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.

Long-term muscle improvements shown in gene therapy study in mice

Injecting a gene responsible for making a specific protein into a mouse that's used as a model for muscular dystrophy can lead to long-term improvements in the animal's muscle size and strength, a new study shows.

14 Mar 2008

Good news for people with glycogen storage disease type Ia

A gene therapy treatment that restores a missing liver enzyme in test animals could provide a cure for a rare metabolic disorder in humans, according to Duke University Medical Center researchers.

13 Mar 2008

Potential new gene therapy strategy for muscle-wasting diseases

Investigators in The Research Institute at Nationwide Children's Hospital have identified the role of a protein that could potentially lead to new clinical treatments to combat musculoskeletal diseases, including Duchenne muscular dystrophy (DMD).

11 Mar 2008

Long-term muscle improvements shown in gene therapy study in mice

11 Mar 2008

Research in diabetes surgery suggests origins of disease

Growing evidence shows that surgery may effectively cure Type 2 diabetes -- an approach that not only may change the way the disease is treated, but that introduces a new way of thinking about diabetes.

5 Mar 2008

New insight into the fundamentals of Parkinson's disease

A single change in a protein may play a role in whether someone develops Parkinson's disease, say University of Florida Genetics Institute researchers writing in a recent issue of the Proceedings of the National Academy of Sciences.

27 Feb 2008

Genetic defect causes immune deficiency and balance disorder

A genetic defect that causes a severe immune deficiency in humans may also produce balance disorders, according to a new study by researchers at the University of Iowa, The Jackson Laboratory and East Carolina University.

21 Feb 2008

Gene therapy - immune system discovery may lead to effective treatment for glioblastoma multiforme

A new gene therapy approach that attracts and "trains" immune system cells to destroy deadly brain cancer cells also provides long-term immunity, produces no significant adverse effects and -- in the process of destroying the tumor -- promotes the return of normal brain function and behavioral skills, according to a study conducted by researchers at Cedars-Sinai Medical Center's Board of Governors Gene Therapeutics Research Institute.

20 Feb 2008

New key finding in neurodevelopment

A longstanding puzzle in neurodevelopment may have yielded up a key secret. A team led by scientists at Weill Cornell Medical College says they have determined how events at the very tips of the developing neuron's long, skinny axon affect gene transcription back in the cell's distant nucleus.

20 Feb 2008

Transplanted cells can cure hemophilia

Scientists at the Albert Einstein College of Medicine of Yeshiva University have shown for the first time that transplanted cells can cure hemophilia A (the most common form of the disease) in an animal model.

18 Feb 2008

Major milestone in the culturing of human blood stem cells

Patients with leukemia, certain autoimmune diseases and genetic defects such as sickle-cell anemia can be treated with blood stem cells either from a donor's bone marrow or from cord blood - but the supply of effective stem cells often runs short.

17 Feb 2008

Gene therapy protocol at UCSD activates immune system in patients with leukemia

A research team at the Moores Cancer Center at University of California, San Diego (UCSD) reports that patients with chronic lymphocytic leukemia (CLL) who were treated with a gene therapy protocol began making antibodies that reacted against their own leukemia cells.

12 Feb 2008

Discovery of possible target for prevention and treatment of pneumonia

Researchers at Children's Hospital of Pittsburgh of UPMC have identified a key protein target that may be a crucial factor in the development of a vaccine to prevent and new therapies to treat pneumonia, the leading killer of children worldwide.

12 Feb 2008

Henry Ford Hospital expands research on gene and radiation therapy for prostate cancer

Henry Ford Hospital is embarking on an expanded major clinical trial involving the use of gene therapy in combination with radiation therapy, to determine if the combined treatment is more effective than radiation therapy alone for patients with intermediate risk prostate cancer.

7 Feb 2008

Chemical chaperone could open door to treatment of neurological disorder

An unexpected finding turned out to be a clue leading researchers at Washington University School of Medicine in St. Louis to propose a new treatment approach for Niemann-Pick disease, a rare, deadly neurodegenerative disorder.

7 Feb 2008

New cancer center at NewYork-Presbyterian/Weill Cornell

NewYork-Presbyterian Hospital/Weill Cornell Medical Center has created an expanded cancer program dedicated to advancing research, prevention and treatment of the disease. The new Cancer Center is led by renowned cancer physician-scientist Dr. Andrew Dannenberg.

5 Feb 2008

New nanomedicine research for prostate cancer

The Prostate Cancer Foundation, largely through the generosity of David H. Koch, has given $5 million to four institutions, including Weill Cornell Medical College, to support novel research in prostate cancer. The gift is one of the largest-ever individual donations for prostate cancer research.

29 Jan 2008

New target found for asthma

An enzyme released by mast cells in the lungs appears to play a key role in the tightening of airways that is a hallmark of asthma -- pointing to a potential new target for treatment against the illness.

29 Jan 2008

New approach to gene therapy

RNA interference (RNAi) represents an innovative new strategy for using small RNA molecules to silence specific genes associated with disease processes, and a series of review articles describing the state-of-the-art and potential therapeutic applications of RNAi and microRNAs will begin with two review papers in the January 2008 issue (Volume 19, Number 1) of Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc.

24 Jan 2008

Gene therapy for pain

Researchers in the Department of Medicine and Department of Neurosciences at Mount Sinai School of Medicine have discovered that chronic pain can be successfully treated with novel targeted gene therapy.

23 Jan 2008