CRISPR-Cas Genome Editing in <i>Ex Vivo</i> Human Lungs to Rewire the Translational Path of Genome-Targeting Therapeutics.

ABSTRACT: 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 owing to the limited modalities available. This study introduces a novel platform for testing genome editing in human lungs ex vivo, effectively simulating preimplantation genetic engineering

SUBMITTER: Mesaki K 

PROVIDER: S-EPMC11386987 | biostudies-literature | 2024 Jun

REPOSITORIES: biostudies-literature