gene therapy
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.
Child Receives Transgenic Skin
Child Receives Transgenic Skin
Ruth Williams | Jan 1, 2018
A combination gene-and-cell therapy has given a boy with a grievous skin disease a new lease on life, and resolved a dermatology debate to boot.
CRISPR Proves Promising for Treating ALS in Mice
CRISPR Proves Promising for Treating ALS in Mice
Katarina Zimmer | Dec 21, 2017
The gene-editing tool was effective in disabling a defective gene responsible for some forms of amyotrophic lateral sclerosis. 
CRISPR Helps Mice Hear
CRISPR Helps Mice Hear
Abby Olena | Dec 20, 2017
Researchers reduce the severity of hereditary deafness in mice with the delivery of CRISPR-Cas9 protein-RNA complexes that inactivate a mutant gene in their inner ears. 
Study Pinpoints Potential “Master Regulator” of Age-Related Cognitive Decline
Study Pinpoints Potential “Master Regulator” of Age-Related Cognitive Decline
Shawna Williams | Dec 18, 2017
Upping a gene’s expression in rat brains made them better learners and normalized the activity of hundreds of other genes to resemble the brains of younger animals.
Optogenetic Therapies Move Closer to Clinical Use
Optogenetic Therapies Move Closer to Clinical Use
Shawna Williams | Nov 16, 2017
With a clinical trial underway to restore vision optogenetically, researchers also see promise in using the technique to treat deafness, pain, and other conditions.
Man Receives First In Vivo Gene-Editing Therapy
Man Receives First In Vivo Gene-Editing Therapy
Kerry Grens | Nov 15, 2017
The 44-year-old patient has Hunter syndrome, which doctors hope to treat using zinc finger nucleases.
Child Receives Transgenic Skin Over Most of His Body
Child Receives Transgenic Skin Over Most of His Body
Ruth Williams | Nov 8, 2017
A combination gene-and-cell therapy has given a boy with a grievous skin disease a new lease on life, and has resolved a dermatology debate to boot.