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:	eng ; fre
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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Beschreibung
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. L'invention concerne des procédés de traitement d'un primate ayant besoin d'une protéase de tripeptidyl 1 (TPP1), comprenant (a) la fourniture d'un vecteur d'un virus adéno-associé (AVV) recombinant comprenant un acide nucléique codant la TPP1 ; et (b) l'administration d'une quantité du vecteur AAV recombinant au système nerveux central (SNC) du primate, la TPP1 étant exprimé dans le primate.