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Therapeutic Genome Editing and In Vivo Delivery.
Ramirez-Phillips AC, Liu D. Ramirez-Phillips AC, et al. AAPS J. 2021 Jun 2;23(4):80. doi: 10.1208/s12248-021-00613-w. AAPS J. 2021. PMID: 34080099 Review.
Several CRISPR/Cas9-based therapeutics have reached the clinical stage, including treatments for inherited red blood cell disorders and Leber Congenital Amaurosis 10, as well as CRISPR/Cas9-edited T cells designed to target and destroy cancer cells. .. …
Several CRISPR/Cas9-based therapeutics have reached the clinical stage, including treatments for inherited red blood cell disorders and L …