RNA editing as a gene therapy approach for Rett Syndrome
Guoping Feng, Ph.D., Feng Zhang, Ph.D.
Chenjie received a B.S. in pharmaceutical engineering from Wenzhou Medical University. In 2019, he completed his Ph.D. in neuroscience from Zhejiang University under the supervision of Prof. Xiao-Ming Li. In his doctoral work, he discovered neuropeptide cholecystokinin as a marker to define the valence of different BLA-NAc sub-circuits and found CB1R in the CCKBLA-D2NAc circuit as a powerful determinant of depressive-like behavior in mice. As a Simons’ fellow in Feng lab, he aims to develop and test the effectiveness of Cas13-based RNA editing in mice as a means for gene therapy targeting Rett Syndrome.
CRISPR/Cas-based genome editing approaches pose the threat of introducing permanent off -target mutations into the genome. To avoid these limitations of DNA nucleases, approaches that instead directly target RNA would be highly desirable, as these would enable tunability, reversibility, and importantly no off-target mutations would be permanent. In Feng lab, I aim to develop therapeutics targeting Rett syndrome by using the CRSIPR/Cas13 platform that can edit RNA.
- Shen, C., Zheng, D., Li, K. et al. Cannabinoid CB1 receptors in the amygdalar cholecystokinin glutamatergic afferents to nucleus accumbens modulate depressive-like behavior. 2019. Nature Medicine, 25, 337–349
- Yang, J., Shen, C., Chen, X. et al. Erbb4 Deficits in Chandelier Cells in the Medial Prefrontal Cortex Confer Cognitive Dysfunctions: Implications for Schizophrenia. 2018. Cerebral Cortex, 22. doi:10.1093/cercor/bhy316
Keywords: Programmable RNA editing, Rett syndrome, Mecp2, Cas13-ADAR system