New hope for Huntington's families as gene therapy shows remarkable results

A company called uniQure has announced promising results from a trial of a new gene therapy for Huntington's disease. The ...
Nature

Huntington’s disease treated for first time using gene therapy

Preliminary results from a small trial offer the clearest evidence yet that the brain disease’s progression can be slowed.
BioPharma Dive

UniQure to seek approval of Huntington’s gene therapy after trial win

New study data showed treatment slowed signs of disease progression by 75% after three years, a finding that could have ...
Ars Technica

From birth to gene-edited in 6 months: Custom therapy breaks speed limits

News broke yesterday that researchers in Philadelphia appear to have successfully treated a 6-month-old baby boy, called KJ, with a personalized CRISPR gene-editing therapy. The treatment corrects an ...

Preliminary but promising gene therapy shows 75% reduction in Huntington's disease progression

A one-time experimental treatment shows unprecedented success in slowing Huntington's disease, according to UCL researchers.

Experimental gene therapy found to slow Huntington’s disease progression, company says

An experimental gene therapy was found to slow the progression of Huntington’s disease in a “pivotal” study, according to ...

Gene Therapy Could End Lifelong Transfusions For Rare Blood Disorder

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the ...
Live Science on MSN

Science history: A tragic gene therapy death that stalled the field for a decade — Sept. 17, 1999

Jesse Gelsinger died after receiving a gene therapy treatment to treat a liver disease. The death sparked an investigation ...