A new UCLA Health study has discovered in mouse models that genes associated with repairing mismatched DNA are critical in eliciting damages to neurons that are most vulnerable in Huntington's disease ...

Ongoing launch of CASGEVY ® continues to gain momentum; new cell patient collection initiations expected to significantly grow in 2025- -More than 50 authorized treatment centers (ATCs) activated ...

A new study has discovered in mouse models that genes associated with repairing mismatched DNA are critical in eliciting damages to neurons that are most vulnerable in Huntington's disease and ...

Vimentin is a type III intermediate filament (IF) protein normally expressed in cells that develop into connective tissue, ...

January 31, 2025 expert reaction to NICE final draft guidance on exagamglogene autotemcel (exa-cel) for severe sickle cell disease . Scientists comment on final draft guidance fro ...

CHICAGO (Reuters) — Scientists have characterized the role of thousands of mutations in the BRCA2 cancer gene, findings that may help reassure worried patients about their cancer risk or guide doctors ...

However, the impact of this therapeutic approach on cardiac iron overload requires further investigation. Gene therapy is a potentially curative treatment for transfusion-dependent thalassemia (4). It ...

Casgevy enables CRISPR/Cas-9 to edit the BCL11A gene in stem cells to produce fetal hemoglobin. The edited genes are infused into a patient's bone marrow to start producing red blood cells with ...