Small interfering RNA (siRNA), sometimes known as short interfering RNA or silencing RNA, is a class of double-stranded RNA molecules, 20-25 nucleotides in length, that play a variety of roles in biology. Most notably, siRNA is involved in the RNA interference (RNAi) pathway, where it interferes with the expression of a specific gene. In addition to their role in the RNAi pathway, siRNAs also act in RNAi-related pathways, e.g., as an antiviral mechanism or in shaping the chromatin structure of a genome; the complexity of these pathways is only now being elucidated.
Understanding how memories form and are retrieved has applications to psychiatric, neurological and neurodegenerative disorders, and may be helpful to attenuate maladaptive memories in psychiatric disorders.
Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today that the European Commission (EC) has granted marketing authorization for ONPATTRO (patisiran) for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adults with stage 1 or stage 2 polyneuropathy.
Excessive body fat is associated with negative effects on female fertility and pregnancy. In mice, maternal obesity impairs proper development of egg precursor cells called oocytes.
The U.S. Food and Drug Administration today approved Onpattro (patisiran) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients.
Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today that the Committee for Medicinal Products for Human Use has adopted a Positive Opinion recommending marketing authorization of patisiran for the treatment of hereditary transthyretin-mediated amyloidosis in adults with stage 1 or stage 2 polyneuropathy.
Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today new positive results from its Phase 1/2 study with lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase for the treatment of Primary Hyperoxaluria Type 1.
Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today that the Company has achieved delivery of novel small interfering RNA conjugates to the central nervous system and is planning to advance a pipeline of investigational RNAi therapeutics into clinical development.
Not all of your genome needs to be active at any given time. Some regions are prone to hopping around the genome in problematic ways if left unchecked; others code for genes that need to be turned off in certain cells or at certain times.
Sylentis, Pharmaceutical Company belonging to the PharmaMar Group, has presented results from the clinical studies carried out with tivanisiran for the treatment of dry eye syndrome and that has enabled the start-up of the Phase III "Helix" clinical trial.
Porvair Sciences has collaborated with Persomics Inc. to develop and supply ANSI/SLAS format Arrays as an OEM product for their ImagineArray™ platform.
A newly designed three-part molecule could be the one answer patients with a certain form of breast cancer are looking for, scientists report.
The first direct comparison of in vitro and in vivo screening techniques for identifying nanoparticles that may be used to transport therapeutic molecules into cells shows that testing in lab dishes isn't much help in predicting which nanoparticles will successfully enter the cells of living animals.
The biological complexity of cancer and other diseases demands a more formidable arsenal of therapies than currently available. Most therapeutic approaches ignore the dynamic molecular network of genes, targeting instead only very few selected disease-related genes.
A new Tel Aviv University study pinpoints the inverse correlation between a known oncogene -- a gene that promotes the development of cancer -- and the expression of an oncosuppressor microRNA as the reason for extended pancreatic cancer survival.
A new study shows that attaching antibody-like RNA nanoparticles to microvesicles can deliver effective RNA therapeutics such as small interfering RNA specifically to cancer cells.
The Spanish firm Sylentis has developed a compound to treat diseases of the retina, such as age-related macular degeneration and diabetic retinopathy, which will be administered by ophthalmic drops instead of intraocular injections.
Research at the Southwest National Primate Research Center on the campus of Texas Biomedical Research Institute helped advance a new treatment now in human trials for chronic hepatitis B virus (HBV) infection.
Researchers in Georgia State University's Institute for Biomedical Sciences have received a four-year, $1.4 million federal grant to study novel therapeutic approaches for the treatment of intestinal inflammation.
Early phase Northwestern Medicine research has demonstrated a potential new therapeutic strategy for treating deadly glioblastoma brain tumors.
Genetic manipulation of exosomes, virus-sized particles released by all cells, may offer a new therapeutic approach to treating pancreatic cancer, according to a study at The University of Texas MD Anderson Cancer Center.