Current approaches and potential challenges in the delivery of gene editing cargos into hematopoietic stem and progenitor cells

Frontiers in Genome Editing

Genome Editing

Srujan Marepally¹  Saravanabhavan Thangavel¹  Karthik V. Karuppusamy²  Ramya Murugesan² 

[1] Centre for Stem Cell Research (CSCR), A Unit of InStem Bengaluru, Christian Medical College Campus, Vellore, Tamil Nadu, India;Centre for Stem Cell Research (CSCR), A Unit of InStem Bengaluru, Christian Medical College Campus, Vellore, Tamil Nadu, India;Manipal Academy of Higher Education, Manipal, Karnataka, India;

关键词: hematopoietic stem cells;    gene therapy;    gene editing;    gene delivery;    in vivo;   

DOI  :  10.3389/fgeed.2023.1148693

 received in 2023-01-20, accepted in 2023-08-17,  发布年份 2023

来源: Frontiers

【 摘 要 】

Advancements in gene delivery and editing have expanded the applications of autologous hematopoietic stem and progenitor cells (HSPCs) for the treatment of monogenic and acquired diseases. The gene editing toolbox is growing, and the ability to achieve gene editing with mRNA or protein delivered intracellularly by vehicles, such as electroporation and nanoparticles, has highlighted the potential of gene editing in HSPCs. Ongoing phase I/II clinical trials with gene-edited HSPCs for β-hemoglobinopathies provide hope for treating monogenic diseases. The development of safe and efficient gene editing reagents and their delivery into hard-to-transfect HSPCs have been critical drivers in the rapid translation of HSPC gene editing into clinical studies. This review article summarizes the available payloads and delivery vehicles for gene editing HSPCs and their potential impact on therapeutic applications.

【 授权许可】

Unknown   
Copyright © 2023 Murugesan, Karuppusamy, Marepally and Thangavel.