These clinical SMA studies suggest that the technology developed in the Wilson laboratory is capable of safely and efficiently delivering genes to motor neurons, which in turn can be a mechanism for treating ALS.

The Motor Neuron Disease Program of Excellence will proceed in three phases. Initial studies will focus on gene therapy for inherited forms of ALS, beginning with patients who have defects in the C9orf72 gene. In parallel, Wilson and his colleagues will evaluate strategies based on the expression of genes encoding neuroprotective factors. This approach has the potential to benefit a larger population of patients with ALS, although it is associated with more technical risk of failure. Third, the researchers will explore the use of genome editing technologies as a more precise and durable approach to correcting mutations in genes such as C9orf72.


A group of six international experts will serve as external advisors for the program. "I am honored to serve as the chair of the advisory committee of the Program of Excellence in Motor Neuron Disease of the Orphan Disease Program at Penn,” said Siddharthan Chandran, PhD, FRCP, who is chair of advisory committee and director of the Centre for Clinical Brain Sciences at the University of Edinburgh. “A greater understanding of the pathogenesis of ALS, together with advances in gene therapy that have come out of Penn, create incredible opportunities to make a difference against this devastating disease."

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