Early Results of First In Vivo Gene-Editing Trial Encouraging
Early Results of First In Vivo Gene-Editing Trial Encouraging
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.
Early Results of First In Vivo Gene-Editing Trial Encouraging
Early Results of First In Vivo Gene-Editing Trial Encouraging

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.

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.

gene therapy
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.
The NIH Loosens Grip on Gene Therapy Trials
The NIH Loosens Grip on Gene Therapy Trials
Kerry Grens | Aug 16, 2018
The agency proposes ceding its scrutiny of these studies to the FDA.
Reprogrammed Müller Glia Restore Vision in Mice
Reprogrammed Müller Glia Restore Vision in Mice
Ashley Yeager | Aug 15, 2018
A double gene-transfer therapy transformed the non-neuronal cells into rod photoreceptors in the retinas of animal models of congenital blindness.
A Noninvasive Way to Control Individual Brain Regions
A Noninvasive Way to Control Individual Brain Regions
Catherine Offord | Jul 13, 2018
Researchers use a combination of ultrasound waves, genetic engineering, and synthetic drugs to switch specific neurons on and off in mice.
Positive Trial Results for Experimental DMD Gene Therapy
Positive Trial Results for Experimental DMD Gene Therapy
Catherine Offord | Jun 20, 2018
Preliminary data from Sarepta Therapeutics has exceeded expectations, causing a surge in company stock prices this week.