	US 11,730,829 B2
	Intrathecal delivery of recombinant adeno-associated virus 9
Brian K. Kaspar, Westerville, OH (US); Arthur Burghes, Columbus, OH (US); and Paul Porensky, Columbus, OH (US)
Assigned to NATIONWIDE CHILDREN'S HOSPITAL, Columbus, OH (US); and OHIO STATE INNOVATION FOUNDATION, Columbus, OH (US)
Filed by NATIONWIDE CHILDREN'S HOSPITAL, Columbus, OH (US); and OHIO STATE INNOVATION FOUNDATION, Columbus, OH (US)
Filed on Jun. 4, 2018, as Appl. No. 15/997,433.
Application 15/997,433 is a continuation of application No. 14/417,823, abandoned, previously published as PCT/US2013/053065, filed on Jul. 31, 2013.
Claims priority of provisional application 61/678,458, filed on Aug. 1, 2012.
Prior Publication US 2019/0151477 A1, May 23, 2019
This patent is subject to a terminal disclaimer.
Int. Cl. A61K 48/00 (2006.01); A61K 35/76 (2015.01); C12N 15/864 (2006.01); A61K 38/17 (2006.01); A61K 47/00 (2006.01); C12N 15/86 (2006.01); A61K 49/04 (2006.01); C07K 14/47 (2006.01); C07H 21/04 (2006.01)

CPC A61K 48/0075 (2013.01) [A61K 38/1709 (2013.01); A61K 48/0008 (2013.01); A61K 49/0438 (2013.01); C07K 14/47 (2013.01); C12N 15/86 (2013.01); A61K 48/00 (2013.01); C07H 21/04 (2013.01); C12N 2750/14143 (2013.01)]

10 Claims

1. A method of delivering a polynucleotide sequence to the central nervous system of a patient suffering from spinal muscle atrophy, the method comprising intrathecally administering a combination of rAAV9 and a non-ionic, low-osmolar contrast agent to the patient,

wherein the non-ionic, low-osmolar contrast agent is iobitridol, iohexol, iomeprol, iopamidol, iopentol, iopromide, ioversol or ioxilan, and

wherein the rAAV9 comprises a self-complementary genome comprising a promoter and the polynucleotide sequence encoding the survival motor neuron (SMN) protein, and

wherein the polynucleotide sequence is delivered to the central nervous system of the patient suffering from spinal muscular atrophy.