Single-Cell Brain Atlas Links Noncoding DNA to Neurodegenerative Risk
A single-cell brain atlas links APA and 3′UTR variation to Alzheimer’s, Parkinson’s and other brain-disease risk.
Research into genes, DNA and the genome, and what they reveal about health and disease.
Ultracompact Gene Tool Could Open the Door to Smaller, More Flexible Therapies
Stanford researchers unveiled TIGRa, an ultracompact gene activation tool that may improve delivery of future therapies.
Researchers Report a Turning Point for Gene Editing in Severe Blood Disorders
Gene editing for severe blood disorders shows new promise after trial results suggest a reliable therapeutic strategy.
First CRISPR Phase 3 Trial Points to a Potential One-Time Treatment for Hereditary Angioedema
First phase 3 CRISPR trial for hereditary angioedema shows major attack reduction after one infusion.
CRISPR Therapy Restores Partial Vision in Early Human Trial, Offering Hope for Inherited Retinal Disease
A first-in-human CRISPR gene-editing trial for inherited retinal disease showed early improvements in vision and no serious treatment-related adverse events, say researchers.
Early Clinical Success for CRISPR Base Editing in Inherited Retinal Disease
Preliminary phase 1/2 results show safety and visual improvements with a CRISPR base-editing therapy for an inherited retinal disorder, prompting calls for larger trials.
