gene therapy
Slow March Toward a Canavan Cure
Slow March Toward a Canavan Cure
Ashley Yeager | May 1, 2018
Two decades after a successful crowdfunding campaign, some clinical trial patients have seen improvements—but there’s still no approved treatment for the disease.
The Philadelphia Family Helping to Drive Research on MLD
The Philadelphia Family Helping to Drive Research on MLD
Catherine Offord | May 1, 2018
Though Calliope Joy’s disease is too far progressed to be treatable, her parents have helped other children with metachromatic leukodystrophy get access to an experimental therapy.
A Devastating Diagnosis, a Viral Video, and a Clinical Trial
A Devastating Diagnosis, a Viral Video, and a Clinical Trial
Diana Kwon | May 1, 2018
When the O’Neills learned that their daughter had Sanfilippo syndrome, a devastating rare disease, they created a GoFundMe campaign that raised $2 million in less than a year.
Families of Children with Rare Diseases Fuel Gene Therapy Research
Families of Children with Rare Diseases Fuel Gene Therapy Research
The Scientist Staff | May 1, 2018
Crowdfunding can power investigations into diseases that would otherwise receive little attention.
Salk Institute Suspends Cancer Scientist Inder Verma
Salk Institute Suspends Cancer Scientist Inder Verma
Ashley Yeager | Apr 23, 2018
The prominent researcher has been put on administrative leave pending an investigation into unspecified allegations.
World’s Largest Cell and Gene Therapy Plant Opens
World’s Largest Cell and Gene Therapy Plant Opens
Catherine Offord | Apr 11, 2018
Lonza will employ more than 200 full-time staff to work at the Texas-based facility, the company says.
Vision Restored: The Latest Technologies to Improve Sight
Vision Restored: The Latest Technologies to Improve Sight
Anna Azvolinsky | Apr 10, 2018
Cell implants, gene therapy, even optogenetics are making headway in clinical trials to treat various forms of blindness.  
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. 
Sanofi and Celgene Spend $20 Billion in Major Biotech Acquisitions
Sanofi and Celgene Spend $20 Billion in Major Biotech Acquisitions
Katarina Zimmer | Jan 23, 2018
Pharmaceutical giant Sanofi to take over hemophilia drugmaker Biovertiv, while Celgene will buy cancer drugmaker Juno Therapeutics.
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.