Molecular Therapy

Medical research

Patient-specific stem cells and personalized gene therapy

(Medical Xpress)—Columbia University Medical Center (CUMC) researchers have created a way to develop personalized gene therapies for patients with retinitis pigmentosa (RP), a leading cause of vision loss. The approach, ...

Genetics

Genetic editing shows promise in Duchenne muscular dystrophy

Using a novel genetic 'editing' technique, Duke University biomedical engineers have been able to repair a defect responsible for one of the most common inherited disorders, Duchenne muscular dystrophy, in cell samples from ...

Medical research

Engineered stem cell advance points toward treatment for ALS

Transplantation of human stem cells in an experiment conducted at the University of Wisconsin-Madison improved survival and muscle function in rats used to model ALS, a nerve disease that destroys nerve control of muscles, ...

HIV & AIDS

Immune cells engineered in lab to resist HIV infection

Researchers at the Stanford University School of Medicine have found a novel way to engineer key cells of the immune system so they remain resistant to infection with HIV, the virus that causes AIDS.

Oncology & Cancer

Oral breast cancer vaccine may offer new prevention tool

(Medical Xpress)—A new oral vaccine that produces a novel two-pronged immune system attack on cancer cells could be effective in preventing breast cancer recurrence, according to findings from a collaborative research team ...

Medical research

Gene therapy generates new neurons to treat Huntington's disease

Huntington's disease (HD) is a rare disease characterized by abnormal chorea movement and caused by the Huntingtin (Htt) gene mutation and neurodegeneration in a brain area called the striatum. A research group led by Dr. ...

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