CRISPR/Cas9: An inexpensive, efficient loss of function tool to screen human disease genes in Xenopus.

ABSTRACT: Congenital malformations are the major cause of infant mortality in the US and Europe. Due to rapid advances in human genomics, we can now efficiently identify sequence variants that may cause disease in these patients. However, establishing disease causality remains a challenge. Additionally, in the case of congenital heart disease, many of the identified candidate genes are either novel to embryonic development or have no known function. Therefore, there is a pressing need to develop inexpensive and efficient technologies to screen these candidate genes for disease phenocopy in model systems and to perform functional studies to uncover their role in development. For this purpose, we sought to test F0 CRISPR based gene editing as a loss of function strategy for disease phenocopy in the fr

SUBMITTER: Bhattacharya D 

PROVIDER: S-EPMC4684459 | biostudies-literature | 2015 Dec

REPOSITORIES: biostudies-literature