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.

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

Participants in the gene therapy market discover new ways to improve clinical efficiency and generate revenue

The gene therapy market has had limited success with products during the clinical development process. Due to toxicological complications and poor therapy efficacy, healthcare authorities have suspended several gene therapy trials worldwide.

28 Jul 2005

$4.7million grant for research on neuroblastoma

Significant funding from Australia's National Health and Medical Research Council will enable researchers at Children’s Cancer Institute Australia (CCIA) for Medical Research to make progress on the treatment of one the most aggressive, drug-resistant childhood cancers - neuroblastoma - over the next five years.

28 Jul 2005

Nerve-insulating substance called myelin can lead to functional improvements in animals with spinal cord injury

Combining partially differentiated stem cells with gene therapy can promote the growth of new "insulation" around nerve fibers in the damaged spinal cords of rats, a new study shows.

26 Jul 2005

Potential new treatment for nerve damage caused by diabetes

Research into a new treatment for nerve damage caused by diabetes could bring relief to millions of diabetic patients, say experts.

From The University of Manchester 26 Jul 2005

Molecular link between a viral infection and development of a common and fatal form of liver cancer

Researchers at The University of Texas M. D. Anderson Cancer Center have uncovered a crucial molecular link between a viral infection and development of a common and fatal form of liver cancer. In the process, they have identified a possible way to treat this disease as well as a number of other cancers.

26 Jul 2005

Role of IL-24 in regulating the immune system

Researchers at Introgen Therapeutics and their collaborators at The University of Texas M. D. Anderson Cancer Center have published new findings on the role of IL-24 in regulating the immune system in the current online issue of the Journal of Leukocyte Biology.

21 Jul 2005

Gene Therapy News and Research

Further Reading

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

Participants in the gene therapy market discover new ways to improve clinical efficiency and generate revenue

$4.7million grant for research on neuroblastoma

Nerve-insulating substance called myelin can lead to functional improvements in animals with spinal cord injury

Potential new treatment for nerve damage caused by diabetes

Molecular link between a viral infection and development of a common and fatal form of liver cancer

Role of IL-24 in regulating the immune system

Crucial molecular link between a hepatitis B virus (HBV) and hepatocellular carcinoma (HCC)

Chromos Molecular Systems issued four key patents

AAV2 has great potential to be developed as an anti-cancer agent

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

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