Gene Silencing News and Research

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Alnylam achieves delivery of novel siRNAs conjugates to the central nervous system

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today that the Company has achieved delivery of novel small interfering RNA conjugates to the central nervous system and is planning to advance a pipeline of investigational RNAi therapeutics into clinical development.

8 May 2018

Sylentis presents clinical study results of new treatment for dry eye syndrome

Sylentis, Pharmaceutical Company belonging to the PharmaMar Group, has presented results from the clinical studies carried out with tivanisiran for the treatment of dry eye syndrome and that has enabled the start-up of the Phase III "Helix" clinical trial.

3 May 2018

Researchers use CRISPR/Cas9 technique to turn off gene that regulates cholesterol levels in adult mice

Biomedical engineers at Duke University have used a CRISPR/Cas9 genetic engineering technique to turn off a gene that regulates cholesterol levels in adult mice, leading to reduced blood cholesterol levels and gene repression lasting for six months after a single treatment.

27 Apr 2018

Researchers target telomerase for killing NRAS-mutant melanoma cells

Targeting telomerase was effective at killing NRAS-mutant melanoma cells, and the impact was further enhanced when the strategy was paired with an inhibitor of mitochondrial function, according to study results by The Wistar Institute published in Oncogene.

26 Apr 2018

Study reveals key function of ALS-linked protein

The protein FUS, whose mutation or disruption causes many cases of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), works as a central component of one of the most important regulatory systems in cells, according to a new study in Molecular Cell from scientists at Johns Hopkins Bloomberg School of Public Health.

8 Mar 2018

New understanding of how molecules regulate enzyme could lead to potential cancer targets

New understandings of how molecules affect the activity of an enzyme could lead to potential targets for the treatment of cancers and neurodegenerative diseases.

5 Jan 2018

Newly described super-silenced DNA reveals novel approach to reprogram cells

Newly described stretches of super-silenced DNA reveal a fresh approach to reprogram cell identity to use in regenerative medicine studies and one day in the clinic, according to a study in Molecular Cell last week by investigators from the Perelman School of Medicine at the University of Pennsylvania.

5 Jan 2018

MGH study points toward potential strategy for treating X-linked disorders

A study from a team of Massachusetts General Hospital (MGH) investigators points toward a potential strategy for treating X-linked disorders - those caused by mutations in the X chromosome - in females.

4 Jan 2018

Texas Biomedical Research Institute tests promising new treatment for HBV infection

Research at the Southwest National Primate Research Center on the campus of Texas Biomedical Research Institute helped advance a new treatment now in human trials for chronic hepatitis B virus (HBV) infection.

7 Nov 2017

UAB researchers reveal key role for RSF1 protein in silencing genes

A fertilized human egg develops into multiple tissues, organs and about 200 distinct cell types. Each cell type has the same genes, but they are expressed differently during development and in mature cells.

31 Aug 2017

Long non-coding RNAs could represent novel male infertility drug targets

Scientists have found distinctive portions of genetic material--known as lncRNAs--that help sperm develop. Male mice lacking a particular lncRNA have low sperm count, suggesting lncRNAs could represent novel infertility drug targets.

24 Aug 2017

New imaging approach reveals dynamic and structural interactions of DNA in cell's nucleus

Sixty-four years ago, James Watson and Francis Crick described the now-iconic double helix structure of DNA.

29 Jul 2017

Study uncovers potential new therapeutic targets for HCV infections

An international study led by researchers at Monash University' Biomedicine Discovery Institute (BDI) has shone light on the way the Hepatitis C Virus (HCV) hijacks the communication systems in the host cells it infects, uncovering potential new therapeutic targets for the disease.

8 May 2017

New gene-silencing drugs cut cholesterol levels by half in early research

The first in a new class of gene-silencing drugs, known as inclisiran, has been shown to halve cholesterol levels in patients at risk of cardiovascular disease.

21 Mar 2017

KAUST researchers develop biocompatible nanostructures for use in gene delivery

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.

24 Sep 2016

Researchers find switch that helps restore damaged axons

Researchers at the University of Wisconsin-Madison have found a switch that redirects helper cells in the peripheral nervous system into "repair" mode, a form that restores damaged axons.

16 Sep 2016

Novel dual molecular tuner offers easy way to perform in-depth analyses in mammalian cells

A research team headed by investigators at the Icahn School of Medicine at Mount Sinai has developed a tool that allows scientists to quickly manipulate levels of two proteins in the same cell.

5 Aug 2016

Overcoming gene silencing barriers to target neurological conditions: an interview with Dr Errol de Souza

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.

1 Aug 2016

Researchers study how skin stem cells respond to forces

All cells share the same genetic code, no matter if they are skin or brain cells. However, these cells are exposed to very different types of mechanical environments and mechanical stresses.

13 Jul 2016

Researchers identify PKD1 protein as potential cause for impaired insulin signaling in diabetics

Medication can help trigger the enzyme that kick starts insulin production in the body, but the drugs don't always work for those who are obese or diabetic, and most need to regulate their glucose and insulin levels. That's why a recent discovery made by Rudy Valentine and a team of researchers holds so much promise.