Mark Kleinman, MD, is the corresponding author for the article “Long-Term Evaluation of Retinal Morphology and Function in Rosa26-Cas9 Knock-In Mice” published in the International Journal of Molecular Sciences.
Dr. Mark Kleinman says that a focus on his lab is "the development of diagnostic bio-imaging tools and the design of new therapeutic approaches to vision and life-threatening eye diseases." However, one of the challenges that his lab has encountered has been limitations in the use of CRISPR/Cas9 gene editing in traditional animal models.This article evaluates the use of Rosa26-Cas9 knock-in mice to overcome the CRISPR technique limitations in murine models.The team used a number of methods to thoroughly evaluate this novel approach.
Dr. Kleinman states, “Our data suggest that the Rosa26-Cas9 knock-in mouse is an excellent model system for genetic manipulation in the RPE/choroid and neural retina using CRISPR tools. To the best of our knowledge, no study has thus far comprehensively assessed toxicity due to prolonged SpCas9 expression in the posterior segment of the eye.”
Mark Kleinman, MD, is a professor in the Quillen College of Medicine Department of Surgery and is a CIIDI member.He currently holds funding awarded by the National Eye Institute (R01EY028206) and a BrightFocus Macular Degeneration Research Award. Sushil Dubey, PhD, is a postdoctoral fellow in the Kleinman lab. He made extensive contributions to the study and publication through experiment design and approach, data analysis, and manuscript design and preparation.
To the best of our knowledge, no study has thus far comprehensively assessed toxicity
due to prolonged SpCas9 expression in the posterior segment of the eye.
Authors: Kabhilan Mohan, Sushil Kumar Dubey, Kyungsik Jung, Rashmi Dubey, Qing Jun Wang, Subhash Prajapati, Jacob Roney, Jennifer Abney, and Mark Ellsworth Kleinman (CIIDI members in bold).