AAV VECTOR TREATMENT METHODS FOR LATE INFANTILE NEURONAL CEROID LIPOFUSCINOSIS TYPE 2

Disclosed herein are methods for treating a primate in need of tripeptidyl peptidase 1 (TPP1), comprising (a) providing a recombinant adeno-associated virus (AAV) vector comprising a nucleic acid encoding TPP1; and (b) administering an amount of the recombinant AAV vector to the central nervous syst...

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Hauptverfasser: ANDERSON DAVID WILLIAM, TOTO MARYANN, DASEN SUE E I
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creator ANDERSON DAVID WILLIAM
TOTO MARYANN
DASEN SUE E I
description Disclosed herein are methods for treating a primate in need of tripeptidyl peptidase 1 (TPP1), comprising (a) providing a recombinant adeno-associated virus (AAV) vector comprising a nucleic acid encoding TPP1; and (b) administering an amount of the recombinant AAV vector to the central nervous system (CNS) of the primate, wherein the TPP1 is expressed in the primate. 本文公开了用于治疗需要三肽基肽酶1(TPP1)的灵长类动物的方法,该方法包括(a)提供包含编码TPP1的核酸的重组腺相关病毒(AAV)载体;以及(b)向灵长类动物的中枢神经系统(CNS)施用一定量的重组AAV载体,其中TPP1在灵长类动物中表达。
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subjects CHEMISTRY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
ORGANIC CHEMISTRY
PEPTIDES
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
title AAV VECTOR TREATMENT METHODS FOR LATE INFANTILE NEURONAL CEROID LIPOFUSCINOSIS TYPE 2
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