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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3 in 1: team finds the gene responsible for three forms of childhood neurodegenerative diseases

A Montreal-led international team has identified the mutated gene responsible for three forms of leukodystrophies, a group of childhood-onset neurodegenerative disorders. Mutations in this gene were identified in individuals from around the world but one mutation occurs more frequently in French-Canadian patients from Quebec. Published in the September issue of the American Journal of Human Genetics and selected for the Editors’ Corner of the journal, the findings are crucial to the development of diagnostic tests and genetic counseling for families, and provide insights into a new mechanism for these disorders of the brain.

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Gene therapy kills breast cancer stem cells, boosts chemotherapy

Gene therapy delivered directly to a particularly stubborn type of breast cancer cell causes the cells to self-destruct, lowers chance of recurrence and helps increase the effectiveness of some types of chemotherapy, researchers at The University of Texas MD Anderson Cancer Center reported in the Sept. 13 edition of Cancer Cell.

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Scientists unravel the cause of rare genetic disease: Goldman-Favre Syndrome explained

Bethesda, MD—A new research report published in The FASEB Journal (https://www.fasebj.org) will help ophthalmologists and scientists better understand a rare genetic disease that causes increased susceptibility to blue light, night blindness, and decreased vision called Enhanced S-Cone Syndrome or Goldman-Favre Syndrome. In the report, scientists found that the expression of genes responsible for the healthy renewal of rods and cones in the retina was reduced and that this problem originates in the photoreceptors themselves rather than in the adjacent retinal pigment epithelial layer as once thought.

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‘Gene overdose’ causes extreme thinness

Scientists have discovered a genetic cause of extreme thinness for the first time, in a study published today in the journal Nature.

The research shows that people with extra copies of certain genes are much more likely to be very skinny. In one in 2000 people, part of chromosome 16 is duplicated, making men 23 times and women five times more likely to be underweight.

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Single vaccines to protect against both rabies and Ebola

Partnering with the NIH, Thomas Jefferson University successfully tests a vaccine in mice that could lead to a more effective public health tool in Africa

PHILADELPHIA—Researchers from Thomas Jefferson University, among other institutions, including the National Institute of Allergy and Infectious Diseases, have developed single vaccines to protest against both rabies and the Ebola virus.

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Building a better antipsychotic drug by treating schizophrenia’s cause

The classic symptoms of schizophrenia – paranoia, hallucinations, the inability to function socially—can be managed with antipsychotic drugs. But exactly how these drugs work has long been a mystery.

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