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.

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

Single dose breast cancer treatment helps forego 6-7 week radiation regimen

5 Jul 2012

Nicotine addiction: An interview with Dr. Crystal

Cigarette smoking and nicotine addiction are widespread problems throughout the world. Many people continue to smoke despite high taxes and the available public information on the dangers of smoking.

3 Jul 2012

Treating Sickle Cell Disease with L-Glutamine

By focusing on how best to help patients, researchers can sometimes find solutions that are much simpler than anticipated. That has been the guiding approach to our efforts to develop a treatment for sickle cell disease.

3 Jul 2012

Safer alternative to experimental gene therapy against HIV infection

Scientists at The Scripps Research Institute have discovered a surprisingly simple and safe method to disrupt specific genes within cells. The scientists highlighted the medical potential of the new technique by demonstrating its use as a safer alternative to an experimental gene therapy against HIV infection.

2 Jul 2012

Vaccine for nicotine addiction developed

Researchers at Weill Cornell Medical College have developed and successfully tested in mice an innovative vaccine to treat nicotine addiction.

28 Jun 2012

Gene transfer technique eliminates specific neural circuit linked to Parkinson's disease

In the brains of humans and non-human primates, over 100 billion nerve cells build up complicated neural circuits and produce higher brain functions. When an attempt is made to perform gene therapy for neurological diseases like Parkinson's disease, it is necessary to specify a responsible neural circuit out of many complicated circuits.

27 Jun 2012