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

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 ...

Medical research

One-time treatment rescues lethal metabolic liver disease in mice

To treat the liver disorder tyrosinaemia type I, one of the most severe forms of the disease, doctors typically use drug therapy. However the treatment is lifelong and a residual risk of liver cancer persists, which is usually ...

Medical research

Technique uses 3-D weaving to grow a living hip replacement

With a goal of treating worn, arthritic hips without extensive surgery to replace them, scientists have programmed stem cells to grow new cartilage on a 3-D template shaped like the ball of a hip joint. What's more, using ...

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

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.

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 ...

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