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.

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

Gene therapy success for the treatment of congenital muscular dystrophy (CMD) in mice

Researchers from the University of Pittsburgh report the first study to achieve success with gene therapy for the treatment of congenital muscular dystrophy (CMD) in mice, demonstrating that the formidable scientific challenges that have cast doubt on gene therapy ever being feasible for children with muscular dystrophy can be overcome.

15 Aug 2005

Experimental therapy could offer new way to treat spine damage

An experimental therapy that combines stem cells and gene therapy to repair spinal cord injuries in rats may lead to a new way to treat the same injury in humans.

15 Aug 2005

Combined gene therapy eliminates glioblastoma multiforme in lab studies

Despite aggressive treatment, glioblastoma multiforme (GBM) - the most common and deadly of brain cancers - usually claims the lives of its victims within six to 12 months of diagnosis. Because GBM is so aggressive, the disease has been the target of a number of laboratory and clinical studies investigating the effectiveness of gene therapy to deliver novel therapies to the brain.

15 Aug 2005

New gene technique useful for identifying genes and drug targets for diseases such as cancers and diabetes

A collaborative project between American and Chinese researchers developed a way to study the function of genes in mice and man by using a moveable genetic element from moths, according to a report in the journal Cell.

13 Aug 2005

How negatively charged lipids stick to negatively charged DNA and self-organize into structures

Scientists studying the structure and interaction of negatively charged lipids and DNA molecules have created a "cookbook" for a class of nontoxic DNA delivery systems that will assist doctors and clinicians in the safe and effective delivery of genetic medicine.

13 Aug 2005

Findings could lead to gene therapy cure for childhood blindness

In findings that could lead to curing some forms of congenital blindness through gene therapy, researchers at UCLA have discovered that RPE65, a gene missing in infants born with the blinding disease Leber congenital amaurosis, is also a key enzyme in the visual cycle. The identity of this enzyme has long been a mystery to scientists.

11 Aug 2005

Gene therapy used to successfully treat haemophilia in dogs

Progress in gene therapy to treat haemophilia has been impressive in the past few years. Gene therapy has been used to successfully treat haemophilia in dogs.

9 Aug 2005

Researchers develop new diagnostic methods to better detect future monkeypox or smallpox outbreaks

The research also sheds new light on the 2003 monkeypox outbreak in the Midwest - monkeypox is closely related to smallpox. This new information suggests that the 2003 outbreak was larger than the 72 cases reported by the Centers for Disease Control and Prevention (CDC).

9 Aug 2005

Study results reaffirm cyclin D1 as a candidate target for molecular therapeutic control of breast tumor development

For about a decade, scientists have recognized that many cases of hereditary breast cancer result from a mutation of a specific gene called BRCA1, which, in its normal state, helps keep tumor formation in check. About five to 10 percent of breast cancer cases arise from these genetic miscues, about half of which are linked to the abnormal functioning of BRCA1.

9 Aug 2005

Scientists use gene transfer to prevent retinoschisis

University of Florida scientists used a healthy human gene to prevent blindness in mice with a form of an incurable eye disease that strikes boys.

2 Aug 2005

Protein called cyclin D1 disrupts BRCA1's normal role as a cancer inhibitor

For about a decade, scientists have recognized that many cases of hereditary breast cancer result from a mutation of a specific gene called BRCA1, which, in its normal state, helps keep tumor formation in check. About five to 10 percent of breast cancer cases are linked to genetic miscues, about half of which are linked to BRCA1.

2 Aug 2005

Gene Therapy News and Research

Further Reading

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

Scientists reverse memory loss in mice with Alzheimer's

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

Next generation of effective viral-based therapies for cancer

Nitric oxide synthase plays a role in peripheral vascular disease

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

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

Gene therapy advance shows potential for developing improved treatment for hemophilia

Malfunctioning bone marrow cells cause neuropathy in many people with diabetes

Gene therapy success for the treatment of congenital muscular dystrophy (CMD) in mice

Experimental therapy could offer new way to treat spine damage

Combined gene therapy eliminates glioblastoma multiforme in lab studies

New gene technique useful for identifying genes and drug targets for diseases such as cancers and diabetes

How negatively charged lipids stick to negatively charged DNA and self-organize into structures

Findings could lead to gene therapy cure for childhood blindness

Gene therapy used to successfully treat haemophilia in dogs

Researchers develop new diagnostic methods to better detect future monkeypox or smallpox outbreaks

Study results reaffirm cyclin D1 as a candidate target for molecular therapeutic control of breast tumor development

Scientists use gene transfer to prevent retinoschisis

Protein called cyclin D1 disrupts BRCA1's normal role as a cancer inhibitor

Sensing a Healthier Future

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

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