Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

Chk1 gene alone is sufficient to kill cancer cells

Researchers at Case Western Reserve University School of Medicine have discovered a mutant form of the gene, Chk1, that when expressed in cancer cells, permanently stopped their proliferation and caused cell death without the addition of any chemotherapeutic drugs.

1 Aug 2012

Obesity also affects sexual and urinary health in men

As a man's waistline grows, so can his experience with sexual dysfunction and frequent urination, say researchers at NewYork-Presbyterian Hospital/Weill Cornell Medical Center. The study, published in the August issue of the British Journal of Urology International (BJUI), is the first to comprehensively show that obesity in men affects not just their hearts and metabolism, but also their sexual and urinary health.

31 Jul 2012

Australia's top medical researchers for 2012 announced

Finalists for one of Australia’s longest running and most prestigious awards for medical research were announced today. This year’s winner will join a list of alumni winners that includes many of the country’s most respected scientists.

31 Jul 2012

Researchers isolate elusive human gene that causes NMNAT1-related Leber congenital amaurosis

Researchers from the Massachusetts Eye and Ear Infirmary, The Children's Hospital of Philadelphia, Loyola University Chicago Health Sciences Division and their collaborators have isolated an elusive human gene that causes a common form of Leber congenital amaurosis (LCA), a relatively rare but devastating form of early-onset blindness. The new LCA gene is called NMNAT1.

30 Jul 2012

Cedars-Sinai to develop stem cell treatments for patients with ALS

Cedars-Sinai Regenerative Medicine Institute today was awarded a $17.8 million grant from the California Institute for Regenerative Medicine to develop stem cell treatments for patients with ALS, also known as Lou Gehrig's Disease, a progressive, fatal and currently incurable neurodegenerative disease.

30 Jul 2012

LPL deficiency treatment poised to be first gene therapy in Europe

For the first time, the European Medicines Agency has recommended gene therapy for approval in Europe.

26 Jul 2012

AAQ chemical temporarily restores vision in blind mice

A team of University of California, Berkeley, scientists in collaboration with researchers at the University of Munich and University of Washington in Seattle has discovered a chemical that temporarily restores some vision to blind mice, and is working on an improved compound that may someday allow people with degenerative blindness to see again.

26 Jul 2012

bluebird bio completes $60 million Series D financing

bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, today announced the successful completion of a $60 million Series D financing. In this round, new investors Deerfield Partners, RA Capital, Ramius Capital Group, and two undisclosed blue chip public investment funds joined existing investors ARCH Venture Partners, Third Rock Ventures, TVM Capital, and Forbion Capital Partners. In addition, Shire plc joined the round as a strategic investor.

25 Jul 2012

New cell regeneration method uses gene therapy to repair bone tissue

Researchers at the Royal College of Surgeons in Ireland (RCSI) have developed a new method of repairing bone using synthetic bone graft substitute material, which combined with gene therapy, can mimic real bone tissue and has potential to regenerate bone in patients who have lost large areas of bone from either disease or trauma.

25 Jul 2012

IAS releases 'road map' to find HIV cure ahead of AIDS 2012 Conference

The International AIDS Society (IAS) on Thursday in Washington, D.C., "released what they call a road map for research toward a cure for HIV -- a strategy for global teams of scientists to explore a number of intriguing leads that just might, years from now, pan out," the Associated Press reports (Neergaard, 7/20).

21 Jul 2012

uniQure receives positive recommendation from EMA CHMP for Glybera

uniQure announced today that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion that recommends marketing authorization of Glybera (alipogene tiparvovec) as a treatment for lipoprotein lipase deficiency (LPLD) under exceptional circumstances.

21 Jul 2012

Natural compound can virtually eradicate HIV in infected culture cells

A new study by scientists on the Florida campus of The Scripps Research Institute shows, in cell culture, a natural compound can virtually eliminate human immunodeficiency virus (HIV) in infected cells. The compound defines a novel class of HIV anti-viral drugs endowed with the capacity to repress viral replication in acutely and chronically infected cells.

20 Jul 2012

WCMC receives NHLBI grant to study smoking-induced COPD

Researchers at Weill Cornell Medical College were awarded a $6.5 million grant from the National Heart, Lung, and Blood Institute for a five-year investigation into metabolic changes occurring within airway epithelial cells in the lungs of chronic obstructive pulmonary disease (COPD) patients caused by cigarette smoking. In addition, researchers aim to identify which cigarette smokers are at highest risk of developing COPD as well as novel biomarkers to assist in the development of new therapeutic treatments for the disease.

17 Jul 2012

Introducing missing gene could help extend lives of patients with SMA

A team of University of Missouri researchers has found that introducing a missing gene into the central nervous system could help extend the lives of patients with Spinal Muscular Atrophy (SMA) - the leading genetic cause of infantile death in the world.

17 Jul 2012

Communication between brain and muscle cells is short-lived without LRP4 protein

Communication between the brain and muscle must be strong for us to eat, breathe or walk. Now scientists have found that a protein known to be on the surface of muscle cells must be present in both tissues to ensure the conversation is robust.

12 Jul 2012

Article addresses tremendous impact of genetic testing for Alzheimer’s

Genetic tests exist to identify risk for the rare inherited form of early-onset Alzheimer's disease (AD) and to predict susceptibility to the more common, late-onset form of AD, but do people want to know, and how do they react? The answers can be found in the article published in Genetic Testing and Molecular Biomarkers, a peer-reviewed journal from Mary Ann Liebert, Inc.

11 Jul 2012

Novel gene therapy approach may treat dysferlinopathies

The challenge of treating patients with genetic disorders in which a single mutated gene is simply too large to be replaced using traditional gene therapy techniques may soon be a thing of the past. A Nationwide Children's Hospital study describes a new gene therapy approach capable of delivering full-length versions of large genes and improving skeletal muscle function. The strategy may hold new hope for treating dysferlinopathies and other muscular dystrophies.

9 Jul 2012

Effect of spaceflight on C. elegans could help it to live longer

The effect of spaceflight on a microscopic worm - Caenorhabditis elegans (C. elegans) - could help it to live longer.

8 Jul 2012

Presence of SPDEF protein may predict prostate cancers that are unable to metastasize

Prostate cancer doesn't kill in the prostate - it's the disease's metastasis to other tissues that can be fatal. A University of Colorado Cancer Center study published this week in the Journal of Biological Chemistry shows that prostate cancer cells containing the protein SPDEF continue to grow at the same pace as their SPDEF- cousins, but that these SPDEF+ cells are unable to survive at possible sites of metastasis.

8 Jul 2012

NewYork-Presbyterian/WCMC first in N.Y. City to offer INTRABEAM radiotherapy

NewYork-Presbyterian Hospital/Weill Cornell Medical Center is the first and only hospital in the five boroughs of New York City to offer women with early stage breast cancer INTRABEAM radiotherapy, an innovative radiation treatment delivered in a single dose at the time of surgery.

6 Jul 2012