Intrathecal delivery of recombinant adeno-associated virus 9

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United States of America Patent

PATENT NO 11730829
APP PUB NO 20190151477A1
SERIAL NO

15997433

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The present invention relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as SMA and ALS as well as Pompe disease and lysosomal storage disorders. It is disclosed that administration of a non-ionic, low-os-molar contrast agent, together with a rAA9 vector for the expression of Survival Motor Neuron protein, improves the survival of SMN mutant mice as compared to the administration of the expression vector alone.

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Patent OwnerAddress
OHIO STATE INNOVATION FOUNDATION1524 NORTH HIGH STREET COLUMBUS OH 43201

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Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Burghes, Arthur Columbus, US 16 65
Kaspar, Brian K Westerville, US 42 289
Porensky, Paul Columbus, US 13 64

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