• RNA-based therapeutics, including siRNA, miRNA, mRNA and CRISPR/Cas9 components, have unprecedented therapeutic potential 1) and hold the promise of treating any disease with a genetic component. (phospholipid-research-center.com)
  • The RNA therapeutics market is believed to overtake conventional therapies and is expected to become one of the fastest growing therapeutic classes in the pharmaceutical market by 2020. (phospholipid-research-center.com)
  • Endosomal escape and subsequent cytosolic delivery of small interfering RNA (siRNA) therapeutics is believed to be highly inefficient. (lu.se)
  • 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 potential value of EVs to therapeutics is thought to be enormous, thanks to the role of EVs in cell-cell communication as a robust, native delivery vehicle. (asbmb.org)
  • In March, the Company presented its Axiomer RNA editing technology platform at the 8 th annual Oligonucleotide and Precision Therapeutics (OPT) Congress and at the bi-annual RNA Editing Gordon Research Conference (GRC) . (globenewswire.com)
  • We are actively seeking a commercial partner with experience in RNA therapeutics and targeted drug delivery to help rapidly commercialize this technology. (harvard.edu)
  • For example, Arcturus Therapeutics, an American mRNA therapeutic developer, established a Japanese company in Chiba Prefecture in April 2021 through a joint venture with Axcelead, Inc. and is currently constructing a production facility in Minamisoma City, Fukushima Prefecture. (businesswire.com)
  • RNAimmune is a biopharmaceutical company specializing in discovery and development of messenger RNA (mRNA) therapeutics and vaccines. (prnewswire.com)
  • The therapeutic areas of focus include oncology and anti-fibrotic therapeutics. (prnewswire.com)
  • Mina Therapeutics, a subsidiary of MiNA (Holdings) Ltd, is a developer of small activating ribonucleic acid therapeutics that selectively upregulate therapeutic proteins. (pharmaceutical-technology.com)
  • Mina Therapeutics develops therapeutics using its proprietary RNA activation technology platform. (pharmaceutical-technology.com)
  • This article provides an overview of the major pharmaceutical challenges facing siRNA therapeutics, focusing on the delivery strategies for synthetic siRNA duplexes in vivo, as this remains one of the most important issues to be resolved. (jci.org)
  • Researchers at Regulus Therapeutics described how one such miRNA (miR-21) may provide a novel therapeutic target for hepatocellular carcinoma. (genengnews.com)
  • Aera Therapeutics is announcing its launch today, they said, with "a vision to harness its proprietary delivery platform to unlock the potential of genetic medicines. (genengnews.com)
  • Cloverleaf Bio has developed a new type of RNA therapeutics, based on tRNAs. (massbio.org)
  • Officials of Kylin Therapeutics Inc. has announced on Thursday (July 5) that it has obtained from Purdue University an exclusive license to a RNA interference (RNAi) delivery technology called "pRNA. (technologynetworks.com)
  • pRNA solves this problem for RNAi and other RNA-based therapeutics. (technologynetworks.com)
  • Kylin's Therapeutics lead product is a pRNA-based therapeutic to treat cancer, and the company's initial plans are to focus on cancer therapy, with follow-on treatments for viral diseases such as AIDS. (technologynetworks.com)
  • SAN DIEGO , Feb. 9, 2017 /PRNewswire/ -- Arcturus Therapeutics, Inc. ("Arcturus" or the "Company"), a leading RNA medicines company, today announced that President & CEO Joseph E. Payne will present a corporate update at the 2017 Annual BIO CEO & Investor Conference on Monday, February 13 th at 1:00 p.m. (prnewswire.com)
  • Arcturus has several partnered and fully funded RNA therapeutics in its pipeline (including HBV, NASH, and orphan diseases), and is appraising investors interested in the Company's wholly owned rare disease messenger RNA programs. (prnewswire.com)
  • Founded in 2013 and based in San Diego , Arcturus Therapeutics, Inc. is an RNA medicines company with enabling technologies - UNA Oligomer chemistry and LUNAR™ nanoparticle delivery. (prnewswire.com)
  • Arcturus' versatile RNA therapeutics platform can be applied toward all types of RNA medicines including small interfering RNA, messenger RNA, antisense RNA, microRNA and gene editing therapeutics. (prnewswire.com)
  • The Company's commitment to the development of novel RNA therapeutics has led to significant partnerships with Janssen Pharmaceuticals , Inc., part of the Janssen Pharmaceutical Companies of Johnson & Johnson, Ultragenyx Pharmaceutical , Inc., and Takeda Pharmaceutical Inc. For more information, visit www.ArcturusRx.com . (prnewswire.com)
  • The fragments integrate into a multi-subunit protein called the RNA-induced silencing complex, which contains Argonaute proteins that are essential components of the RNAi pathway. (wikipedia.org)
  • Exosomes contain various molecular constituents of their cell of origin, including proteins and RNA. (exosome-rna.com)
  • Antisense oligonucleotide (ASO) therapy has the potential to ameliorate many neurodegenerative diseases at the genetic level to suppress the production of harmful proteins or non-coding RNAs. (news-medical.net)
  • Dr. Zhang's lab recently published an article in Nature showing how Fanzor proteins can be programmed to precisely target DNA using RNA as a guide. (dlapiper.com)
  • Dr. Zhang and his colleagues have filed patent applications with claims to engineered Fanzor proteins, guide RNAs, and associated vector systems. (dlapiper.com)
  • They've named this class of compounds HERMES, for Horizontally-transferred Eukaryotic RNA-guided Mobile Element Systems, to reflect their findings that these proteins likely jumped from prokaryotes to eukaryotes and then diversified. (dlapiper.com)
  • They also have demonstrated that these HERMES proteins can be programed to edit the genome of human cells, which suggests that this large class of eukaryotic RNA-guided nucleases could be designed for use in gene editing applications. (dlapiper.com)
  • 2011) Expression of therapeutic proteins after delivery of chemically modified mRNA in mice. (jenabioscience.com)
  • Apart from proteins and lipids, SEVs contain RNAs, but how RNAs are selectively loaded into SEVs remains poorly understood. (izon.com)
  • Aera's strategy is to harness these proteins, and structures, to move the cargo of genetic medicines: RNAi, antisense RNA, mRNA, or a genetic editing payload, for example. (genengnews.com)
  • Overall, this study proposes a new strategy for the bacteria-mediated delivery of therapeutic proteins to tumors. (nature.com)
  • Extracellular vesicles (EVs) have emerged as important mediators of intercellular communication due to their ability to transfer bioactive lipids, proteins and different species of RNA into cells. (selectbiosciences.com)
  • Researchers have now created a new way to deliver various types of RNA cargo to cells, which utilizes one of the human body's natural proteins to create particles that can bind to RNA. (labroots.com)
  • Zhang's team knew that these proteins can bind and package different molecules, so they began to study them as delivery systems. (labroots.com)
  • Binding of viral proteins to their ligands activates signaling pathways-focal adhesion kinase for human herpesvirus 8, JAK1, tyk2 and STAT1/2 for West Nile virus (WNV), I κ B-kinase, and nuclear transcription factor κ B (NF-κB) for influenza virus-resulting in virus entry, DNA delivery to nucleus, and modulation of protein expression that stimulate or inhibit virus replication. (cdc.gov)
  • They are naturally equipped to transfer RNA molecules to other cells in a functional and selective manner, thereby potentially outperforming synthetic RNA nanocarriers. (phospholipid-research-center.com)
  • This study demonstrates the versatility of cESG as a platform for dual delivery of small molecules and oligonucleotides, and the potential for further development of this system in combination therapy applications. (nih.gov)
  • These molecules can be composed of single-stranded DNA or RNA and are generally 13-25 nucleotides long. (wikipedia.org)
  • Ribozymes are catalytic RNA molecules used to inhibit gene expression. (wikipedia.org)
  • Sidney Altman and Thomas Cech first discovered catalytic RNA molecules, RNase P and group II intron ribozymes, in 1989 and won the Nobel Prize for their discovery. (wikipedia.org)
  • These catalytic RNA molecules bind to a specific site and attack the neighboring phosphate in the RNA backbone with their 2' oxygen, which acts as a nucleophile, resulting in the formation of cleaved products with a 2'3'-cyclic phosphate and a 5' hydroxyl terminal end. (wikipedia.org)
  • Recent technological advancements have further enhanced mRNA stability and improved cell delivery by encapsulating the molecules in lipids, which in turn boosts spike protein production and enhances the immunological response. (businesswire.com)
  • To do this, the scientists based their sequence inputs on microRNA, small RNA molecules that regulate cellular gene expression. (udel.edu)
  • RNAi is activated when RNA molecules occur as double-stranded pairs in the cell. (europa.eu)
  • Double-stranded RNA (dsRNA) activates a biochemical machinery into the cell which degrades those mRNA molecules with a genetic code complementary to that of the dsRNA. (europa.eu)
  • RNA interference can be induced by synthetic short double-stranded RNA molecules containing around 21-25 nucleotides (short interfering RNAs - siRNAs). (europa.eu)
  • Such silencing RNA molecules can be introduced into the cell and activate the RNA interference machinery to silence the complementary mRNA sequence. (europa.eu)
  • Chemical modifications to provide drug-like properties to RNA molecules are used in the synthesis of both siRNAs and antimicroRNAs (antimiRs). (genengnews.com)
  • Because alternative RNA splicing allows different types of mRNA molecules to be created from a single gene, it generates the diversity of protein function and structure that is essential to complex organisms. (genomicglossaries.com)
  • RNA molecules which hybridize to complementary sequences in either RNA or DNA altering the function of the latter. (genomicglossaries.com)
  • More specifically, antisense oligonucleotides that are useful as reagents for target validation , or as drugs, are engineered molecules that differ from natural RNA but that have a base sequence that is recognized as being complementary to a very specific mRNA sequence. (genomicglossaries.com)
  • It enables in situ delivery of the produced bioactive molecules to tumor site, which improves the therapeutic efficacy. (nature.com)
  • This protein can bind to RNA and will typically form a capsule around RNA molecules, protecting them from degradation. (labroots.com)
  • it can apparently be used to package therapeutic RNA molecules and deliver them to cells. (labroots.com)
  • Then they engineered PEG10's untranslated regions to add various RNA molecules that would also be identified and packaged by PEG10. (labroots.com)
  • The process to silence genes first begins with the entrance of a double-stranded RNA (dsRNA) molecule into the cell, which triggers the RNAi pathway. (wikipedia.org)
  • 2021) Cholesterol-functionalized DNA/RNA heteroduplexes cross the blood-brain barrier and knock down genes in the rodent CNS. (news-medical.net)
  • Cells can inhibit the expression of intrinsic or foreign genes at the mRNA level by RNA interference. (europa.eu)
  • RNA interference occurs in plants, animals, and humans and it is already being used as a method to study the function of genes and as a defence strategy against diseases. (europa.eu)
  • Synthetic antisense RNAs are used to effect the functioning of specific genes for investigative or therapeutic purposes. (genomicglossaries.com)
  • USF researchers have developed a novel method that delivers genes or small interfering RNA into the brain by nasal insufflations using nanoparticles. (usf.edu)
  • The current method of delivering therapeutic genes or viral vectors into the brain is by minimally invasive neurosurgical injection. (usf.edu)
  • The emerging technology known as targeted therapy facilitates selective delivery of chemotherapeutic agents involving therapeutic genes and drugs to tumor sites, which enhances the therapeutic efficacy and reduces side effects 2 . (nature.com)
  • Her primary focus is on the therapeutic application of short RNA double-strands - so-called siRNAs - which can silence genes involved in pathogenesis in specific cell types. (uni-muenchen.de)
  • Through the use of small interfering RNA (siRNA), RNAi-based technologies can be used to silence disease-causing genes. (technologynetworks.com)
  • Arcturus' proprietary UNA technology can be used to target any gene in the human genome, as well as viral genes, and other species for therapeutic purposes. (prnewswire.com)
  • A molecular phenomenon called RNA interference (RNAi) -in which small pieces of RNA silence the expression of individual genes-could provide an alternative solution for breast and other cancers. (vectorblog.org)
  • Though it was first discovered in plants, researchers have known for about a decade that small interfering RNAs (siRNAs) are active in mammals like us, and are already working on ways to harness them for shutting down genes promoting cancer and other diseases. (vectorblog.org)
  • ClinGen definitively disease-associated inherited heart disease genes were amplified using RNA extracted from fresh blood, derived cardiomyocytes, and myectomy tissue. (cdc.gov)
  • 2003). The therapeutic effects of different genes delivered by ment of the hGFAP promoter used here has been extensively studied and lentiviral vectors have been documented in a number of animal found to be up-regulated after several different stimuli both in vitro and models, both in rodents and primates, and examples include delivery in vivo in transgenic mice (Brenner et al. (lu.se)
  • RNA interference (RNAi) is a natural process used by cells to regulate gene expression. (wikipedia.org)
  • The strategy is based on the RNA interference (RNAi), a process which has been the winning research of the Nobel Prize in Physiology or Medicine in 2006. (europa.eu)
  • Some of the same strategies already developed for delivery of siRNA for RNA interference (RNAi) also are being applied to miRNA. (genengnews.com)
  • RNAi was the subject of the 2006 Nobel Prize for Medicine and Physiology and is regarded as one of the most important therapeutic discovery in decades," Guo said. (technologynetworks.com)
  • To achieve widespread delivery of siRNAs to the brain they have peripherally injected modified exosomes expressing Ravies virus glycoprotein loaded with siRNA. (exosome-rna.com)
  • Both miRNAs and siRNAs need to be delivered into the target tissue or cell in order to activate the desired therapeutic effect," Muthiah Manoharan, Ph.D., senior vp, drug discovery, Alnylam Pharmaceuticals, explained. (genengnews.com)
  • According to Dr. Manoharan, Alnylam is utilizing second-generation LNPs to enhance delivery of siRNAs in several models. (genengnews.com)
  • We conjugated an N-acetylgalactosamine moiety (GalNac) to siRNAs and achieved improved potency using subcutaneous delivery at low, clinically relevant doses. (genengnews.com)
  • Alnylam Pharmaceuticals is using its lipid nanoparticles platform to enhance delivery of siRNAs in several models. (genengnews.com)
  • Small interfering RNAs, or siRNAs, could be great targeted treatment tools for breast and other cancers. (vectorblog.org)
  • Genomic Imprinting Paramutation Transposon silencing (or Histone Modifications) Transgene silencing Position effect RNA-directed DNA methylation RNA interference RNA silencing Nonsense mediated decay Transvection Meiotic silencing of unpaired DNA Antisense oligonucleotides were discovered in 1978 by Paul Zamecnik and Mary Stephenson. (wikipedia.org)
  • The majority of antisense drugs function through the RNase H-dependent mechanism, in which RNase H hydrolyzes the RNA strand of the DNA/RNA heteroduplex. (wikipedia.org)
  • Endogenous antisense RNAs function as regulators of gene expression by a variety of mechanisms. (genomicglossaries.com)
  • Collectively, this information is essential for the selection of optimally acting siRNA delivery system combinations for the many proposed applications of RNA interference. (jci.org)
  • Recently, an endogenous RNA transport system has emerged, based on the release and uptake of small, phospholipid membrane-enclosed entities called extracellular vesicles (EVs) or exosomes . (phospholipid-research-center.com)
  • The oppositions were filed in February 2021 with the European Patent Office (EPO) by two separate strawmen against ProQR's granted patent EP 3234134 B1 , which is related to targeted RNA editing using endogenous ADARs and is part of ProQR's intellectual property estate surrounding its Axiomer RNA editing platform. (globenewswire.com)
  • They described the development of selective endogenous encapsidation for cellular delivery (or SEND) by engineering an LTR retrotransposon homolog (PEG10) to package, secrete, and deliver specific RNAs. (genengnews.com)
  • The new SEND technique (Selective Endogenous eNcapsidation for cellular Delivery) takes advantage of a natural protein encoded in the human genome called PEG10. (labroots.com)
  • An aspect of gene delivery to the brain that is currently being regulate the endogenous GFAP expression in a lesioned brain. (lu.se)
  • 87(5): 1663-1667] Specifically, the investigators started by priming whole cerebellar RNA with a synthetic oligonucleotide containing a T7 RNA polymerase promoter sequence. (genomicglossaries.com)
  • The research team had recently developed DNA/RNA heteroduplex oligonucleotide (HDO) technology capable of highly efficient RNA degradation in vivo. (news-medical.net)
  • Continuing developments in oligonucleotide research have begun to unleash their potential as therapeutic agents. (waterstones.com)
  • These small fragments, which include small interfering RNAs (siRNA) and microRNA (miRNA), are approximately 21-23 nucleotides in length. (wikipedia.org)
  • In the case of lung cancer, "delivery of miR-200a and -200b members resulted in a significant decrease in primary tumor burden as compared with control miRNA," they wrote. (genomeweb.com)
  • In normal mice the researchers we detected significantly reduced α-Syn messenger RNA (mRNA) and protein levels throughout the brain 3 and 7 days after treatment with RVG-exosomes loaded with siRNA to α-Syn. (exosome-rna.com)
  • This is the final seminar of a series of Biologics TIN events to showcase innovative technologies developed at UCL for therapeutic applications involving oligonucleotides, antibodies, vaccines, peptides, exosomes and how to turn them in a biopharmaceutical product. (exosome-rna.com)
  • These vaccines are relatively simple to manufacture, with RNA, their active component, often produced in vitro using linear DNA as a template. (businesswire.com)
  • Through processes like codon optimization, nucleoside modification, and improved delivery methods, mRNA stability and translation efficiency can be enhanced, resulting in nucleic acid vaccines with high specificity and durable stability. (businesswire.com)
  • mRNA vaccines have paved the way for new opportunities in pharmacology and have become a significant therapeutic category. (businesswire.com)
  • Lawyer Tom Renz has exposed that the COVID-19 vaccines, widely advertised as mRNA (messenger RNA) vaccines, are in fact lab-created hybrids known as modRNA. (republicbroadcasting.org)
  • They claimed the COVID-19 vaccines were mRNA & that meant MESSENGER RNA (which occurs in life everywhere). (republicbroadcasting.org)
  • Immunosuppressives may diminish therapeutic effects of vaccines and increase risk of adverse effects (increased risk of infection). (medscape.com)
  • Simultaneous delivery of TPPS and siRNA was achieved, reducing Sod2 protein expression to 48%, while maintaining the photodynamic properties of TPPS under light exposure and maintaining low dark toxicity. (nih.gov)
  • Administration of a CRISPR-based therapeutic could trigger immune reactions to the Cas9 enzyme, a large foreign protein. (dlapiper.com)
  • The DNA circuit and protein activity was turned "on" by specific RNA/DNA sequence inputs, while in the absence of said inputs the system stayed "off. (udel.edu)
  • Tezcat Biosciences is developing a portfolio of protein-drug conjugates to provide therapeutic options for patients harboring mutant RAS cancers. (massbio.org)
  • Small Extracellular Vesicle Enrichment of a Retrotransposon-Derived Double-Stranded RNA: A Means to Avoid Autoinflammation? (izon.com)
  • RNA folding analyses indicate that the motif is likely to form a long double-stranded RNA hairpin and, consistent with this, its overexpression was associated with induction of a potent type I interferon response. (izon.com)
  • By integrating multiple established platform technologies, RNAimmune is aiming to develop a comprehensive mRNA drug discovery and development platform, from which the Company will enrich its therapeutic and vaccine product pipeline addressing tremendous unmet needs in treatments of viral infections, cancer and rare diseases. (prnewswire.com)
  • These include viral and nonviral methods of gene delivery, transgenic approaches, strategies for the regulation of transgene expression, and modification of the host response. (cshlpress.com)
  • In vivo delivery requires administration of the gene editing system to the patient, typically through viral vectors or nanoparticles. (dlapiper.com)
  • Guide RNAs and the viral vectors used for the delivery of the CRISPR-Cas9 also have immunogenic potential. (dlapiper.com)
  • Furthermore, to facilitate the transfer of the functional interfering RNA sequences to infected animals, we have constructed a recombinant DNA viral vector commonly used as a vaccine. (europa.eu)
  • After transcription, the shRNA is processed into active 21-nucleotides RNAs by a cellular enzyme called Dicer, and it recognises and destroys the viral mRNA target. (europa.eu)
  • Cell-derived vesicles (CDVs) are one of the most promising non-viral drug delivery platforms. (selectbiosciences.com)
  • Viral proteases (HCV and SARS-CoV) and viral RNA-dependent RNA polymerases (Lassa virus, HCV, WNV) are major targets, but treatments targeting viral enzymes often induce the rapid emergence of drug-resistant viruses. (cdc.gov)
  • The company's lead therapeutic, MTL-CEBPA, is developed for the treatment of liver cancer. (pharmaceutical-technology.com)
  • Entrada anticipates that its cash, cash equivalents and marketable securities as of March 31, 2023, together with ongoing research support and the anticipated achievement of certain milestones under the Vertex Agreement, will be sufficient to extend its cash runway into the second half of 2025, supporting the Company's expansion and continued development of EEV-therapeutic candidates targeting Duchenne as well as other indications beyond neuromuscular diseases. (biospace.com)
  • Sequence-specific gene silencing using small interfering RNA (siRNA) is a Nobel prize-winning technology that is now being evaluated in clinical trials as a potentially novel therapeutic strategy. (jci.org)
  • Built using a translational lens with a multi-omics approach, its proprietary discovery platform uncovers novel therapeutic avenues previously unavailable to patients with complex diseases. (massbio.org)
  • We believe this platform has broad applicability across multiple therapeutic areas and, based on the encouraging initial in vivo data we've generated, look forward to advancing initial programs for diseases that originate in the liver. (globenewswire.com)
  • Improvements of lipid nanoparticle formulations as a vehicle for in vivo systemic delivery of mRNA has greatly favored the development of in vivo transfection strategies. (pipelinereview.com)
  • The pharmaceutical challenges to effective siRNA delivery and activity in vivo. (jci.org)
  • The challenges for effective siRNA delivery and activity in vivo range from the effective empirical or in silico design and selection of an effective siRNA sequence to its chemical modification or formulation with delivery vectors to improve biological stability and pharmacokinetics (steps i-iv). (jci.org)
  • Cationic enzymatically synthesized glycogen (cESG) is a naturally-derived, nano-scale carbohydrate dendrite that has shown promise as a cellular delivery vehicle owing to its flexibility in chemical modifications, biocompatibility and relative low cost. (nih.gov)
  • In the present work, cESG was modified and evaluated as a vehicle for tetraphenylporphinesulfonate (TPPS) in order to improve cellular delivery of this photosensitizer and investigate the feasibility of co-delivery with short interfering ribonucleic acid (siRNA). (nih.gov)
  • Adapting EVs for drug delivery requires cellular engineering that allows for selective loading of biotherapeutics inside or on the surface of EVs. (selectbiosciences.com)
  • Lieberman's laboratory in the Program in Cellular and Molecular Medicine at Boston Children's Hospital and the Immune Disease Institute (PCMM/IDI) has worked for several years to exploit RNAi's therapeutic potential. (vectorblog.org)
  • These naturally occurring, noncoding, single-stranded RNAs (21-25 nucleotides long) base-pair with their target mRNA within the RISC (RNA-induced silencing complex). (genengnews.com)
  • Dr. Zeiher has over 25 years of experience in the pharmaceutical industry and has supported the development and approval of 15 products across multiple therapeutic areas. (biospace.com)
  • In her new project RatInhalRNA (Rational and Simulation-Supported Design of Inhalable RNA Nanocarriers) , Merkel is seeking to develop efficient siRNA-based drugs that can be administered to the lungs through inhalation. (uni-muenchen.de)
  • RNA which contains an intron sequence that has an enzyme- like catalytic activity. (genomicglossaries.com)
  • Aera's technology also includes a licensed therapeutic enzyme platform based on the discovery of novel, compact, and programmable gene-editing enzymes. (genengnews.com)
  • Wyss researchers have discovered a new class of immunostimulatory double-stranded RNAs (dsRNAs) that produce pan-antiviral and anti-cancer activity by inducing the expression of multiple types of IFNs. (harvard.edu)
  • Taken together, we propose that preferential loading into SEVs of the VL30 RNA containing this immunostimulatory motif enables cells to remove a potentially toxic RNA and avoid autoinflammation. (izon.com)
  • Researchers from the University College London have used α-Syn small interfering RNA (siRNA) to reduce total and aggregated α-Syn levels in mouse brains. (exosome-rna.com)
  • Future research will help define the specific molecular pathways thus optimizing delivery of ASO pharmacotherapy to the CNS. (news-medical.net)
  • Consequently, there is an urgent need for novel, original approaches to overcome the delivery challenges. (phospholipid-research-center.com)
  • This review focuses on the barriers that impact pulmonary siRNA delivery and successful recent approaches to advance this field forward. (cdc.gov)
  • Gene Transfer: Delivery and Expression of DNA and RNA provides the first comprehensive guide to technical approaches for delivering nucleic acids into cells and organisms and of ensuring (even manipulating) appropriate expression. (cshlpress.com)
  • A session was dedicated to new preventive or therapeutic vaccine approaches. (cdc.gov)
  • High amounts of these vesicles are produced through mechanical extrusion of cells and used to carry payloads with therapeutic benefit. (selectbiosciences.com)
  • Now, researchers from Japan and the USA have developed a drug delivery platform that overcomes this hurdle. (news-medical.net)
  • Researchers at Stanford University used such a method to produce amplified heterogeneous populations of RNA from limited quantities of cDNA. (genomicglossaries.com)
  • More recently, another group of researchers reported that they had developed a process for optimizing low- abundance RNA, by combining aRNA amplification with template- switching. (genomicglossaries.com)
  • Also, the nanoparticles can be visualized by MRI and other similar techniques, allowing clinicians and researchers to visualize the uptake of the nanoparticles and the distribution of the therapeutic. (usf.edu)
  • The researchers then tested the therapeutic effects of miR-200 in orthotopic cancer models. (genomeweb.com)
  • The last line of the paper's abstract noted that "these results demonstrate that SEND is a modular platform suited for development as an efficient therapeutic delivery modality. (genengnews.com)
  • Abstract: Disclosed are therapeutic compounds, protocols, and compositions of matter useful for treatment of neurological conditions. (therapeuticsolutionsint.com)
  • Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer ® RNA editing technology platform, today reported its financial and operating results for the first quarter ended March 31, 2023, and provided a business update. (globenewswire.com)
  • RNAimmune is a spin-off venture from Sirnaomics, Inc. and has received a global exclusive right to the proprietary Polypeptide Lipid Nanoparticle (PLNP) technology for mRNA delivery from Sirnaomics. (prnewswire.com)
  • Its proprietary therapeutic delivery platform can deliver RNA, gene editing, and other therapeutic options to hard-to-penetrate tissues such as cartilage, kidney, brain, and some solid tumors. (massbio.org)
  • Cancer prodrugs are inactive until they are metabolized into their therapeutic form. (udel.edu)
  • With an eye toward clinical applications of the miR-200 family, Sood and his colleagues tested the effects of miR-200 delivery in mouse lung cancer models. (genomeweb.com)
  • The delivery of genetic payloads into cells is a huge barrier to gene therapy and genomic medicines. (genengnews.com)
  • Pulmonary delivery of siRNA-based biopharmaceuticals offers the potential to address multiple unmet medical needs in lung-related diseases because of the specific physiology of the lung and characteristic properties of siRNA. (cdc.gov)
  • Our innovative therapeutic platform for blood-to-brain delivery of ASOs may revolutionize management of neurodegenerative diseases,' senior author Takanori Yokota claims. (news-medical.net)
  • In conclusion, MSCs in musculoskeletal system, mediated by the interaction between MSC paracrine and Mφs, have therapeutic potential in musculoskeletal diseases. (frontiersin.org)
  • The 96 patents that USF inventors earned in 2018 was tops for any Florida university, and span a wide array of technologies - from clean energy solutions to sustainable water technologies to nanoscale particles designed to improve drug delivery in the fight against central nervous system diseases. (usf.edu)
  • This study highlights the therapeutic potential of RVG-exosome delivery of siRNA to delay and reverse brain α-Syn pathological conditions. (exosome-rna.com)
  • cESG-TPPS was then further evaluated as a co-delivery system with siRNA for potential combination therapy, by charge-based condensation of an siRNA directed at reducing expression of manganese superoxide dismutase (Sod2) as a proof of principle target. (nih.gov)
  • These programs share several key characteristics including a deep rooting in human genetics, the potential to have a major impact in indications with high unmet medical need, the ability to leverage the existing proven delivery technology to the liver, the opportunity to monitor early biomarkers to establish target engagement in Phase I trials for human proof of concept, and the availability of well-defined clinical endpoints. (globenewswire.com)
  • Although mesenchymal stem cells (MSCs) have been shown as a novel approach in tissue regeneration, the therapeutic potential of MSCs mediated by the interaction between MSC-derived paracrine mediators and Mφs remains elusive. (frontiersin.org)
  • Our results suggest the potential use of the Ad-shRNAN1 for the delivery of siRNA in PPRV-infected animals, its use as a therapeutic tool against this disease and to open up vast prospects in terms of animal health. (europa.eu)
  • The therapeutic potential of BACE will also be considered. (jneurosci.org)
  • Approval of the first siRNA treatments in humans has opened the door for further exploration of this therapeutic strategy for other disease states. (cdc.gov)
  • The numerous benefits mRNA offers over other therapeutic modalities, such as higher biological efficacy, enhanced potent immunogenicity, and versatile delivery platforms at low toxicity levels, are the main drivers of the ongoing research and development efforts being made in this field. (businesswire.com)
  • Recent advances have provided a variety of new strategies for delivering DNA and RNA into cells and probing their expression, as well as new clinical applications that rely upon the introduction of genetic material. (cshlpress.com)
  • This presentation will cover our recent developments in EV engineering, with focus on active loading and functional delivery of gene editing modalities. (selectbiosciences.com)
  • A multitude of randomized clinical trials and other treatment studies have demonstrated that currently available therapeutic methods are 22%-94% effective in clearing external exophytic genital warts, and that recurrence rates are high (usually at least 25% within 3 months) with all modalities. (cdc.gov)
  • However, inefficient delivery into diseased cells hinders their clinical progress. (phospholipid-research-center.com)
  • Inhalation-based siRNA delivery designed for efficient, targeted delivery to specific cells within the lung holds great promise. (cdc.gov)
  • The introduction of isRNAs into living cells induces IFN-I production in cells' nuclei (right, in light blue), while the introduction of scrambled control RNA does not (left). (harvard.edu)
  • With this adenovirus vector, an active siRNA sequences (N1-PPRV) was transcribed into the infected cells as a short hairpin RNA precursor (shRNA, with approximately 50 nt). (europa.eu)
  • One of the most difficult aspects of gene therapy might be ensuring that it gets into the right cells safely so it can have a therapeutic effect. (labroots.com)
  • We focus on the pulmonary barriers that affect siRNA delivery, the disease-dependent pathological changes and their role in pulmonary disease and impact on siRNA delivery, as well as the recent development on the pulmonary siRNA delivery systems. (cdc.gov)
  • The company collaborates with drug delivery companies for the development of saRNA. (pharmaceutical-technology.com)
  • In February 2023, Entrada announced the closing of its strategic collaboration and license agreement with Vertex for the discovery and development of intracellular EEV-therapeutic candidates for myotonic dystrophy type 1 (DM1). (biospace.com)
  • PathCision's leading program is focused on developing a biologic modality for metastasis-selective delivery for multiple indications. (massbio.org)
  • The therapeutic tool is an adeno-associated virus vector, which shows excellent safety for gene delivery in the brain. (lu.se)
  • Furthermore, recent technological progress in translation, stability, and delivery methods has positioned messenger RNA as a promising therapeutic tool. (businesswire.com)
  • For a long time, messenger RNA (mRNA) was thought to be a poor choice for a therapeutic agent given its relatively short half-life and its immunogenicity. (pipelinereview.com)
  • They are targeting the messenger RNA of the nucleoprotein gene of these viruses, then blocking the virus multiplication process. (europa.eu)
  • modRNA (modified messenger RNA) is a synthesized form of mRNA that has been altered at specific sites. (republicbroadcasting.org)
  • Pfizer admitted that during its clinical studies, participants aged 16 years and older received 30 mcg of nucleoside-modified messenger RNA (modRNA) . (republicbroadcasting.org)
  • Nucleoside-modified messenger RNA (modRNA) is a modified form of mRNA that encodes the spike (S) glycoprotein of the SARS-CoV-2 virus, the virus that causes COVID-19. (republicbroadcasting.org)
  • A complementary RNA sequence that binds to a naturally occurring (sense) mRNA molecule, thus blocking its translation. (genomicglossaries.com)
  • We may also strategize by initiating treatment with intrathecal dosing to rapidly achieve therapeutic concentrations, followed by intravenous or subcutaneous maintenance as needed. (news-medical.net)
  • The therapeutic candidate is administered through intravenous route. (pharmaceutical-technology.com)
  • Thus, BACE is a prime drug target for the therapeutic inhibition of Aβ production in AD. (jneurosci.org)
  • Olivia Merkel is Professor of Drug Delivery at LMU's Department of Pharmacy. (uni-muenchen.de)
  • Since October 2015, she has been a professor at LMU and has been Chair of Drug Delivery since 2022. (uni-muenchen.de)
  • Current delivery systems are not efficient, may integrate their cargo improperly, and can cause serious immune reactions. (labroots.com)
  • Hammerhead, hairpin, and hepatitis delta virus (HDV) ribozyme motifs are generally found in viruses or viroid RNAs. (wikipedia.org)