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US 10,369,193 B2Granted
Gene therapy for neurodegenerative disorders
AssigneeGenzyme Corporation
InventorsMarco A. Passini, Lamya Shihabuddin, Seng H. Cheng
Life of this patent
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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.
Claims13
A recombinant adeno-associated virus (rAAV) virion for use in treating spinal muscular atrophy (SMA) in a subject comprising a self-complementary adeno-associated virus (scAAV) vector, wherein the scAAV vector comprises a polynucleotide encoding survival motor neuron (SMN), wherein the scAAV vector comprises AAV2 inverted terminal repeats (ITRs), and wherein the rAAV virion comprises an AAV8 capsid.
Dependencies are inferred from the claim text.