Gene Silencing News and Research

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Research into “insulating” brain cells could help to cure neurodegenerative diseases such as ALS

Johns Hopkins researchers say they have discovered that the central nervous system's oligodendroglia cells, long believed to simply insulate nerves as they "fire" signals, are unexpectedly also vital to the survival of neurons. Damage to these insulators appears to contribute to brain injury in neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease for the Yankee baseball great who died from the disease.

11 Jul 2012

Arrowhead initiates Adipotide Phase 1 trial in obesity

Arrowhead Research Corporation, a targeted therapeutics company, today announced that the first patient has been dosed in a Phase 1 clinical trial with Adipotide, a new class of treatment for obesity.

11 Jul 2012

DRCRF names 21 new Damon Runyon Fellows

The Damon Runyon Cancer Research Foundation, a non-profit organization focused on supporting innovative early career researchers, named 21 new Damon Runyon Fellows at its spring Fellowship Award Committee review.

11 Jul 2012

FDA provides Orphan Drug Designation to Alnylam’s ALN-TTR02

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the U.S. Food & Drug Administration (FDA) has provided Orphan Drug Designation to ALN-TTR02 as a therapeutic for the treatment of familial amyloidotic polyneuropathy (FAP), one of the predominant clinical manifestations of transthyretin (TTR)-mediated amyloidosis (ATTR).

20 Jun 2012

Arrowhead initiates final IND-enabling steps for ARC-520 to treat HBV

Arrowhead Research Corporation, a targeted therapeutics company, today announced that its hepatitis B virus (HBV) program has completed all internal preclinical requirements and has named a clinical candidate.

7 Jun 2012

Alnylam announces results from ALN-VSP Phase I study on liver cancers

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced results today from its Phase I extension study with ALN-VSP, a systemically delivered RNAi therapeutic for the treatment of advanced solid tumors with liver involvement.

5 Jun 2012

Top-line results from Alnylam’s ALN-RSV01 Phase IIb trial on RSV infection

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today top-line results from a Phase IIb trial with ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection in lung transplant patients.

31 May 2012

Neural protein plays two opposing roles

A protein produced by the central nervous system’s support cells seems to play two opposing roles in protecting nerve cells from damage, an animal study by Johns Hopkins researchers suggests: Decreasing its activity seems to trigger support cells to gear up their protective powers, but increasing its activity appears to be key to actually use those powers to defend cells from harm.

29 May 2012

Short hairpin RNAi gene silencing technology receives CSIRO's foundational patents

Australia's leading science agency, the Commonwealth Scientific and Industrial Research Organisation (CSIRO), has been granted foundational patents in the US and Europe for short hairpin RNAi (shRNA) gene silencing technology.

29 May 2012

Researchers generate neuronal cells from patients with Fragile X syndrome

Researchers at the Hebrew University of Jerusalem have achieved, for the first time, the generation of neuronal cells from stem cells of Fragile X patients. The discovery paves the way for research that will examine restoration of normal gene expression in Fragile X patients.

23 May 2012

Inexpensive sleep disorder drug appears to be potent inhibitor of cancer cells

An inexpensive "orphan drug" used to treat sleep disorders appears to be a potent inhibitor of cancer cells, according to a new study led by scientists at Fred Hutchinson Cancer Research Center.

22 May 2012

Final results from Alnylam’s ALN-TTR01 Phase I trial on TTR-mediated amyloidosis

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today final results from its completed Phase I clinical trial with ALN-TTR01, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis.

11 May 2012

Molecule that starts DNA repairs also halts them

Repairing DNA breaks can save a cell's life - but shutting off the repair machinery can be just as critical. How cells accomplish this feat was unknown. However, new research by Johns Hopkins scientists, published in the Feb. 22 issue of Nature, suggests that shutting down the repair machinery relies heavily on the same molecule used to start repair in the first place.

8 May 2012

Alnylam first quarter revenues decrease to $20.6 million

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, today reported its consolidated financial results for the first quarter 2012, and company highlights.

4 May 2012

Scientists discover new type of dynamic change in human stem cells

A team led by scientists at The Scripps Research Institute and the University of California (UC) San Diego has discovered a new type of dynamic change in human stem cells.

4 May 2012

Positive results from Alnylam’s ALN-PCS Phase I trial on severe hypercholesterolemia

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today positive results from its Phase I clinical trial of ALN-PCS, an RNAi therapeutic targeting PCSK9 for the treatment of severe hypercholesterolemia.

21 Apr 2012

Researchers discover intra-cellular function of LOXL2 protein

Researchers at the Hospital de Mar Research Institute (IMIM) have discovered that the protein LOXL2 has a function within the cell nucleus thus far unknown. They have also described a new chemical reaction of this protein on histone H3 that would be involved in gene silencing, one of which would be involved in the progression of breast, larynx, lung and skin tumours.

16 Apr 2012

PFKFB4 gene leads to excessive growth of prostate cancer cells

Cancer cell metabolism may present a new target for therapy as scientists have uncovered a possible gene that leads to greater growth of prostate cancer cells.

23 Mar 2012

Alnylam commences dosing in ALN-TTR02 Phase I clinical trial for TTR-mediated amyloidosis

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has initiated dosing in its Phase I clinical trial with ALN-TTR02, an RNAi therapeutic targeting the transthyretin (TTR) gene for the treatment of TTR-mediated amyloidosis (ATTR). The study is aimed at evaluating the safety and tolerability of ALN-TTR02 in healthy volunteers.

19 Mar 2012

PCP signaling restricts neural growth during regeneration, homeostasis and development

The genetic pathway that regulates the way cells align themselves relative to each other has been found to act as a "stop sign" that signals organisms when to halt cell growth, according to new research published by biologists at the Center for Regenerative and Developmental Biology in Tufts University's School of Arts and Sciences.

16 Mar 2012