Medical research

First evidence of clinical stabilization in Tay-Sachs

Preliminary data from an expanded access study of an investigational gene therapy in two patients with infantile Tay-Sachs disease indicates the potential to modify the rate of disease progression, according to an upcoming ...

Genetics

Promising gene replacement therapy moves forward

Research led by Dr. Krystof Bankiewicz, who recently joined The Ohio State University College of Medicine, shows that gene replacement therapy for Niemann-Pick type A disease is safe for use in nonhuman primates and has therapeutic ...

Genetics

Brighter possibilities for treating blindness

Advances in preclinical research are now being translated into innovative clinical solutions for blindness, a review published in the 10th Anniversary Series of Science Translational Medicine reports.

Genetics

New data suggests nicotine while pregnant alters genes

The Akay Lab biomedical research team at the University of Houston is reporting in the journal Nature Scientific Reports that a possible cure for addiction may be found by following the pathways of significantly altered dopamine ...

Parkinson's & Movement disorders

Innovative gene therapy trial for Parkinson's disease

A trial for a new gene therapy, known as AXO-Lenti-PD, aimed at improving the supply of dopamine in the brains of people with Parkinson's disease has been launched by researchers at UCL and University College London Hospitals ...

Medical research

New therapy could treat children with Hunter disease

A team at the University of Manchester have developed a novel stem cell gene therapy approach to treat children with a devastating genetic disease. The approach is currently being developed for clinical trial in patients ...

Genetics

Gene therapy used to treat Fabry disease—a world first

A team of Canadian physicians and researchers is believed to be the first in the world to have used gene therapy to treat a patient with Fabry disease, a rare inherited enzyme deficiency that can damage major organs and shorten ...

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