Gene Therapy Effective for Severe Combined Immunodeficiency
Gene Therapy Effective for Severe Combined Immunodeficiency
Researchers report they’ve found a way to restore immune function in infants with one form of “bubble boy disease.”
Gene Therapy Effective for Severe Combined Immunodeficiency
Gene Therapy Effective for Severe Combined Immunodeficiency

Researchers report they’ve found a way to restore immune function in infants with one form of “bubble boy disease.”

Researchers report they’ve found a way to restore immune function in infants with one form of “bubble boy disease.”

gene therapy
Image of the Day: Cone Preservation
Image of the Day: Cone Preservation
Chia-Yi Hou | Apr 30, 2019
Researchers treat mice with retinitis pigmentosa using a gene therapy that reduces cone loss.
Preliminary Results Point to Success of In Vivo Gene Editing
Preliminary Results Point to Success of In Vivo Gene Editing
Carolyn Wilke | Feb 12, 2019
Two studies show signs that the introduced DNA is functioning, but it’s too early to know if patients actually benefit.
CRISPR Editing Heads Off Disease in Mouse LiversCRISPR Editing Heads Off Disease in Mouse Livers
CRISPR Editing Heads Off Disease in Mouse Livers
Shawna Williams | Oct 9, 2018
Separate proof-of-concept studies report success against two inherited diseases.
Early Results of First In Vivo Gene-Editing Trial Encouraging
Early Results of First In Vivo Gene-Editing Trial Encouraging
Kerry Grens | Sep 5, 2018
Two patients who received Sangamo’s zinc finger–based treatment for Hunter syndrome have lower biomarkers of the condition, but no signs of new enzyme production.
Putting Exosomes to Work
Putting Exosomes to Work
Ruth Williams | Sep 1, 2018
Researchers identify a handy tool for tinkering with the versatile vesicles.
Infographic: Directing Exosome Traffic
Infographic: Directing Exosome Traffic
Ruth Williams | Sep 1, 2018
Researchers harness a protein to send cargo-carrying exosomes to mouse muscles.
CRISPR Treatment for Duchenne Muscular Dystrophy Helps Dogs
CRISPR Treatment for Duchenne Muscular Dystrophy Helps Dogs
Catherine Offord | Aug 31, 2018
Researchers boosted levels of the dystrophin protein to nearly normal levels in a canine model of the disease.
Gene Editing Could One Day Treat Muscle Disorders
Gene Editing Could One Day Treat Muscle Disorders
Sandeep Ravindran | Aug 30, 2018
Scientists race to develop CRISPR therapies that could save the lives of kids with muscle-wasting conditions.
Cell and Gene Therapy Tracker: Global CAR T-Cell Trials
Cell and Gene Therapy Tracker: Global CAR T-Cell Trials
The Scientist Staff | Aug 22, 2018
Investigators have launched numerous clinical trials that test the efficacy of the immunotherapy. Here is a global accounting of these experiments.