Pan, Meiliang, 2017-2018

Meiliang Pan
Meiliang
Pan

"Treating Charcot-Marie-Tooth disease by using Genome Surgery Approach "

Host Mentor: Bruce Conklin, MD
Gladstone Institute of Cardiovascular Disease, UCSF

Genetics diseases are difficult to treat and the available drugs target small percentage of genetics diseases and provide no cures. With more understanding in human genome and CRISPR technology, genome editing could lead to cures. Dominate negative disease such as Charcot-Marie-Tooth provides a promising therapeutic targets for CRISPR technology to selectively inactivate the disease allele to test if cellular function can be restored. The overall goals of my project are establish a robust protocol for motor neuron differentiation in patient derived induced pluripotent stem cell for physiological testing of Charcot-Marie-Tooth type2 (CMT2) disease and identify most efficient methods of introducing CRISPR guide RNA into iPSC derived motor neuron for allele specific editing.