Gene Silencing News and Research

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New topical film patch can effectively close dermal wounds, reduce scarring

Currently there is no product available based on polymer film containing active molecules to accelerate dermal wound healing that also reduces the scar formation.

8 Jul 2016

Researchers discover mechanism of gene silencing in hormone-dependent breast cancer cells

Sometimes, the silencing of a gene is as important as its activation. Nonetheless, up to now, most studies on hormone-mediated gene regulation have focused on researching the factors that influence the activation of certain genes. Little attention has been paid to gene silencing.

8 Jul 2016

Alnylam reports new results from investigational RNAi therapeutic programs

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today preliminary results from its ongoing Phase 2 open-label extension (OLE) studies with patisiran and revusiran, both investigational RNAi therapeutics targeting transthyretin (TTR) for the treatment of hereditary TTR-mediated amyloidosis (hATTR amyloidosis).

6 Jul 2016

New technology could help deliver treatments for brain injuries

A new study led by scientists at the Sanford Burnham Prebys Medical Discovery Institute describes a technology that could lead to new therapeutics for traumatic brain injuries. The discovery, published today in Nature Communications, provides a means of homing drugs or nanoparticles to injured areas of the brain.

29 Jun 2016

PharmaMar announces initiation of pivotal plitidepsin clinical trial in patients with T-cell lymphomas

PharmaMar today announced the start of a multicenter, prospective, pivotal study to analyze the efficacy of the antitumoral compound of marine origin, plitidepsin in patients with relapsed and refractory angioimmunoblastic T-cell lymphoma.

15 Jun 2016

Phase I study of triple drug combination shows promise in multiple myeloma patients

PharmaMar announces the positive results from a Phase I study of plitidepsin in combination with bortezomib and dexamethasone in patients with relapsed and/or refractory multiple myeloma.

21 May 2016

Researchers develop novel ss-siRNAs to turn off disease-related genes

Combining the therapeutic potential and advantages of existing oligonucleotide-based approaches to turn off disease-related genes, a type of single-stranded silencing RNAs (ss-siRNAs) has shown significantly improved potency and activity.

13 May 2016

Histone mutation linked to fatal pediatric brain tumor

A single defect in a gene that codes for a histone — a "spool" that wraps idle DNA — is linked to pediatric cancers in a study published today in the journal Science.

13 May 2016

Plitidepsin in combination with dexamethasone shows top-line results in Phase III multiple myeloma trial

PharmaMar today announced positive top-line results of its Phase III clinical trial -ADMYRE- with Aplidin (plitidepsin) in combination with dexamethasone versus dexamethasone alone in patients with relapsed/refractory multiple myeloma (MM).

2 Apr 2016

Altered metabolism of two essential amino acids helps drive development of glioblastoma

The altered metabolism of two essential amino acids helps drive the development of the most common and lethal form of brain cancer, according to a new study led by researchers at The Ohio State University Comprehensive Cancer Center - Arthur G. James Cancer Hospital and Richard J. Solove Research Institute.

2 Apr 2016

Mtss1 'tumour suppressor' for CML

The metastasis suppressor 1 protein acts a tumour suppressor in chronic myeloid leukaemia stem cells, reveals research published in Leukemia.

17 Feb 2016

ST Asia signs licensing deal with PharmaMar to distribute new multiple myeloma drug in South East Asia

International biopharmaceutical company Specialised Therapeutics Asia will supply and distribute a novel oncology drug candidate throughout South East Asia, following an exclusive licensing deal with European pharmaceutical company PharmaMar.

3 Feb 2016

Research sheds light on mechanism by which hemangioblasts become blood cells

A research group led by Lecturer Makoto Kobayashi of the University of Tsukuba Faculty of Medicine, has managed to shed light on the puzzling mechanism by which hemangioblasts become blood cells. Although hemangioblasts are the common precursor cells for blood cells and vascular endothelia that are present during the fetal period, the discovery of hemangioblasts in adult organisms has begun to garner attention from the medical world.

21 Jan 2016

New TAU study offers tangible hope of curing Mantle Cell Lymphoma

With a median survival rate of just five to seven years, Mantle Cell Lymphoma (MCL) is considered the most aggressive known blood cancer -- and available therapies are scarce. Three thousand Americans are diagnosed with MCL every year, and despite progress in personalized therapies to treat metastases elsewhere in the body, systemic therapeutic drug delivery to cancerous blood cells continues to challenge the world of cancer research.

6 Jan 2016

Mutant protein responsible for cystic fibrosis talks to wrong cellular neighbors

Scientists at The Scripps Research Institute have found evidence that a mutant protein responsible for most cases of cystic fibrosis is so busy "talking" to the wrong cellular neighbors that it cannot function normally and is prematurely degraded.

1 Dec 2015

Exploring how absence of ASS1 protein affects sick children may provide a 'lens' on cancer

What can a rare genetic childhood disease teach us about cancer? Dr. Ayelet Erez of the Weizmann Institute of Science's Department of Biological Regulation says: "A single-mutation disease can act as a 'lens.' If we find exactly what malfunctions in the sick child, we can zoom in and understand the role of the same gene among the many genetic changes that accompany cancer."

26 Nov 2015

Research reveals MECP2 Duplication Syndrome can be reversed

The methyl CpG binding protein 2 gene (MECP2) produces a protein of the same name, the level of which is critical for normal brain function. Mutations leading to protein under-expression cause Rett Syndrome while gene duplication causing over-expression lead to MECP2 Duplication Syndrome. Both disorders are severely debilitating childhood neurological diseases.

26 Nov 2015

Alnylam presents positive ongoing phase 2 open-label extension data for Patisiran and Revusiran

Alnylam Pharmaceuticals, Inc. has announced new results from its ongoing Phase 2 open-label extension (OLE) studies with patisiran and revusiran, investigational RNAi therapeutics targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR amyloidosis).

3 Nov 2015

First patients receive doses of antisense drug for Huntington's disease

The first few patients have received doses of an experimental RNA-targeting drug for Huntington's disease, it was announced today.

20 Oct 2015

Set of genes identified in human genome essential for survival, proliferation of human cell lines

Using two complementary analytical approaches, scientists at Whitehead Institute and Broad Institute of MIT and Harvard have for the first time identified the universe of genes in the human genome essential for the survival and proliferation of human cell lines or cultured human cells.

16 Oct 2015