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US 2025/0288644 A1Published application
Gene Therapy For Neurodegenerative Disorders
AssigneeGenzyme Corporation
InventorsMarco A. PASSINI, Lamya SHIHABUDDIN, Seng H. CHENG
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Abstract
Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed.
Claims19
A self-complementary adeno-associated virus (scAAV) vector comprising a polynucleotide encoding a protein that modulates motor function in a subject with a motor neuron disorder.
Dependencies are inferred from the claim text.