CRISPR Editing Heads Off Disease in Mouse Livers
CRISPR Editing Heads Off Disease in Mouse Livers
Separate proof-of-concept studies report success against two inherited diseases.
CRISPR Editing Heads Off Disease in Mouse Livers
CRISPR Editing Heads Off Disease in Mouse Livers

Separate proof-of-concept studies report success against two inherited diseases.

Separate proof-of-concept studies report success against two inherited diseases.

gene therapy
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.
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.