Jennifer K Sullivan

US Patent:

20210261625, Aug 26, 2021

Filed:

Jan 29, 2021

Appl. No.:

17/162356

Inventors:

- Cambridge MA, US
Xiaoying Jin - Cambridge MA, US
Lin Liu - Cambridge MA, US
Catherine O'Riordan - Cambridge MA, US
Jennifer Sullivan - Cambridge MA, US

International Classification:

C07K 14/005
C12N 15/86

Abstract:

Provided herein are modified adeno-associated viral (AAV) capsid proteins, compositions (e.g., rAAV) comprising the capsid proteins, and nucleic acids encoding the capsid proteins. The AAV capsids provided herein confer retinal cell tropism and/or corneal cell tropism, and mediate improved transduction efficiency in clinically relevant ocular cell types such as photoreceptors and/or corneal endothelial cells. Also provided are nucleic acids encoding the capsid proteins, and AAV particles comprising the capsid proteins.

US Patent:

20170096683, Apr 6, 2017

Filed:

May 2, 2015

Appl. No.:

15/308335

Inventors:

- Cambridge MA, US
Jennifer SULLIVAN - Hudson MA, US
Lisa M. STANEK - Natick MA, US

International Classification:

C12N 15/86
C12N 15/113
A61K 48/00
C12N 7/00

Abstract:

Provided herein are improved rAAV (e.g., rAAV2, rAAVrh8R, etc.) for enhanced gene therapy of ocular disorders or CNS disorders wherein the rAAV comprise one or more substitutions of amino acids that interact with heparan sulfate proteoglycan. The invention provides methods for improved transduction of retinal cells and methods for treating ocular diseases with improved compositions of rAAV particles. Further provided herein are improved recombinant adeno-associated virus (rAAV) (e.g., rAAV2, rAAVrh8R, etc.) for enhanced gene therapy of disorders of the CNS. The invention provides methods for delivering the rAAV to the CNS, methods for treating disorders of the CNS with improved compositions of rAAV particles, and kits for delivering the rAAV to the CNS and/or treating a CNS disorder.