Researchers at Oregon Health & Science University have identified a gene that plays a crucial role in the brain's ability to ...

Remember The Girls, a 501(c)(3) non-profit organization dedicated to supporting females impacted by X-linked genetic ...

Kiran Musunuru, a University of Pennsylvania cardiologist and leading gene-editing researcher, thinks it’s possible. Musunuru cofounded Verve Therapeutics, a company developing a therapy that uses ...

Researchers have identified two genes from the human genome’s “dark matter” that influence brain size and synaptic signaling.

What scientists once dismissed as junk DNA may actually be some of the most powerful code in our genome. A new international ...

What makes the human brain distinctive? A new study published in Cell identifies two genes linked to human brain features and ...

Well, cells have confounders, too.” As one example of how causarray can be used, Roeder said that the tool will be essential in the analysis of data from CRISPR (which stands for clustered regularly ...

Background Alexander disease is an autosomal dominant leukodystrophy caused by heterozygous pathogenic variants in the glial ...

Sangamo Therapeutics is continuing to enroll and treat patients with a rare genetic condition called Fabry disease in an early clinical trial of a gene therapy it is developing, announcing Tuesday ...

Acumen and JCR aim to develop a product leveraging Acumen's amyloid beta oligomer-selective antibody expertise and JCR's transferrin-receptor-targeting blood-brain barrier-penetrating technologyPrecli ...

Preclinical candidate data expected in early 2026, with exclusive option for Acumen to develop up to two development candidates for the treatment of Alzheimer’s disease Agreement follows more than one ...

Efforts to accelerate research in rare lysosomal diseases are gaining momentum through newly awarded grants supporting ...