Archives:
NF1 Gene Rescue in Translational Animal Models
The goal of this project is to assess gene replacement and gene-editing strategies to restore neurofibromin activity in genetically engineered animals harboring NF1 patient specific …
Genome Editing for the Treatment of Neurofibromatosis Type I
Therapies currently in development for NF1 do not address the fundamental underlying genetic mutation that causes this disorder. In contrast, gene editing technologies, including those …
Pre-clinical development of AAV9-NF1 gene therapy for neurofibromatosis type 1
In vivo delivery of adeno-associated virus (AAV) vectors is the leading approach to develop gene therapies for genetic diseases with AAV9 being the most utilized …
RAFT-polymerized nanoparticles for delivery of NF1 cDNA to Schwann cells
Reconstituting functional neurofibromin by delivering the correct version of the gene to cells in the NS presents a promising treatment that has high potential for …
Optimization of Base and Prime Editing Strategies for NF1 Therapeutics
Recent advances in CRISPR-based gene editing strategies may offer lifelong restoration of neurofibromin deficiency by permanent correction of NF1 mutations. In this project, we plan …
Targeted delivery of full length NF1 cDNA using non-viral vectors
Development of an effective gene replacement therapy for NF1 is complicated by the large size of the gene affected in this disease, preventing its packaging …
AAV, Zinc Finger Protein and Antisense Oligonucleotide Strategies for Restoration of Neurofibromin Expression
Neurofibromatosis type I (NF1) is a genetic disorder caused by mutations in the neurofibromin gene. Current treatment options are limited and do not address the …
NF1 Gene Rescue in Translational Animal Models
The goal of this project is to test new technologies for correcting NF1 mutations at the genome or DNA level instead of current efforts that …
Genome Editing with Engineered Vectors to Correct Neurofibromatosis Type I
Neurofibromatosis type I (NF1) is caused by mutations to the NF1 gene that lead to the development of tumors of the peripheral nervous system. Gene …