UMASS Exploring New Gene Therapy Project for NF1

Author: Miguel Sena-Esteves, PhD - UMASS Medical School
Published On: 08/20/2019

Karen Peluso and Lori Ryan of NF Northeast and Kim Bischoff, executive director of the NF Network, met with Miguel Sena-Esteves, PhD, a researcher at UMASS Medical School. Dr. Sena- Esteves informed us about his exciting new project to develop gene therapies for NF1.

The funding for Dr. Sena-Esteves’ project was awarded by The Gilbert Family Foundation of Detroit.  The Gilbert Family Foundation has committed to a $12 million gene therapy initiative to address the underlying cause of NF1.  This work will accelerate the development of therapies that address the core genetic abnormalities in NF1. This three-year initiative hopes to identify promising gene therapy techniques for treating NF1.

The first two years will focus on two different approaches which will be explored first in cell cultures and proceed to the use of NF1 mouse models.  Efforts will be focused on two principal areas: 1) the design of improved viral delivery systems for delivering therapeutic cargo such as neurofibromin to target cell types such as Schwann cells, and 2) the development of specialty proteins and molecules to boost neurofibromin levels in cells with insufficient neurofibromin.  The goal, should the project succeed, is to seek FDA approval for a Phase 1 study in humans within three years.  

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