TN2016000460A1 - Aav vectors for retinal and cns gene therapy. - Google Patents

Aav vectors for retinal and cns gene therapy.

Info

Publication number
TN2016000460A1
TN2016000460A1 TN2016000460A TN2016000460A TN2016000460A1 TN 2016000460 A1 TN2016000460 A1 TN 2016000460A1 TN 2016000460 A TN2016000460 A TN 2016000460A TN 2016000460 A TN2016000460 A TN 2016000460A TN 2016000460 A1 TN2016000460 A1 TN 2016000460A1
Authority
TN
Tunisia
Prior art keywords
cns
raav
gene therapy
improved
disorders
Prior art date
Application number
TN2016000460A
Inventor
Abraham Scaria
Lisa Stanek
Jennifer Sullivan
Original Assignee
Genzyme Corp
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Priority claimed from PCT/US2015/028966 external-priority patent/WO2015168666A2/en
Publication of TN2016000460A1 publication Critical patent/TN2016000460A1/en

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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

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.
TN2016000460A 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy. TN2016000460A1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201461988131P 2014-05-02 2014-05-02
PCT/US2015/028966 WO2015168666A2 (en) 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy

Publications (1)

Publication Number Publication Date
TN2016000460A1 true TN2016000460A1 (en) 2018-04-04

Family

ID=58700040

Family Applications (1)

Application Number Title Priority Date Filing Date
TN2016000460A TN2016000460A1 (en) 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy.

Country Status (3)

Country Link
AR (1) AR100274A1 (en)
MA (1) MA39439B2 (en)
TN (1) TN2016000460A1 (en)

Also Published As

Publication number Publication date
AR100274A1 (en) 2016-09-21
MA39439B2 (en) 2020-11-30
MA39439A1 (en) 2018-06-29

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