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

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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Bibliographische Detailangaben
Hauptverfasser: ANDERSON DAVID WILLIAM, TOTO MARYANN, DASEN SUE E I
Format: Patent
Sprache:chi ; eng
Schlagworte:
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
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Zusammenfassung: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。