Positive Trial Results for Experimental DMD Gene Therapy
Preliminary data from Sarepta Therapeutics has exceeded expectations, causing a surge in company stock prices this week.
Positive Trial Results for Experimental DMD Gene Therapy
Positive Trial Results for Experimental DMD Gene Therapy

Preliminary data from Sarepta Therapeutics has exceeded expectations, causing a surge in company stock prices this week.

Preliminary data from Sarepta Therapeutics has exceeded expectations, causing a surge in company stock prices this week.

adeno-associated virus
Severe Toxicity Reported in High-Dose AAV Gene Therapy in Animals
Severe Toxicity Reported in High-Dose AAV Gene Therapy in Animals
Jim Daley | Jan 31, 2018
Biotech stocks fell in response to the news. 
Gene Therapy Temporarily Reverses Type 1 Diabetes in Mice
Gene Therapy Temporarily Reverses Type 1 Diabetes in Mice
Shawna Williams | Jan 4, 2018
Pancreatic cells engineered to produce insulin did not immediately provoke an immune response.
Ancient Viruses as Gene Therapy Vectors
Ancient Viruses as Gene Therapy Vectors
Ashley P. Taylor | Jul 31, 2015
Researchers deploy ancestors of today’s adeno-associated viruses to deliver gene therapies without immune system interference.
Reducing Gene Therapy-Related Risk
Reducing Gene Therapy-Related Risk
Kerry Grens | Jan 22, 2015
In a mouse model of a rare disease, scientists have figured out how to reduce the elevated cancer risk tied to a gene therapy treatment.
Gene Therapy Arrives in Europe
Gene Therapy Arrives in Europe
Sabrina Richards | Nov 6, 2012
The European Commission approves the Western hemisphere’s first gene therapy, aimed at correcting a lipid-processing disorder.