作者
Namrata Chaudhari, Amanda M Rickard, Suki Roy, Peter Dröge, Harshyaa Makhija
发表日期
2020/12
期刊
Stem Cell Research & Therapy
卷号
11
页码范围
1-16
出版商
BioMed Central
简介
Background
The precise, functional and safe insertion of large DNA payloads into host genomes offers versatility in downstream genetic engineering-associated applications, spanning cell and gene therapies, therapeutic protein production, high-throughput cell-based drug screening and reporter cell lines amongst others. Employing viral- and non-viral-based genome engineering tools to achieve specific insertion of large DNA—despite being successful in E. coli and animal models—still pose challenges in the human system. In this study, we demonstrate the applicability of our lambda integrase-based genome insertion tool for human cell and gene therapy applications that require insertions of large functional genes, as exemplified by the integration of a functional copy of the F8 gene and a Double Homeobox Protein 4 (DUX4)-based reporter cassette for potential hemophilia A gene therapy and …
引用总数
学术搜索中的文章
N Chaudhari, AM Rickard, S Roy, P Dröge, H Makhija - Stem Cell Research & Therapy, 2020