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.
Precision NanoSystems has launched the NanoAssemblr™ Scale-Up system to support the clinical development of nanomedicines.
Spherical nucleic acids are structures that are made by taking a nanoparticle template and using chemistry to arrange short strands of DNA or RNA on the surface of those particles. The spherical core of the nanoparticle creates a spherical arrangement of DNA or RNA, similar to tiny little balls of nucleic acids.
Timothy Blake, a postdoctoral fellow in the Waymouth lab, was hard at work on a fantastical interdisciplinary experiment. He and his fellow researchers were refining compounds that would carry instructions for assembling the protein that makes fireflies light up and deliver them into the cells of an anesthetized mouse. If their technique worked, the mouse would glow in the dark.
Working with human immune cells in the laboratory, Johns Hopkins researchers report they have identified a critical cellular "off" switch for the inflammatory immune response that contributes to lung-constricting asthma attacks.
In the fight against cancer, doctors dish out combination-blows of surgery, chemotherapy and other drugs to beat back a merciless foe. Now, scientists have taken early steps toward adding a stinging punch to clinicians' repertoire.
Osteoarthritis is a debilitating condition that affects at least 27 million people in the United States, and at least 12 percent of osteoarthritis cases stem from earlier injuries.
A tiny therapeutic delivery system that can control the body’s ability to manufacture proteins has been developed by Saudi Arabia's King Abdullah University of Science and Technology (KAUST) researchers.
Scientists have engineered a sort of biological barbell that can get inside cancer cells and do damage to two proteins that work independently and together to enable cancer's survival and spread.
Many gene mutations that cause neurological disorders have been identified. For example, in a rare neurological disorder such as Huntington’s disease, an autosomal dominant mutation through expansion of CAG (cytosine-adenine-guanine) triplet repeats in the gene coding for the Huntingtin protein results in abnormal protein production.
Currently there is no product available based on polymer film containing active molecules to accelerate dermal wound healing that also reduces the scar formation.
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today preliminary results from its ongoing Phase 2 open-label extension (OLE) studies with patisiran and revusiran, both investigational RNAi therapeutics targeting transthyretin (TTR) for the treatment of hereditary TTR-mediated amyloidosis (hATTR amyloidosis).
Combining the therapeutic potential and advantages of existing oligonucleotide-based approaches to turn off disease-related genes, a type of single-stranded silencing RNAs (ss-siRNAs) has shown significantly improved potency and activity.
Infections continue to threaten human health. With remarkable genetic flexibility, pathogenic organisms outsmart available therapies. Fortunately, microbial versatility is matched by the host immune system, which evolves in dialogue with the microbes. Therapies that enhance the beneficial effects of the immune response represent a promising, but under-explored, therapeutic alternative to antibiotics.
The closely related Hendra and Nipah viruses (referred to jointly as henipaviruses) are deadly cousins of the more common mumps, measles, and respiratory syncytial viruses, all members of the paramyxovirus family. Henipavirus outbreaks are on the rise, but little is known about them, partly because research has to be conducted under extreme level containment conditions.
microRNAs (miRs) are small endogenous noncoding RNA molecules (20–23 nucleotides) derived from imperfectly paired hairpin RNA structures naturally encoded in the genome that act specifically as triggering molecules to control translational repression or mRNA degradation.
Researchers show that the protein CCN4 positively regulates the generation of cartilage matrix, which are depleted in osteoarthritis.
DNA, RNA, protein -- the end. Or is it? Until recently, the pattern used to encode genetic information into our cells was considered to be relatively straightforward: four letters (A,G,C,T) for DNA and four (A,G,C,U) for RNA. This equation, however, turned out to be oversimplified -- RNA was holding out.
RXi Pharmaceuticals Corporation a biotechnology company focused on discovering and developing innovative therapies primarily in the areas of dermatology and ophthalmology today announced the initiation of a Phase 1/2 clinical trial in ophthalmology. The clinical study RXI-109-1501 will evaluate the safety and clinical activity of RXI-109 to prevent the progression of retinal scarring, a harmful component of numerous retinal diseases.
Alnylam Pharmaceuticals, Inc. has announced new results from its ongoing Phase 2 open-label extension (OLE) studies with patisiran and revusiran, investigational RNAi therapeutics targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR amyloidosis).
RXi Pharmaceuticals Corporation, a biotechnology company that owns a broad intellectual property portfolio including a unique self-delivering RNAi platform, today announced that results from a blinded panel and an Investigator review show that incision sites treated with RXI-109 after scar revision surgery achieved better scores as compared to control incision sites in the same subjects, three months post scar revision surgery.