The new gene therapies for sickle cell disease—including the gene-editing treatment Casgevy, based on research at Boston Children's Hospital—have been game-changing for the patients who have ...

For example, Bcl11a deficiency contributes to both the abnormal generation of natural killer-like cells in the thymus and the underdevelopment of the cortex, whereas outright loss of Bcl11b does not.

BCL11A: Evidence for neuroprotective effect Researchers at the University of Bonn investigate the role of the transcription factor for dopamine-producing neurons Peer-Reviewed Publication ...

A self-inactivating lentivirus vector with a short hairpin RNA designed to interfere with BCL11A expression was inserted into autologous hematopoietic stem cells of patients with sickle cell ...

BCL11A controlled hemoglobin, silenced fetal hemoglobin expression in adult erythroid cells You've successfully added to your alerts. You will receive an email when new content is published.

Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to reactivate fetal hemoglobin synthesis through ex vivo clustered regularly interspaced short palindromic repeats (CRISPR ...

FDA approves first CRISPR therapy—here’s how it works against sickle cell The landmark treatment turns on another blood protein that prevents sickling.

Researchers from the UK have found that overactivity in a gene called BCL11A may lead to the development and progression of triple-negative breast cancer.

Drug regulators have approved a CRISPR therapy called Casgevy to treat inherited blood disorders. But what is it and how does it work?

BCL11A: Evidence for neuroprotective effect Date: September 14, 2021 Source: University of Bonn Summary: The neurotransmitter dopamine influences the activity of a wide variety of brain areas. A ...