Testing the efficacy of new NF1 gene therapy candidates in human cell types

While intervention approaches range from NF1 gene replacement to targeting a genetic factor in the Ras signaling pathway are promising, there is currently no humanized model to develop an efficient in vivo transduction. In this proposal, we will develop a new gene therapy platform with NF1-depleted human Schwann cells, neurons, and cardiomyocytes, for a new NF1 gene replacement candidate and revealing a genetic ‘Achilles heel’ to neutralize a key signaling, followed by in vivo testing with a humanized mouse model. Our study will provide a humanized pre-clinical model to accelerate current efforts toward curing neurofibromatosis.

Investigators

Gabsang Lee, PhD

Johns Hopkins University School of Medicine

Gabsang Lee, PhD

Johns Hopkins University School of Medicine

Current Stage

Discovery

Discovery
In Vivo Proof of Concept
IND Enabling
Clinical Trial Phase 1
Clinical Trial Phase 2
Clinical Trial Phase 3