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Systemic delivery of antisense oligonucleotides neutralizes mutant RNA toxicity in mice with DM1

By targeting the specific mutation that causes the hereditary neuromuscular disease myotonic dystrophy, it is possible to neutralize the mutant RNA toxicity and minimize or even eliminate the disabling symptoms of the disease.

25 Feb 2013

Marine-based active substances show promise against multiple myeloma

There is fierce competition in the ocean habitat. In order to survive there, marine organisms defend themselves against their rivals using an entire arsenal of chemical weapons. With the help of these substances they attack hostile cells, overcome their defence system and thus are interesting active substance candidates for cancer research.

22 Feb 2013

Florida Hospital Pepin Heart Institute, USF partner for novel gene therapy trial

Cardiovascular disease specialists at Florida Hospital Pepin Heart Institute and Dr. Kiran C. Patel Research Institute affiliated with the University of South Florida announced they have enrolled their first patients into a clinical trial testing a novel gene therapy for the treatment of heart failure after ischemic injury. The therapy may promote the regeneration of heart tissue by encouraging the body to deploy more stem cells to the injury site.

22 Feb 2013

New bio-mimicking therapeutics could help treat skin wounds

In "before" and "after" photos from advertisements for wound-healing ointments, bandages and antibiotic creams, we see an injury transformed from an inflamed red gash to smooth and flawless skin. What we don't appreciate is the vital role that our own natural biomolecules play in the healing process, including their contribution to the growth of new cells and the development of new blood vessels that provide nutrients to those cells.

21 Feb 2013

Two research studies to examine factors related to early development of breast cancer

The Cancer Institute of New Jersey and its System Partner Meridian Health are working together on two research studies to examine factors related to the early development of breast cancer.

21 Feb 2013

EMA designates Osiris as Orphan Drug title holder for Prochymal

Osiris Therapeutics, Inc., the leading stem cell company developing and marketing products to treat medical conditions in inflammatory, cardiovascular, orthopedic and wound healing markets, announced today that the European Medicines Agency (EMA) has designated Osiris as the Orphan Drug title holder for Prochymal (remestemcel-L).

19 Feb 2013

Researchers discover genetic defect that can halt cell growth

A Melbourne-based research team has discovered a genetic defect that can halt cell growth and force cells into a death-evading survival state.

18 Feb 2013

Spherical nucleic acids could revolutionize biomedicine

Northwestern University's Chad A. Mirkin, a world-renowned leader in nanotechnology research and its application, has invented and developed a powerful material that could revolutionize biomedicine: spherical nucleic acids (SNAs).

16 Feb 2013

Third Rock Ventures announces formation of Jounce Therapeutics

Third Rock Ventures, LLC today announced the formation of Jounce Therapeutics, Inc. with a $47 million Series A financing of the company. Jounce is focused on the discovery and development of first-in-class cancer immunotherapies designed to harness the patient's immune system to seek out and attack cancerous cells and tumors.

15 Feb 2013

Nucleic Acid Therapeutics publishes special research section on noncoding RNAs

As scientists continue to unravel the complexity of the human genome and to uncover vital elements that play a role in both normal physiology and disease, one particular class of elements called noncoding RNAs is gaining a lot of attention. Guest Editor Tom Cech, PhD and Executive Editor Fintan Steele, PhD explore the enormous potential value of this rapidly advancing research area in their Editorial " The (Noncoding) RNA World."

15 Feb 2013

ADAMTS7 enzyme plays a crucial role in coronary heart disease

Scientists investigating how certain genes affect an individual's risk of developing coronary heart disease have identified a new therapeutic target, according to research published today in The American Journal of Human Genetics.

15 Feb 2013

Researchers ESTABLISH efficacy of tedizolid phosphate for skin infections

Results from the phase III ESTABLISH-1 study show that the new antibiotic tedizolid phosphate is noninferior to linezolid for the treatment of acute bacterial and skin structure infections.

14 Feb 2013

FDA acknowledges United Therapeutics’ oral treprostinil NDA to treat PAH

United Therapeutics Corporation announced today that the U.S. Food and Drug Administration (FDA) has acknowledged the resubmission of the new drug application (NDA) for treprostinil diolamine extended release tablets (oral treprostinil) for the treatment of pulmonary arterial hypertension.

14 Feb 2013

Researchers identify new way to influence the vital serotonin signaling system

An interdisciplinary team of researchers from the University of Texas Medical Branch at Galveston and the University of Houston has found a new way to influence the vital serotonin signaling system - possibly leading to more effective medications with fewer side effects.

14 Feb 2013

Novel approach uses adult stem cells to save sight of diabetes sufferers

Scientists at Queen's University Belfast are hoping to develop a novel approach that could save the sight of millions of diabetes sufferers using adult stem cells.

14 Feb 2013

Targacept reports net loss of $15.9 million for fourth quarter 2012

Targacept, Inc., a clinical-stage biopharmaceutical company developing novel NNR Therapeutics, today reported its financial results for the fourth quarter and year ended December 31, 2012.

14 Feb 2013

Study demonstrates how CN2097 works to restore neural functions impaired by Angelman syndrome

In a new study in mice, a scientific collaboration centered at Brown University lays out in unprecedented detail a neurological signaling breakdown in Angelman syndrome, a disorder that affects thousands of children each year, characterized by developmental delay, seizures, and other problems.

14 Feb 2013

Antibody therapeutics targeting GPCRs to be developed by alliance between MorphoSys and Heptares

MorphoSys AG (FSE: MOR; Prime Standard Segment, TecDAX) and UK-based Heptares Therapeutics, the leading GPCR drug discovery and development company, have signed an agreement to discover novel antibody therapeutics targeting G protein-coupled receptors (GPCRs), which are membrane proteins involved in a broad range of biological processes and diseases.

13 Feb 2013

New drug delivery system can reduce size of ovarian tumors by as much as 83%

By loading fragile RNA into silicon nanoparticles, researchers from The Methodist Hospital and two other institutions found a new drug delivery system can reduce the size of ovarian tumors by as much as 83 percent -- and stop tumor growth in chemotherapy-resistant ovarian cancer tissue.

13 Feb 2013

Boston College biologist receives $2.7M NIH grant to continue HIV and AIDS research

Boston College biologist Ken Williams, whose research focuses on the role of immunological cells in a range of illnesses that strike people living with HIV and AIDS, has been awarded a five-year, $2.7 million grant from the NIH's National Institute of Neurological Disorders and Stroke.

12 Feb 2013