Gene Silencing News and Research

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Alnylam presents new pre-clinical data with ALN-AT3 for treatment of hemophilia and RBD

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data with ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD), at the 55th Annual Meeting of the American Society of Hematology (ASH) held December 7 - 10, 2013 in New Orleans.

10 Dec 2013

Brown University biologists find way to measure effects of aging

Biologists at Brown University have found a way to measure the effects of aging by watching the ebb and flow of chromatin, a structure along strands of DNA that either silences or permits gene expression. In several newly published experiments they show that gene silencing via chromatin in fruit flies declines with age.

21 Nov 2013

Heidelberg scientists identify protein motifs that influence gene silencing

Bioscientists at Heidelberg University have studied the function of certain proteins, known as Argonaute (Ago) proteins, in the process of gene regulation. They sought to understand why only the Ago2 protein is able to target and directly turn off genes in humans, while the closely related Ago3 protein is not.

16 Nov 2013

Researchers identify novel therapeutic approach for most frequent genetic cause of ALS

A team of scientists led by researchers from the University of California, San Diego School of Medicine and Ludwig Institute for Cancer Research have identified a novel therapeutic approach for the most frequent genetic cause of ALS, a disorder of the regions of the brain and spinal cord that control voluntary muscle movement, and frontal temporal degeneration, the second most frequent dementia.

10 Nov 2013

HSCI researchers discover on-off switch for gene expression

Harvard Stem Cell Institute researchers have settled a century-old debate over whether occurrence of DNA methylation acts to silence gene expression, or if genes are turned off by other means before they are methylated.

11 Oct 2013

Alnylam advances Development Candidate for ALN-AS1 for treatment of hepatic porphyrias

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it is advancing its Development Candidate for ALN-AS1, an RNAi therapeutic targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of hepatic porphyrias including acute intermittent porphyria (AIP).

9 Oct 2013

Metabolic enzymes in opium poppy are used to produce valuable painkilling drugs

University of Calgary scientists have discovered metabolic enzymes in the opium poppy that play "widespread roles" in enabling the plant to make painkilling morphine and codeine, and other important compounds.

5 Oct 2013

FDA grants Orphan Drug Designation to Alnylam ALN-AT3 for treatment of hemophilia B

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that the U.S. Food & Drug Administration (FDA) has granted an Orphan Drug Designation to ALN-AT3 as a therapeutic for the treatment of hemophilia B.

17 Aug 2013

Alnylam releases positive top-line data from ALN-TTRsc Phase I trial for TTR-mediated amyloidosis

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today positive top-line results from its ongoing Phase I trial of ALN-TTRsc, a subcutaneously administered RNAi therapeutic targeting the transthyretin gene for the treatment of TTR-mediated amyloidosis.

12 Jul 2013

Alnylam Pharmaceuticals presents new pre-clinical data of ALN-AT3 at ISTH meeting

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data from its RNAi therapeutic program for the treatment of hemophilia and rare bleeding disorders.

2 Jul 2013

Study suggests new ways to maximize delivery of RNA strands

Nanoparticles that deliver short strands of RNA offer a way to treat cancer and other diseases by shutting off malfunctioning genes. Although this approach has shown some promise, scientists are still not sure exactly what happens to the nanoparticles once they get inside their target cells.

25 Jun 2013

Alnylam presents new pre-clinical data of ALN-CC5 for treatment of complement-mediated diseases

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data with ALN-CC5, an RNAi therapeutic targeting complement component C5.

21 Jun 2013

Bioapplications of nanoparticles: an interview with Dr Catherine Berry, University of Glasgow

These particles are all classed as inorganic particles, which can all be utilised in biomedical applications. They differ in terms of their inherent material and size dependent physiochemical properties, for example, their optical and magnetic properties.

18 Jun 2013

Cenix BioScience, Debiopharm partner to develop novel therapeutic drug candidates

Cenix BioScience, a leading preclinical contract research provider and technology developer specialized in RNAi-, miRNA- and high content-driven pharmacology, and Debiopharm Group, a Swiss-based global biopharmaceutical group of companies with a focus on the development of prescription drugs that target unmet medical needs, including oncology and companion diagnostics, today announced that they have signed a research agreement to support Debiopharm in its ongoing efforts to develop novel therapeutic drug candidates.

23 May 2013

Alnylam reports pre-clinical data from ALN-AS1 program for treatment of AIP

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented key pre-clinical proof-of-concept data from its RNAi therapeutic program targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of porphyria including acute intermittent porphyria.

18 May 2013

Scientists identify new way to boost gene-silencing mechanism

A team led by scientists at The Scripps Research Institute (TSRI) has found how to boost or inhibit a gene-silencing mechanism that normally serves as a major controller of cells' activities. The discovery could lead to a powerful new class of drugs against viral infections, cancers and other diseases.

10 May 2013

Wistar researchers find surprising role for RNA-editing protein in gene silencing

RNA, once considered a bit player in the grand scheme by which genes encode protein, is increasingly seen to have a major role in human genetics.

2 May 2013

Research on miRNAs offers new therapeutic potential to fight diseases

RNA molecules, made from DNA, are best known for their role in protein production. MicroRNAs (miRNAs), however, are short (~22) nucleotide RNA sequences found in plants and animals that do not encode proteins but act in gene regulation and, in the process, impact almost all biological processes - from development to physiology to stress response.

26 Apr 2013

Benitec to conduct phase I/II clinical trial of TT-034 in patients with HCV in UCSD site

RNAi-based therapeutics company Benitec Biopharma Limited today announced the selection of the University of California, San Diego, Health Sciences as the second site for its upcoming phase I/II first-in-man trial for TT-034 in Hepatitis C infections.

16 Apr 2013

Novel nanoparticle-based approach enables more efficient delivery of siRNA drugs

New classes of drugs that can silence specific genes, such as small interfering RNAs (siRNAs), offer great therapeutic potential. But the specific delivery of siRNAs to target cells to exert their effects remains a significant challenge. A novel nanoparticle-based approach that enables more efficient delivery of siRNA drugs is presented in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc. publishers.

12 Apr 2013