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.

RetroNectin reagent now available for gene therapy clinical research

In an effort to aid progress in gene therapy clinical research, representatives of Clontech laboratories, Inc. and its parent company Takara Bio Inc. announce the availability of clinical grade RetroNectin reagent for direct supply to biomedical researchers.

17 Jun 2013

Effective method to restore sight to patients with blinding diseases

Researchers at the University of California, Berkeley, have developed an easier and more effective method for inserting genes into eye cells that could greatly expand gene therapy to help restore sight to patients with blinding diseases ranging from inherited defects like retinitis pigmentosa to degenerative illnesses of old age, such as macular degeneration

13 Jun 2013

Whitehead Institute researchers identify drug target for anemia

Researchers at Whitehead Institute have identified a protein that is the target of glucocorticoids, the drugs that are used to increase red blood cell production in patients with certain types of anemia, including those resulting from trauma, sepsis, malaria, kidney dialysis, and chemotherapy.

10 Jun 2013

Nation's leading experts join forces to accelerate effective treatments for brain tumors

The nation's leading brain tumor and biotech industry experts again joined forces Friday, June 7, in a bid to accelerate more effective treatments for brain tumors and promote funding for the latest emerging therapies, as the Weill Cornell Brain and Spine Center and Voices Against Brain Cancer hosted its second annual Brain Tumor Biotech Summit.

10 Jun 2013

Study sheds light on origin of wide range of brain disorders

Researchers at Weill Cornell Medical College have uncovered a mechanism that guides the exquisite wiring of neural circuits in a developing brain -- gaining unprecedented insight into the faulty circuits that may lead to brain disorders ranging from autism to mental retardation.

10 Jun 2013

Findings have implications for potential gene therapies for autism spectrum disorders

A gene linked to autism spectrum disorders that was manipulated in two lines of transgenic mice produced mature adults with irreversible deficits affecting either learning or social interaction.

6 Jun 2013

Duke researchers find novel way to repair gene responsible for Duchenne muscular dystrophy

Using a novel genetic 'editing' technique, Duke University biomedical engineers have been able to repair a defect responsible for one of the most common inherited disorders, Duchenne muscular dystrophy, in cell samples from Duchenne patients.

5 Jun 2013

BACH2 gene may play a central role in the development of allergic and autoimmune diseases

Scientists at the National Institutes of Health, and their colleagues, have discovered that a gene called BACH2 may play a central role in the development of diverse allergic and autoimmune diseases, such as multiple sclerosis, asthma, Crohn's disease, celiac disease, and type-1 diabetes.

4 Jun 2013

Phase I/II study shows safety and efficacy of VB-111 in patients with rGBM

VBL Therapeutics, a clinical-stage biotechnology company committed to the development of novel treatments for immune-inflammatory diseases and cancer, today announced clinical data demonstrating the utility of VB-111's in targeted cancer treatment.

3 Jun 2013

New nanoparticle gene delivery method to prevent stenting procedure complications

Stent angioplasty saves lives, but there often are side effects and complications related to the procedure, such as arterial restenosis and thrombosis. In the June 2013 issue of The FASEB Journal, however, scientists report that they have discovered a new nanoparticle gene delivery method that may overcome current limitations of gene therapy vectors and prevent complications associated with the stenting procedure.

1 Jun 2013

Researchers develop new gene therapy to thwart potential influenza pandemic

Researchers at the Perelman School of Medicine, University of Pennsylvania have developed a new gene therapy to thwart a potential influenza pandemic. Specifically, investigators in the Gene Therapy Program, Department of Pathology and Laboratory Medicine, directed by James M. Wilson, MD, PhD, demonstrated that a single dose of an adeno-associated virus expressing a broadly neutralizing flu antibody into the nasal passages of mice and ferrets gives them complete protection and substantial reductions in flu replication when exposed to lethal strains of H5N1 and H1N1 flu virus.

30 May 2013

Gene therapy techniques show promise in promoting growth of skin, bone and other tissues

Experimental genetic techniques may one day provide plastic and reconstructive surgeons with an invaluable tool-the ability to promote growth of the patient's own tissues for reconstructive surgery.

30 May 2013

$2 million grant to provide instrumentation, core facilities devoted to synthetic biology

The Roy J. Carver Charitable Trust has awarded a $2 million grant to the Institute for Genomic Biology at the University of Illinois at Urbana-Champaign. Under the leadership of Principal Investigator Dr. Huimin Zhao and co-Principal Investigator Dr. Christopher Rao, the grant will be disbursed over two years to provide instrumentation and core facilities for a new research theme devoted to the new scientific subdiscipline of synthetic biology.

29 May 2013

Discovery highlights new method to make HIV vaccine that targets the virus

A new discovery at Oregon Health & Science University highlights an ingenious method to ensure the body effectively reacts when infected with the highly evasive HIV virus that causes AIDS.

26 May 2013

Novel multiplex immunoassay approach to capture HIV antibodies

Detection of HIV antibodies is used to diagnose HIV infection and monitor trials of experimental HIV/AIDS vaccines. New, more sensitive detection systems being developed use microspheres to capture HIV antibodies and can measure even small amounts of multiple antibodies at one time.

24 May 2013

Common type of childhood asthma is not related to allergens and inflammation

Little is known about why asthma develops, how it constricts the airway or why response to treatments varies between patients. Now, a team of researchers at Weill Cornell Medical College, Columbia University Medical Center and SUNY Downstate Medical Center has revealed the roots of a common type of childhood asthma, showing that it is very different from other asthma cases.

24 May 2013

Researchers reveal roots of a common type of childhood asthma

24 May 2013

Dean of Weill Cornell Medical College wins AWSM Award for Excellence

Dr. Laurie H. Glimcher, the Stephen and Suzanne Weiss Dean of Weill Cornell Medical College and provost for medical affairs of Cornell University, is the winner of a prestigious award from a group of female scientists from The Feinstein Institute for Medical Research dedicated to celebrating outstanding women in science and medicine.

22 May 2013

UTMB researchers awarded grant to study gene therapy techniques to eliminate neuropathic pain

University of Texas Medical Branch at Galveston researchers have been awarded a five-year, $1.8 million grant by the National Institute of Neurological Disorders and Stroke to apply the techniques of gene therapy to the problem of neuropathic pain - that is, pain that arises from a malfunction in the nervous system.

21 May 2013

Scientists reveal inside story of European regulatory review and approval of Glybera

The scientists who led the team that developed Glybera, the first gene therapy drug approved for use in the Western world, provide a fascinating first-person account of their pioneering work in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.

16 May 2013

Gene Therapy News and Research

Further Reading

RetroNectin reagent now available for gene therapy clinical research

Effective method to restore sight to patients with blinding diseases

Whitehead Institute researchers identify drug target for anemia

Nation's leading experts join forces to accelerate effective treatments for brain tumors

Study sheds light on origin of wide range of brain disorders

Findings have implications for potential gene therapies for autism spectrum disorders

Duke researchers find novel way to repair gene responsible for Duchenne muscular dystrophy

BACH2 gene may play a central role in the development of allergic and autoimmune diseases

Phase I/II study shows safety and efficacy of VB-111 in patients with rGBM

New nanoparticle gene delivery method to prevent stenting procedure complications

Researchers develop new gene therapy to thwart potential influenza pandemic

Gene therapy techniques show promise in promoting growth of skin, bone and other tissues

$2 million grant to provide instrumentation, core facilities devoted to synthetic biology

Discovery highlights new method to make HIV vaccine that targets the virus

Novel multiplex immunoassay approach to capture HIV antibodies

Common type of childhood asthma is not related to allergens and inflammation

Researchers reveal roots of a common type of childhood asthma

Dean of Weill Cornell Medical College wins AWSM Award for Excellence

UTMB researchers awarded grant to study gene therapy techniques to eliminate neuropathic pain

Scientists reveal inside story of European regulatory review and approval of Glybera

Leveraging the power of automation to boost research

Automating research - solutions and best practices

How can microdialysis benefit drug development

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