#gene therapy

Gene therapy for Huntington’s shows promising clinical trial data

Huntington’s Disease is one of the classic “uncurable” neurodegenerative diseases. A genetic mutation produces a mutant form of a protein (called Huntingtin) which leads to sudden onset of symptoms which gets progressively worse until death. It’s so bad that it’s considered a “textbook” example of the need for genetic counseling when people are considering getting their DNA...