美国马萨诸塞大学医学院Guangping Gao等研究人员,讨论了CRISPR基因组编辑疗法的策略与体内递送。相关综述论文于2020年4月2日发表于《细胞》。
Title: CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors
Author: Dan Wang, Feng Zhang, Guangping Gao
Issue&Volume: 2020/04/02
Abstract: The development of clustered regularly interspaced short-palindromic repeat (CRISPR)-based biotechnologies has revolutionized the life sciences and introduced new therapeutic modalities with the potential to treat a wide range of diseases. Here, we describe CRISPR-based strategies to improve human health, with an emphasis on the delivery of CRISPR therapeutics directly into the human body using adeno-associated virus (AAV) vectors. We also discuss challenges facing broad deployment of CRISPR-based therapeutics and highlight areas where continued discovery and technological development can further advance these revolutionary new treatments.
DOI: 10.1016/j.cell.2020.03.023
Source: https://www.cell.com/cell/fulltext/S0092-8674(20)30285-3
本期文章:《细胞》:Online/在线发表