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.

First steps taken to treat human hereditary blindness

A team led by Krzysztof Palczewski, Ph.D., chair of pharmacology at the Case Western Reserve University School of Medicine, has taken the first steps in treating an eye disease causing irreversible congenital blindness in millions of people worldwide by successfully testing two new treatments in mice.

3 Nov 2005

Treatment for Leber Congenital Amaurosis

U.S. researchers have taken the first steps in treating an eye disease that causes irreversible blindness in humans by successfully testing two novel treatments in mice.

1 Nov 2005

New understanding of how neurogenesis occurs

Scientists at the Salk Institute for Biological Studies have identified a crucial cellular signal that controls the fate of stem cells in the brains of adult mice.

27 Oct 2005

Gene therapy used to successfully treat heart failure

University of Pittsburgh investigators have for the first time used gene therapy to successfully treat heart failure and other degenerative muscle problems in an animal model that is genetically susceptible to a human muscular dystrophy.

25 Oct 2005

St. John's wort plant has the capacity to inhibit expression of the HIV-1 gene

A novel protein, p27SJ, extracted from a callus culture of the St. John's wort plant (Hypericum perforatum) suppresses HIV-1 expression and inhibits its replication, according to researchers at Temple University School of Medicine's department of neuroscience and Center for Neurovirology (CNV).

24 Oct 2005

WWOX gene finding could lead to a new strategy for treating lung cancer

The research focused on a gene known as WWOX, which is lost or silenced in a large majority of lung cancers, and in cancers of the breast, ovary, prostate, bladder, esophagus and pancreas. The work was led by scientists at The Ohio State University Comprehensive Cancer Center - Arthur G. James Cancer Hospital and Richard J. Solove Research Institute.

17 Oct 2005

Gene therapy may prevent the damaging effects of radiation

Gene therapy could be used as an agent to protect normal tissues, including the esophagus and lung, from damage during a second administration of radiation therapy for non-small cell lung cancer, according to an animal study presented by University of Pittsburgh researchers at the 47th Annual Meeting of the American Society for Therapeutic Radiology and Oncology (ASTRO) in Denver.

16 Oct 2005

Discovery of agent that can suppress cancer development

In a case of basic science detective work, researchers at The University of Texas M. D. Anderson Cancer Center have solved the puzzle of the "inconsistent biomarker" and, in the process, may have discovered an agent that can suppress cancer development.

13 Oct 2005

Technique for inserting genes into specific sites on the genome in liver cells

The genes are inserted into non-coding regions of the genome so there is no danger of interfering with the functioning of other genes. Once inserted, the gene remains a permanent part of the cell's genome.

10 Oct 2005

Pain-free needles!

Millions of fearful patients could be saved the pain of vaccinations thanks to revolutionary pain-free needles to be revealed today for the first time at the British Pharmaceutical Conference in Manchester.

28 Sep 2005

Possible therapy that could control blood pressure

A newly developed virus that introduces a blood pressure-lowering gene into cells and enables that gene to maintain blood pressure at healthy levels for four months promises to take gene therapy for the disorder a step closer to reality, said researchers at Baylor College of Medicine in a report released online in the Proceedings of the National Academy of Sciences.

27 Sep 2005

New virus-based gene therapy delivery system to help fight cancer

A Mayo Clinic research team has devised a new virus-based gene therapy delivery system to help fight cancer. Researchers say their findings will help overcome hurdles that have hindered gene therapy cancer treatments.

27 Sep 2005

Scientists reverse memory loss in mice with Alzheimer's

In mice, that had been genetically engineered to develop Alzheimer's disease, scientists were able to reverse the rodents' memory loss by reducing the amount of an enzyme that is crucial for the development of Alzheimer's disease.

26 Sep 2005

Neurologix announces positive interim results of gene therapy clinical trial for patients with Parkinson's disease

Neurologix has announced positive interim results of its landmark gene therapy clinical trial for patients with Parkinson's disease. Neurologix's 12-patient, dose-escalating Phase I trial is the world's first study to use a viral vector (the non-pathogenic adeno-associated virus, or AAV) for the treatment of an adult neurological disease.

26 Sep 2005

Next generation of effective viral-based therapies for cancer

Researchers at Columbia University Medical Center continue to make strides in their work to develop the next generation of effective viral-based therapies for cancer.

21 Sep 2005

Nitric oxide synthase plays a role in peripheral vascular disease

The enzyme nitric oxide synthase plays a role in peripheral vascular disease, a common disease that impairs the mobility of 25 percent of people over the age of 50, according to a Yale study in the Proceedings of the National Academy of Sciences (PNAS).

26 Aug 2005

New strategy to turn off the function of a special group of T cells to suppress immune response to tumors

A special stretch of genetic material may turn off the immune suppression that stymies attempts to fight cancer with a vaccine, said researchers at Baylor College of Medicine (BCM) at Houston.

26 Aug 2005

First clinical trial of a biologic nanoparticle designed to give back to cancer patients the tumor-busting gene they have lost

The phase I clinical study will enroll 20 patients with advanced solid cancers (including most common tumor types), and is the culmination of more than a decade of work by a team of researchers led by Professor Esther H. Chang, Ph.D. at the Lombardi Comprehensive Cancer Center.

25 Aug 2005

Gene therapy advance shows potential for developing improved treatment for hemophilia

A virus that typically infects insects could help with the development of gene therapy treatment for Hemophilia A, a condition in which even a bump on the knee can cause serious internal bleeding in people.

22 Aug 2005

Malfunctioning bone marrow cells cause neuropathy in many people with diabetes

Malfunctioning bone marrow cells that produce insulin appear to cause a dangerous nerve condition called neuropathy that disables many people with diabetes, said a research team led by Baylor College of Medicine (BCM) in Houston.

22 Aug 2005