Gene Silencing News and Research

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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

Researchers develop predictive biomarker to identify cancer patients who may respond to autophagy inhibitors

Autophagy, the process by which cells that are starved for food resort to chewing up their own damaged proteins and membranes and recycling them into fuel, has emerged as a key pathway that cancer cells use to survive in the face of assault by chemotherapy and radiation.

9 Apr 2013

Three researchers receive 2013 Wiley Prize in Biomedical Sciences

Deborah E. Wiley, Chair of The Wiley Foundation, John Wiley & Sons, Inc. (NYSE: JWa & JWb) today awarded the 2013 Wiley Prize in Biomedical Sciences to Dr. Michael Young, Rockefeller University, Dr. Jeffrey Hall, Brandeis University (Emeritus), and Dr. Michael Rosbash, Brandeis University.

9 Apr 2013

Study links mutated histone protein to rare brain stem cancer in children

Most cancer treatments are blunt. In an attempt to eradicate tumors, oncologists often turn to radiation or chemotherapy, which can damage healthy tissue along with the cancerous growths.

2 Apr 2013

Researchers shed new light on role of methylation in gene expression

Researchers at UC Davis and the University of British Columbia have shed new light on methylation, a critical process that helps control how genes are expressed.

28 Mar 2013

Researchers discover protein that play role in spread of pancreatic cancer

Researchers at the University of North Carolina at Chapel Hill have discovered that a protein found in the cells surrounding pancreatic cancers play a role in the spread of the disease to other parts of the body.

26 Mar 2013

Scientists to engineer anti-allergy genetically modified apples

Scientists are trying to engineer apples so that the most widely consumed fruit in Europe no longer triggers allergic reactions.

20 Mar 2013

Alnylam begins dosing in Phase I clinical trial with ALN-TTRsc for treatment of ATTR

Alnylam Pharmaceuticals a leading RNAi therapeutics company, announced today that it has initiated dosing in its Phase I clinical trial with ALN-TTRsc, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR).

19 Mar 2013

Tranylcypromine may also hold promise in treating sickle cell disease

An antidepressant drug used since the 1960s may also hold promise for treating sickle cell disease, according to a surprising new finding made in mice and human red blood cells by a team from the University of Michigan Medical School.

19 Feb 2013

FDA grants orphan drug status to Lentigen’s LG631-CD34 for treatment of glioblastoma multiforme

Lentigen Corporation, a biotechnology company specializing in the development and manufacture of lentiviral gene delivery products, announced today that that the U.S. Food and Drug Administration (FDA) has granted orphan drug status to P140K methylguanine methyltransferase (MGMT) transduced human CD34 cells for bone marrow protection in the treatment of glioblastoma multiforme.

14 Feb 2013

RNAi effective in treating cancer patients

The new study published in Cancer Discovery, the flagship journal of the American Association of Cancer Research (AACR), involving three Spanish and six American research centres, presents significant results in treating cancer patients with nanoparticles containing ribonucleic acid interference (RNAi) molecules.

13 Feb 2013

Brandeis University researchers to receive Wiley Prize in Biomedical Sciences

Deborah E. Wiley, Chair of The Wiley Foundation, John Wiley & Sons, Inc., announced today that the twelfth annual Wiley Prize in Biomedical Sciences will be awarded to Dr. Michael Young, Rockefeller University, Dr. Jeffrey Hall, Brandeis University (Emeritus), and Dr. Michael Rosbash, Brandeis University.

1 Feb 2013

DRCRF announces recipients of Nov. 2012 Damon Runyon Fellows

The Damon Runyon Cancer Research Foundation, a non-profit organization focused on supporting innovative early career researchers, named 15 new Damon Runyon Fellows at its fall Fellowship Award Committee review.

29 Jan 2013

Scientists wrestle to understand why Huntington's can produce variable symptoms

Scientists have wrestled to understand why Huntington's disease, which is caused by a single gene mutation, can produce such variable symptoms. An authoritative review by a group of leading experts summarizes the progress relating cell loss in the striatum and cerebral cortex to symptom profile in Huntington's disease, suggesting a possible direction for developing targeted therapies.

9 Jan 2013