BioengineeringMolecular TherapyPeer-reviewed
TALEN-mediated intron editing of HSPCs enables transgene expression restricted to the myeloid lineage
Eduardo Seclén, Jessica C. Jang, Aminah O. Lawal…·Julien Valton
This paper edits introns of HSPCs to restrict transgene expression to the myeloid lineage, demonstrating efficacy in treating metabolic diseases and reaching the brain.
4 citations