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.

science publishing, clinical trials, microbiology
Adult Cardiac Stem Cells Don’t Exist: Study
Adult Cardiac Stem Cells Don’t Exist: Study
Diana Kwon | Sep 5, 2018
A mouse study adds to the growing body of work disputing the ability of progenitor cells to regenerate muscle tissue in adult mammals’ hearts.
US Companies Launch CRISPR Clinical Trial
US Companies Launch CRISPR Clinical Trial
Catherine Offord | Sep 3, 2018
The Germany-based study will test an ex vivo genome-editing therapy for the inherited blood disorder β-thalassemia.
First iPS Cell Trial for Heart Disease Raises Excitement, Concern
First iPS Cell Trial for Heart Disease Raises Excitement, Concern
Katarina Zimmer | Aug 31, 2018
Without knowing the cells’ mechanism of action, researchers question the best way to administer them to patients.
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.
Definitive Evidence for Water Ice on the Moon: Study
Definitive Evidence for Water Ice on the Moon: Study
Ashley Yeager | Aug 21, 2018
Data from the Indian Space Research Organization’s Chandrayaan 1 spacecraft show frost on the lunar north and south poles.
Synthetic Bacteria Help Treat Phenylketonuria in MiceSynthetic Bacteria Help Treat Phenylketonuria in Mice
Synthetic Bacteria Help Treat Phenylketonuria in Mice
Diana Kwon | Aug 17, 2018
The genetically engineered probiotic, already in clinical trials, may ease patients’ strict dietary regimes.
Drug Approval Could Boost Research on Marijuana Treatment for Autism
Drug Approval Could Boost Research on Marijuana Treatment for Autism
Jessica Wright | Aug 17, 2018
Epidiolex, a cannabis-derived medicine for epilepsy, will prompt federal regulatory changes that could crack open access to study the plant.
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.