US 12,465,657 B2
Enhanced delivery of viral particles to the striatum and cortex
Lisa M. Stanek, Bridgewater, NJ (US); and Lamya S. Shihabuddin, West Newton, MA (US)
Assigned to Genzyme Corporation, Cambridge, MA (US)
Filed by Genzyme Corporation, Cambridge, MA (US)
Filed on Jul. 29, 2022, as Appl. No. 17/816,139.
Application 17/816,139 is a continuation of application No. 15/549,962, abandoned, previously published as PCT/US2016/017210, filed on Feb. 9, 2016.
Claims priority of provisional application 62/114,544, filed on Feb. 10, 2015.
Claims priority of provisional application 62/220,997, filed on Sep. 19, 2015.
Prior Publication US 2022/0395586 A1, Dec. 15, 2022
Int. Cl. A61K 48/00 (2006.01); A61K 9/00 (2006.01); C12N 7/00 (2006.01); C12N 15/86 (2006.01)
CPC A61K 48/0075 (2013.01) [A61K 9/0085 (2013.01); C12N 7/00 (2013.01); C12N 15/86 (2013.01); C12N 2750/14122 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14152 (2013.01)] 10 Claims
 
1. A method for delivering a recombinant adeno-associated viral (rAAV) particle comprising an AAV serotype 2 (AAV2) capsid to the central nervous system of a mammal comprising using a convection enhanced delivery system comprising a cannula to deliver a composition comprising the rAAV particle to the putamen of each hemisphere of the striatum at a rate from about 3 uL/min to about 5 μL/min, wherein the rAAV particle comprises an rAAV vector comprising a heterologous nucleic acid encoding amino acid decarboxylase and the rAAV vector is operably linked to a cytomegalovirus immediate early promoter, wherein the rAAV particle undergoes retrograde transport in the cerebral cortex, and wherein the heterologous nucleic acid is expressed in either the prefrontal association cortical areas and the extensive regions of the occipital cortex of the brain of the mammal.