Early Results Are Positive for Experimental CRISPR Therapies
Early Results Are Positive for Experimental CRISPR Therapies
Two clinical trial participants—one with β-thalassemia and one with sickle cell disease—appeared to benefit from the gene-editing treatments with minimal side effects, according to the companies.
Early Results Are Positive for Experimental CRISPR Therapies
Early Results Are Positive for Experimental CRISPR Therapies

Two clinical trial participants—one with β-thalassemia and one with sickle cell disease—appeared to benefit from the gene-editing treatments with minimal side effects, according to the companies.

Two clinical trial participants—one with β-thalassemia and one with sickle cell disease—appeared to benefit from the gene-editing treatments with minimal side effects, according to the companies.

clinical research
Trial of Gene Therapy for Duchenne Muscular Dystrophy Put on Hold
Trial of Gene Therapy for Duchenne Muscular Dystrophy Put on Hold
Catherine Offord | Nov 12, 2019
The US Food and Drug Administration halts a study by Solid Biosciences after a patient experiences severe side effects following treatment.
CRISPR-Edited T Cells Used in Cancer Patients for the First Time in the US
CRISPR-Edited T Cells Used in Cancer Patients for the First Time in the US
Emily Makowski | Nov 6, 2019
The initial findings from a small clinical trial of patients with multiple myeloma or sarcoma suggest that gene-edited immunotherapy is safe.
Neural Stem Cell Transplantation Crawls Toward the Clinic
Neural Stem Cell Transplantation Crawls Toward the Clinic
Ashley P. Taylor | Oct 29, 2019
Several early-stage clinical trials indicate that implanting patients with the cells is safe. But whether they can alleviate neurological problems remains to be seen.
CBD Linked to Sleep Disturbances in Adolescent Rats
CBD Linked to Sleep Disturbances in Adolescent Rats
Emily Makowski | Oct 23, 2019
The animals showed changes in both slow wave and rapid eye movement sleep.
Image of the Day: Tau Aggregation
Image of the Day: Tau Aggregation
Emily Makowski | Oct 17, 2019
Endolysosome leakiness allows tau to build up in cells.
<em>NPSR1</em> Variant Linked to Less Sleep in People: Study
NPSR1 Variant Linked to Less Sleep in People: Study
Emily Makowski | Oct 16, 2019
“Short sleeper” mice engineered to have the same sequence in the gene sleep less but show the same performance on memory tests as animals that sleep a normal amount.
Study Topic Influences Funding Disparity for Black Scientists
Study Topic Influences Funding Disparity for Black Scientists
Ashley Yeager | Oct 10, 2019
A new analysis finds that black scientists tend to propose projects that have lower rates of funding from the National Institutes of Health than other fields.
Image of the Day: Patient-Derived Organoids
Image of the Day: Patient-Derived Organoids
Emily Makowski | Oct 10, 2019
Three-dimensional tissue cultures grown from cancer patients’ own tumors can predict responses to the drug irinotecan.
Tau Linked to RNA Splicing Errors in Flies
Tau Linked to RNA Splicing Errors in Flies
Emily Makowski | Oct 8, 2019
In brain samples from people with Alzheimer’s disease, the protein aggregates more strongly bound proteins involved in processing RNA, the same study finds.