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.

Important clue to understanding the origins of the AIDS epidemic

Medical Research Council (MRC) scientists have uncovered an important clue to understanding the origins of the AIDS epidemic. The work suggests that harnessing natural mechanisms of resistance to HIV infection might provide new methods for combating AIDS.

10 Jan 2005

£2.5 million to support further research into gene therapy for cystic fibrosis

The funding commitment was made in the Genetics White Paper, 'Our Inheritance, Our Future: Realising the potential of genetics in the NHS' and will help to ensure that those with cystic fibrosis benefit as soon as possible from the latest advances in genetic knowledge and technologies.

4 Jan 2005

Researchers can now inexpensively program the chemical synthesis of entire genes on a single microchip

Devices the size of a pager now have greater capabilities than computers that once occupied an entire room. Similar advances are being made in the emerging field of synthetic biology at the University of Houston, now allowing researchers to inexpensively program the chemical synthesis of entire genes on a single microchip.

23 Dec 2004

Gene therapy effective in helping children with severe combined immunodeficiency (SCID)

A UK study in this week’s issue of THE LANCET provides further evidence that gene therapy can be effective in creating a functional immune system for infants with severe combined immunodeficiency (SCID).

20 Dec 2004

First gene therapy for skin cancer

Researchers at UT Southwestern Medical Center at Dallas have successfully tested the first gene therapy for skin cancer, using a mouse model for the disease xeroderma pigmentosum, or XP.

13 Dec 2004

New advances in sickle cell disease

Excruciating pain episodes, recurrent pneumonias, strokes, severe infections, chronic hemolytic anemia, and secondary pulmonary hypertension are common complications of sickle cell disease, a life-threatening inherited defect in blood that causes normally disc-shaped red blood cells to take on a sickle shape.

6 Dec 2004

Episome vectors offer fresh angle to gene therapy

A European research team is seeking to overcome the early setbacks suffered by the promising new field of ‘gene therapy’ by developing a revolutionary delivery technique, known as ‘episome vectors’. This breakthrough could pave the way to treating hereditary diseases by injecting the body with healthy versions of damaged genes.

6 Dec 2004

Herpes virus offers new hope in search for cure for cancer

In laboratory studies at Cincinnati Children's Hospital Medical Center, researchers have successfully treated the most common malignant abdominal tumor of childhood: neuroblastoma tumors.

4 Dec 2004

Heart researchers have used gene therapy to bring failing rat hearts back to normal

Scientists led by Walter Koch, Ph.D., director of the Center for Translational Medicine in the Department of Medicine in Jefferson Medical College of Thomas Jefferson University in Philadelphia, used a virus to insert the gene for a protein called S100A1 into failing rat hearts.

4 Dec 2004

Why our immune system worsens with age

Scientists at the Vaccine and Gene Therapy Institute at Oregon Health & Science University have made a discovery that helps explain why our immune system worsens with age. The work was led by Janko Nikolich-Zugich, M.D., Ph.D., a senior scientist at the VGTI.

2 Dec 2004

New possibilities for the use of VEGF in the treatment of Amyotrophic Lateral Sclerosis

ALS is an incurable, paralyzing neurodegenerative disorder that strikes 5 persons in every 100,000. The disease commonly affects healthy people in the most active period of their lives − without warning or previous family history.

29 Nov 2004

Scientists have given blind nerve cells the ability to detect light

Scientists at the University of California, Berkeley, have given "blind" nerve cells the ability to detect light, paving the way for an innovative therapy that could restore sight to those who have lost it through disease.

24 Nov 2004

National Cancer Institute symposium exploring the application of nanotechnology in the diagnosis, treatment and prevention of cancer

The Case Comprehensive Cancer Center, which includes The Cleveland Clinic Taussig Cancer Center and the Ireland Cancer Center at University Hospitals of Cleveland, will sponsor a National Cancer Institute symposium exploring the application of nanotechnology in the diagnosis, treatment and prevention of cancer.

25 Oct 2004

A novel gene therapy strategy may help reduce seizures in adults suffering from epilepsy

Deborah Lin, from the Faculty of Medical and Health Sciences, is investigating the effects of a therapeutic gene transfer in a particular form of epilepsy which affects the brain’s temporal lobe region.

12 Oct 2004

Carbon nanotubes offer a new approach to gene therapy

Many genes that are related to particular diseases have now been identified. Scientists are trying to use this knowledge to treat diseases.

11 Oct 2004

Researchers grow mouth lining into pieces of bio-bandage

University of Michigan researchers are testing a new procedure in which they can take a tiny piece of a person's mouth lining, grow it into a dollar-bill sized piece of tissue and graft that expanded piece into the donor's mouth to heal a wound.

5 Oct 2004

Scientists discover a record number of proteins for the highly infectious and stealthy human cytomegalovirus

Scientists have discovered a record number of proteins for one of the largest and most complex viruses, the highly infectious and stealthy human cytomegalovirus, a team from Pacific Northwest National Laboratory and Oregon Health & Science University reported today in the October Journal of Virology.

29 Sep 2004

NIH opens new hospital totally dedicated to clinical research

This new hospital totally dedicated to clinical research — research involving patients — is the most significant addition to the NIH campus in more than 50 years and provides a unique opportunity for scientists, clinicians, and patients to study and conquer both chronic and acute disease in the 21st century.

22 Sep 2004

New gene therapy technique may help treat Alzheimer's disease

Investigators at Rush University Medical Center have successfully initiated a new technique that uses gene therapy to deliver nerve growth factor into regions of the brain where neurons are degenerating, in order to prevent cell death and reverse cell atrophy, two hallmarks of Alzheimer's disease.

21 Sep 2004

Novel strategy for stimulating the production of utrophin

Researchers at the University of Pennsylvania School of Medicine report a novel strategy for stimulating the production of utrophin – an important muscle protein in young mice – for muscular dystrophy therapy.

21 Sep 2004