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Antisense is the non-coding strand in double-stranded DNA. The antisense strand serves as the template for mRNA synthesis.
Novel drug shows promise against metastatic breast cancer in mouse models

Novel drug shows promise against metastatic breast cancer in mouse models

A doctor treating a patient with a potentially fatal metastatic breast tumor would be very pleased to find, after administering a round of treatment, that the primary tumor had undergone a change in character - from aggressive to static, and no longer shedding cells that can colonize distant organs of the body. [More]
U-M research could lead to new ways of fighting X-linked diseases in girls and women

U-M research could lead to new ways of fighting X-linked diseases in girls and women

Nearly every girl and woman on Earth carries two X chromosomes in nearly every one of her cells -- but one of them does (mostly) nothing. That's because it's been silenced, keeping most of its DNA locked up and unread like a book in a cage. [More]
FDA grants Orphan Drug Designation to Ionis Pharmaceuticals' IONIS-HTT Rx for treatment of HD patients

FDA grants Orphan Drug Designation to Ionis Pharmaceuticals' IONIS-HTT Rx for treatment of HD patients

Ionis Pharmaceuticals, Inc. today announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation to IONIS-HTTRx for the treatment of patients with Huntington's disease (HD). [More]
Novel drug shows promise in mouse models of human metastatic breast cancer

Novel drug shows promise in mouse models of human metastatic breast cancer

A doctor treating a patient with a potentially fatal metastatic breast tumor would be very pleased to find, after administering a round of treatment, that the primary tumor had undergone a change in character - from aggressive to static, and no longer shedding cells that can colonize distant organs of the body. Indeed, most patients with breast and other forms of cancer who succumb to the illness do so because of the cancer's unstoppable spread. [More]
Researchers find new way to prevent or reduce pathologies caused by malformed proteins

Researchers find new way to prevent or reduce pathologies caused by malformed proteins

Improperly formed proteins can cause a host of serious illnesses, from muscular dystrophy to cystic fibrosis. A question of enormous import in research, beyond the challenge of determining how malformed proteins contribute to specific disease processes, is figuring out ways to prevent or reduce the pathologies they cause. [More]
Study paves way for development of novel cancer therapeutics

Study paves way for development of novel cancer therapeutics

A team of scientists, comprising researchers from the Institute of Molecular and Cell Biology, a research institute under the Agency for Science, Technology and Research, Singapore, and the VIB Laboratory of Molecular Cancer Biology, has revealed the mechanism by which tumor cells elevate levels of MDM4, a protein that is highly expressed in cancer cells but not in normal adult tissues. [More]
Biogen begins ISIS-SOD1 Rx Phase 1/2 clinical study in patients with amyotrophic lateral sclerosis

Biogen begins ISIS-SOD1 Rx Phase 1/2 clinical study in patients with amyotrophic lateral sclerosis

Isis Pharmaceuticals, Inc. today announced that its partner, Biogen, has initiated a Phase 1/2 clinical study of ISIS-SOD1 Rx (BIIB067) in patients with amyotrophic lateral sclerosis (ALS). [More]
Research reveals MECP2 Duplication Syndrome can be reversed

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. [More]
Idera begins enrollment in IMO-8400 Phase 2 clinical trial for treatment of dermatomyositis

Idera begins enrollment in IMO-8400 Phase 2 clinical trial for treatment of dermatomyositis

Idera Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing Toll-like receptor (TLR) and RNA therapeutics for patients with cancer and rare diseases, today announced that the company has commenced enrollment in a Phase 2 clinical trial of IMO-8400, an investigational TLR 7, 8 and 9 antagonist, in patients with dermatomyositis. Dermatomyositis is a rare and debilitating inflammatory muscle and skin disease associated with significant morbidity, decreased quality of life and an increased risk of premature death. [More]
Isis Pharmaceuticals begins ISIS-FXI Rx Phase 2 study in patients with ESRD on hemodialysis

Isis Pharmaceuticals begins ISIS-FXI Rx Phase 2 study in patients with ESRD on hemodialysis

Isis Pharmaceuticals, Inc. announced the initiation of a Phase 2 study evaluating ISIS-FXIRx in patients with end-stage renal disease (ESRD) on hemodialysis. In May 2015, Isis entered into an exclusive license agreement with Bayer HealthCare (Bayer) to develop and commercialize ISIS-FXIRx for the prevention of clotting disorders. [More]
Researchers identify molecular switch that can convert unhealthy white fat into energy-burning brown fat

Researchers identify molecular switch that can convert unhealthy white fat into energy-burning brown fat

A research team led by UC San Francisco scientists has identified a molecular switch capable of converting unhealthy white fat into healthy, energy-burning brown fat in mice. Drugs that flip this switch rapidly reduced obesity and diabetes risk factors in mice fed a high fat diet. [More]
Ultragenyx, Arcturus sign agreement to develop mRNA therapeutics to certain rare diseases

Ultragenyx, Arcturus sign agreement to develop mRNA therapeutics to certain rare diseases

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Arcturus Therapeutics, Inc., a leading RNA medicines company, today announced that they have entered into a research collaboration and license agreement to discover and develop messenger RNA (mRNA) therapeutics to certain rare disease targets using Arcturus' UNA Oligomer chemistry and LUNAR nanoparticle delivery platform. [More]
Four microRNAs appear to play critical roles in controlling cholesterol, triglyceride metabolism

Four microRNAs appear to play critical roles in controlling cholesterol, triglyceride metabolism

Four tiny segments of RNA appear to play critical roles in controlling cholesterol and triglyceride metabolism. In their report receiving advance online publication in Nature Medicine, a Massachusetts General Hospital-based research team describes finding how these microRNAs could reduce the expression of proteins playing key roles in the generation of beneficial HDL cholesterol, the disposal of artery-clogging LDL cholesterol, control of triglyceride levels and other risk factors of cardiovascular disease. [More]
First patients receive doses of antisense drug for Huntington's disease

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. [More]
New genetic discovery could lead to better treatment for X-linked diseases

New genetic discovery could lead to better treatment for X-linked diseases

Think back to middle school biology class, when you learned that boys have an X and a Y chromosome inside each cell, and girls have two X's. [More]
Sense oligonucleotide antidote reverses actions of antisense antithrombotic drug, prevents bleeding

Sense oligonucleotide antidote reverses actions of antisense antithrombotic drug, prevents bleeding

Researchers from Isis Pharmaceuticals (Carlsbad, CA) and Prysis Biotechnologies (Pudong, Shanghai, China) have demonstrated proof-of-concept for using a sense oligonucleotide to undo the effects of an antisense drug, an antithrombotic agent in this novel study. [More]
Study: RNA editing technique shows promise in treating rare, severe form of muscular dystrophy

Study: RNA editing technique shows promise in treating rare, severe form of muscular dystrophy

An RNA editing technique called "exon skipping" has shown preliminary success in treating a rare and severe form of muscular dystrophy that currently has no treatment, based on a new study from Northwestern Medicine and the University of Chicago. Children with the disease lose significant muscle strength early in life. [More]
Scientists elucidate genetic interaction that may prove key to development, progression of glaucoma

Scientists elucidate genetic interaction that may prove key to development, progression of glaucoma

Scientists at the University of California, San Diego School of Medicine have elucidated a genetic interaction that may prove key to the development and progression of glaucoma, a blinding neurodegenerative disease that affects tens of millions of people worldwide and is a leading cause of irreversible blindness. [More]
Common gene mutation may cause brain damage associated with ALS and FTD

Common gene mutation may cause brain damage associated with ALS and FTD

Johns Hopkins researchers say they have discovered some of the first steps in how a very common gene mutation causes the brain damage associated with both amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). [More]
iCo Therapeutics incurs total comprehensive loss of $738,607 for second quarter 2015

iCo Therapeutics incurs total comprehensive loss of $738,607 for second quarter 2015

iCo Therapeutics, today reported financial results for the quarter ended June 30, 2015. Amounts, unless specified otherwise, are expressed in Canadian dollars and presented under International Financial Reporting Standards ("IFRS"). [More]
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