James Walker, Ph.D. of the Center for Genomic Medicine at MGH was one of two recipients to receive funding from the Children’s Tumor Foundation in the form of a NF1 Gene Therapy Initiative Award. Each award is for $240,000 for a duration of two years.

Dr. Walker’s project, entitled “Development of NF1 therapeutics with CRISPR-based technologies,” focuses on investigating the feasibility of using genome editing (both CRISPR-based homology-directed repair and base editing) as a therapeutic approach to correct three pathogenic NF1 mutations in cultured human Schwann cells. His team will capitalize on recently developed CRISPR/Cas9 and -Cas12a variants, which increase the targeting range, activity, and fidelity (reducing off-targets) of gene-editing in collaboration with Dr. Ben Kleinstiver of the Center for Genomic Medicine at MGH.

With a view to developing the most promising strategies into potential therapies for NF1 tumors, the Walker team will also initiate a screen to optimize viral vehicles for Schwann cells that will be essential for in vivo delivery of CRISPR genome engineering tools with Dr. Ben Deverman, Director of Vector Engineering at the Broad Institute.

Samantha Ginn, Ph.D., at Children’s Medical Research Institute in Australia also received an NF1 Gene Therapy Initiative Award. Dr. Ginn and her team propose to use a CRISPR/Cas9-based homology-independent targeted integration (HITI) approach to replace large sections of mutated NF1 gene. In contrast to methods targeting individual patient-specific mutations, this approach has the advantage of targeting multiple mutations with a single gene editing vector, and thus will be applicable to many NF1 patients.

To find out more information about the CTF NF1 Gene Therapy Initiative and other projects funded by the Children’s Tumor Foundation, visit www.ctf.org.