gene therapy

Cell implants, gene therapy, even optogenetics are making headway in clinical trials to treat various forms of blindness.  

Biotech stocks fell in response to the news. 

Pharmaceutical giant Sanofi to take over hemophilia drugmaker Biovertiv, while Celgene will buy cancer drugmaker Juno Therapeutics.

Pancreatic cells engineered to produce insulin did not immediately provoke an immune response.

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.

The gene-editing tool was effective in disabling a defective gene responsible for some forms of amyotrophic lateral sclerosis. 

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. 

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.

With a clinical trial underway to restore vision optogenetically, researchers also see promise in using the technique to treat deafness, pain, and other conditions.

The 44-year-old patient has Hunter syndrome, which doctors hope to treat using zinc finger nucleases.

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.

Spark Therapeutics’s Luxturna would be the first approved therapy in the U.S. that replaces or repairs a defective gene inherited from one’s parents.

Researchers use gold nanoparticles to deliver CRISPR-Cas9 and correct a point mutation in a mouse model of Duchenne muscular dystrophy. 

A small patch of engineered cells makes an enzyme that stimulates insulin release.

Researchers restored muscle function in animals with muscular dystrophy.

A Food and Drug Administration advisory panel unanimously calls for agency approval of the cell therapy for the treatment of resistant leukemia. 

Sangamo Therapeutics will use zinc finger nucleases to introduce the gene for a missing clotting factor into the livers of men with hemophilia B.

A small Phase 1 clinical trial reports that, even at high doses, the treatment did not lead to adverse reactions.

Scientists shut down cancer-causing fusion genes with CRISPR.

Researchers demonstrate the delivery of AAV-packaged CRISPR-CjCas9 into the muscles and eyes of mice.