Gene Silencing News and Research

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Study describes vulnerability shared by large subset of cancer cell lines

Cancer cells adapt to potentially fatal mutations and other molecular malfunctions by adjusting one or more other genes' activity, in the process becoming dependent on those genes for their survival and growth.

12 Apr 2019

Using gene therapy strategies to rejuvenate aging cells and treat osteoarthritis

Aging is a risk factor for the development of osteoarthritis (OA), a chronic disease characterized by degeneration of articular cartilage leading to pain and physical disability.

28 Mar 2019

Novel DNA nanoplatform delivers anticancer agents to multidrug-resistant tumors

One of the most successful techniques to combat multidrug resistance in cancer cells is the downregulation of those genes responsible for drug resistance.

22 Nov 2018

Shortage of specific gene 'silencing' molecules linked with pediatric low-grade gliomas

Experimenting with lab-grown brain cancer cells, Johns Hopkins Medicine researchers have added to evidence that a shortage of specific tiny molecules that silence certain genes is linked to the development and growth of pediatric brain tumors known as low-grade gliomas.

12 Nov 2018

Alnylam announces positive results from Phase 1/2 study of lumasiran for treating primary hyperoxaluria-1

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today updated positive results from its Phase 1/2 clinical study of lumasiran, an investigational, subcutaneously administered RNAi therapeutic targeting glycolate oxidase for the treatment of primary hyperoxaluria type 1.

4 Oct 2018

Alnylam submits CTA application to MHRA to initiate Phase 1/2 study of ALN-AAT02

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today that it has submitted a Clinical Trial Authorization application to the Medicines and Healthcare products Regulatory Agency to initiate a Phase 1/2 study of ALN-AAT02, an investigational RNAi therapeutic targeting alpha-1 antitrypsin for the treatment of AAT deficiency-associated liver disease.

3 Oct 2018

Alnylam reports further progress on broadening the therapeutic potential of RNAi

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today further progress on the Company's platform efforts in extrahepatic delivery of novel siRNA conjugates, including central nervous system (CNS) and ocular delivery in rat and non-human primates (NHPs).

3 Oct 2018

Alnylam announces positive results from Phase 3 Study of givosiran for treating acute hepatic porphyria

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today positive topline results from the interim analysis of the ENVISION Phase 3 Study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 for the treatment of acute hepatic porphyria.

27 Sep 2018

New study focuses on two-way gene switches controlling gene activity

Led by ICREA research professors at the Centre for Genomic Regulation, Luciano Di Croce and Marc A. Marti-Renom, the study focused on a set of genes with what's known as bivalent promoters - two-way genetic 'control switches' that are poised either to turn on in early development and rapidly drive high levels of gene activity, or to switch off and shut down the gene completely.

20 Sep 2018

Antioxidant in green tea helps sneak therapeutic RNAs into cells

Drinking green tea has been linked to health benefits ranging from cardiovascular disease prevention to weight loss.

19 Sep 2018

Penn researchers find common thread linking almost all TNR expansion diseases

In a class of roughly 30 neurological disorders that includes ALS, Huntington's Disease and Fragile X Syndrome, the relevant mutant gene features sections of repeating base pair sequences known as short tandem repeats, or STRs. Healthy people have STRs of normal lengths distributed across their DNA.

19 Sep 2018

Arrowhead completes dosing of ARO-AAT Phase 1 study to treat rare genetic liver disease

Arrowhead Pharmaceuticals Inc. today announced that it has completed dosing of a Phase 1 clinical study of ARO-AAT, the company's second generation subcutaneously administered RNA interference therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency.

31 Aug 2018

EC grants marketing authorization for ONPATTRO (patisiran) in Europe

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.

30 Aug 2018

MDA announces 34 new grants totaling $9.9 million to accelerate neuromuscular research

The Muscular Dystrophy Association today announced the award of 34 new grants totaling more than $9.9 million for its summer round of funding. These new grants represent a continued commitment by MDA to fund groundbreaking research that will accelerate treatments and cures for the more than 40 diseases in its program.

24 Aug 2018

Researchers use DNA editing tool to reveal mechanisms behind autoimmune diseases

A group of researchers at the University of São Paulo in Brazil used a DNA editing tool called CRISPR/Cas9 to manipulate on of the genes associated to the autoaggressive T lymphocytes responsible for inducing autoimmune diseases such as autoimmune polyglandular syndrome type 1 and type 1 diabetes.

2 Aug 2018

Alnylam’s patisiran receives positive opinion from CHMP for treating hATTR amyloidosis

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.

27 Jul 2018

Newer version of ALS drug may effectively treat inherited form of the disease

NIH-funded researchers delayed signs of amyotrophic lateral sclerosis in rodents by injecting them with a second-generation drug designed to silence the gene, superoxide dismutase 1.

27 Jul 2018

Researchers employ nucleotide-based gene silencing to mitigate common ataxia

In what researchers are calling a game changer for future ataxia treatments, a new study showed the ability to turn down the disease progression of the most common dominantly inherited ataxia.

20 Jun 2018

Alnylam announces new positive results from Phase 1/2 study of lumasiran in patients with PH1

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.

8 Jun 2018

Phase 1/2 study evaluates safety, tolerability of multiple-ascending doses of ARO-HBV in hepatitis B patients

Arrowhead Pharmaceuticals Inc. today announced that it has dosed the first 3 hepatitis B virus patients in the multiple-ascending dose portion of its ongoing Phase 1/2 study of ARO-HBV, the company's third generation subcutaneously administered RNA interference therapeutic being developed as a potentially curative therapy for patients with chronic HBV infection.

14 May 2018