Cellectis: new approach to treating congenital metabolic diseases using modified stem cells







Photo credit © Cellectis – Ramon Martinez

(Boursier.com) — The biotechnology company Cellectis published a scientific article in Molecular Therapy, demonstrating that editing of introns by TALEN on hematopoietic stem and progenitor cells (HSPCs) makes it possible to limit the expression of therapeutic transgenes to the myeloid lineage.

This approach could open new therapeutic avenues for the treatment of congenital metabolic diseases and neurological diseases that require the delivery of therapeutic products into the brain.

During the session, Cellectis shares rose by 0.8% to 2.645 euros.


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