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US 2024/0293581 A1Published application
Neuroprotection gene therapy
OwnerYang Huinventor
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Abstract
Compositions and methods for treating a mammalian subject for an axonopathy, including an optic nerve axonopathy, e.g. glaucoma. Aspects of the composition include a mammalian viral vector, comprising a γ-synuclein promoter, or functional fragment thereof, that promotes expression of a NMNTA2 transgene specifically in retinal ganglion cells (RGCs). Aspects of the methods include intravitreally administering the composition to treat the subject for an ON neuropathy.
Claims16
A composition comprising:
- a mammalian AAV vector, which comprises:
- a γ-synuclein (Sncg) promoter, or functional fragment thereof, that promotes expression of an operably linked coding sequence specifically in retinal ganglion cells (RGCs), and
- a sequence encoding a functional human nicotinamide mononucleotide adenylyl transferase 2 (NMNTA2) protein, or a variant thereof.
Dependencies are inferred from the claim text.