Can gene therapy cure fatal diseases in children?

In order for the body to function, a balance is necessary between the cells that build up the bones in our skeletons and the cells that break them down. In the disease malignant infantile osteopetrosis, MIOP, the cells that break down the bone tissue do not function as they should, resulting in the skeleton not having sufficient cavities for bone-marrow and nerves.

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Gene therapy treatment extends lives of mice with fatal disease, MU study finds

 

A team of University of Missouri researchers has found that introducing a missing gene into the central nervous system could help extend the lives of patients with Spinal Muscular Atrophy (SMA) ? the leading genetic cause of infantile death in the world.

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Gene therapy can correct forms of severe combined immunodeficiency

Severe combined immunodeficiency is defect in the immune system that results in a loss of the adaptive immune cells known as B cells and T cells. Mutations in several different genes can lead to the development of severe combined immunodeficiency, including mutation of the adenosine deaminase (ADA) gene. Traditional treatment options, such as enzyme replacement therapy, are of limited efficacy, but bone marrow transplant from a compatible donor leads to a better response. A recent clinical trial indicated that gene therapy to insert the correct ADA gene in the patient’s own bone marrow cells can also lead to a good response.

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Key gene found responsible for chronic inflammation, accelerated aging and cancer

Researchers at NYU School of Medicine have, for the first time, identified a single gene that simultaneously controls inflammation, accelerated aging and cancer.

“This was certainly an unexpected finding,” said principal investigator Robert J. Schneider, PhD, the Albert Sabin Professor of Molecular Pathogenesis, associate director for translational research and co-director of the Breast Cancer Program at NYU Langone Medical Center. “It is rather uncommon for one gene to have two very different and very significant functions that tie together control of aging and inflammation. The two, if not regulated properly, can eventually lead to cancer development. It’s an exciting scientific find.”

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Delivery of gene-therapy for heart disease boosted 1fold; now in 1patient trial

Cardium Therapeutics (NYSE Amex: CXM) today announced a late-breaking poster presentation at the American Society of Gene & Cell Therapy (ASGCT) 15th Annual Meeting being held May 19, 2012 at the Pennsylvania Convention Center in Philadelphia, PA.

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New York Stem Cell Foundation scientist grows bone from human embryonic stem cells

Dr. Darja Marolt, an Investigator at The New York Stem Cell Foundation (NYSCF) Laboratory, is lead author on a study showing that human embryonic stem cells can be used to grow bone tissue grafts for use in research and potential therapeutic application. Dr. Marolt conducted this research as a post-doctoral NYSCF – Druckenmiller Fellow at Columbia University in the laboratory of Dr. Gordana Vunjak-Novakovic.

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Researchers develop gene therapy that could correct a common form of blindness

A new gene therapy method developed by University of Florida researchers has the potential to treat a common form of blindness that strikes both youngsters and adults. The technique works by replacing a malfunctioning gene in the eye with a normal working copy that supplies a protein necessary for light-sensitive cells in the eye to function. The findings are published today (Monday, Jan. 23) in the Proceedings of the National Academy of Sciences online.

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New gene study of ADHD points to defects in brain signaling pathways

Pediatric researchers analyzing genetic influences in attention-deficit/hyperactivity disorder (ADHD) have found alterations in specific genes involved in important brain signaling pathways. The study raises the possibility that drugs acting on those pathways might offer a new treatment option for patients with ADHD who have those gene variants—potentially, half a million U.S. children.

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Phase I trial begins using gene therapy and bone marrow stem cells in the treatment of brain cancer

Initiated by Lentigen and UH Case Medical Center

Cleveland, OH. and Gaithersburg, MD. – University Hospitals (UH) Case Medical Center, Case Western Reserve University School of Medicine and Lentigen Corporation announced today the initiation of a novel Phase I clinical trial of LG631 gene therapy for the protection of hematopoietic stem cells (HSCs) from the dose limiting toxicity of chemotherapy with Temodar.

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UCLA psychologists discover a gene’s link to optimism, self-esteem

UCLA life scientists have identified for the first time a particlular gene’s link to optimism, self-esteem and “mastery,” the belief that one has control over one’s own life — three critical psychological resources for coping well with stress and depression.

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