Kumi Mesaki, Haruchika Yamamoto, Stephen Juvet, Jonathan Yeung, Zehong Guan, Akhi Akhter, Yan Yao, Cameron Dickie, Henna Mangat, Aizhou Wang, Gavin W Wilson, Andrea Mariscal, Jim Hu, Alan R Davidson, Benjamin P Kleinstiver, Marcelo Cypel, Mingyao Liu, Shaf Keshavjee
The ongoing advancements in CRISPR-Cas technologies can significantly accelerate the preclinical development of both in vivo and ex-vivo organ genome-editing therapeutics. One of the promising applications is to genetically modify donor organs prior to implantation. The implantation of optimized donor organs with long-lasting immunomodulatory capacity holds promise for reducing the need for lifelong potent whole-body immunosuppression in recipients However, assessing genome-targeting interventions in a clinically-relevant manner prior to clinical trials remains a major challenge due to the limited modalities available...
May 8, 2024: Human Gene Therapy