This work will generate RGCs from stem cells that carry NF1 mutations to better understand how changes in the NF1 gene can affect RGC survival and function by 1) analyzing NF1 mutation induced changes in how genes are expressed at the single cell level and 2) discovering and testing molecules that directly interact with and protect Nf1-mutant RGCs.
Investigators
Donald Zack, MD, PhD
The Johns Hopkins University
Donald Zack, MD, PhD
The Johns Hopkins University
Current Stage
Discovery
In Vivo Proof of Concept
IND Enabling
Clinical Trial Phase 1
Clinical Trial Phase 2
Clinical Trial Phase 3