Amyotrophic lateral sclerosis (ALS) is a devastating motor neuron disease characterized by progressive muscle weakness and wasting, with eventual paralysis. Patients with ALS lose the ability to walk, speak, swallow, and breathe, which leads to respiratory failure and death. The only currently approved therapy for ALS delays the onset of ventilator dependence and death by only 1-2 months, leaving a signficant unmet need for disease-modifying therapies. Approximately 10% of cases of ALS are hereditary forms of the disease caused by specific gene mutations that result in the production of abnormal proteins that are toxic to motor neurons. ODC director Dr. Wilson and his team within Penn's Gene Therapy Program are developing AAV gene therapy approaches to treat both hereditary and non-hereditary forms of ALS.
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