A recent review published in the journal Engineering delves into the significant advancements and potential of CRISPR ...

It uses CRISPR/Cas9 to make a patient’s haematopoietic stem cells produce high levels of foetal haemoglobin (HbF) in red blood cells, by introducing a gene known as BCL11A which down-regulates ...

and colleagues that identified BCL11A as a key genetic switch. 4 And of course, the development of CRISPR tools by Nobel laureates Jennifer Doudna, PhD, and Emmanuelle Charpentier, PhD ...

Crispr Therapeutics AG reported significant ... cells are edited at the erythroid specific enhancer region of the BCL11A gene. This edit results in the production of high levels of fetal ...

CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient's own hematopoietic stem and progenitor cells are edited at the erythroid ...