manufacturing DNA/siRNA Transfection Reagents. (signagen.com)

The team's company will seek venture capitalist funding for this method of treatment and will seek to establish an open technology pool to increase the scope of gene therapy. (businessweekly.co.uk)

For example, in cystic fibrosis, a mutation in the gene that codes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein causes the disease. (sciencedaily.com)

This is due to the fact that RGD peptides are naturally occurring molecules that are recognized by the body, and the lipid component of the delivery system is biocompatible and less harmful. (avantilipids.com)
The size of the cationic lipid and DNA complex (CL-DNA), or the lipoplex, is one of the important variations for consistency of gene transfection. (rsc.org)
Here, we present a confocal microscopy-based method to quantify cytosolic delivery of fluorescently labeled siRNA during lipid-mediated delivery. (lu.se)

This feature enables genes to be easily and efficiently mobilised into target genomes. (bioscience.co.uk)

Moreover, the liposome component of pDNA/CDG2-LHNPs increased its cell uptake efficiency , and the CDG2 polymer component increased its proton buffer capacity, so the hybrid nanoparticles taken up by CME could also successfully escape from the lysosome . (bvsalud.org)

Lacking a complete mechanistic picture of the effects of polymer composition and architecture on its efficiency and toxicity, polymer chemists rely on iterative â one-polymer-at-a-timeâ approaches that are often plagued by trial and error. (aiche.org)

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)
Gene therapy viruses are non-replicating, but still hijack host cell machinery to express transgenes of interest in the nucleus. (frontiersin.org)

The coefficient of variation ( CV ) of transfection efficiency is 0.05 and 0.30 for PMRI-based and conventional methods, respectively. (rsc.org)

CDG2-LHNPs showed significantly superior transfection efficiencies compared to either CDG2 or liposomes . (bvsalud.org)

A research team from Case Western Reserve University School of Medicine has discovered an approach that could make gene therapy dramatically more effective for patients. (health.am)

This route is minimally invasive and therefore clinically relevant for gene therapy targeting to peripheral nerve soma. (frontiersin.org)

The purpose of this study is to develop viable and biocompatible nanoparticles for astrocyte-targeted gene therapy. (tdl.org)

However, there is no single non-viral gene delivery strategy that "fits-all" cell types and tissues. (ku.edu)
However, one of the challenges in AAV-mediated gene therapy is achieving efficient targeting of specific cell types and tissues. (lu.se)

Dynamic PolyConjugates for targeted in vivo delivery of siRNA to hepatocytes. (wikipedia.org)

Organisms2

Chemicals and Drugs8

Analytical, Diagnostic and Therapeutic Techniques and Equipment10

Phenomena and Processes6

Technology, Industry, Agriculture4

Information Science2

Vectors25

Viability5

Nanoparticles for gene delivery1

Vitro8

Nucleic6

CRISPR-Cas96

Advancing gene therapy1

Nonviral3

Proteins4

Transduction8

Vector10

Plasmid4

SiRNA delivery1

Polymers4

Diseases10

Therapies6

Cells20

Editing11

Genetic5

Cellular uptake1

Drug and gene delivery2

Involves1

Expression8

Therapeutics3

Clinical3

Lipids1

Inhibit1

High3

Widely2

Transfection reagents2

Scope of gene therapy1

Cystic fibrosis1

Lipid3

Enables1

Uptake1

Polymer1

Viruses2

Methods1

Significantly1

Make gene therapy1

Clinically1

Viable1

Cell types and tissues2

Vivo delivery1