GENE THERAPY FOR NEURONAL CEROID LIPOFUSCINOSES

Provided herein are methods and compositions for treatment of Batten disease. Such compositions include a recombinant adeno-associated virus (rAAV), said rAAV comprising an AAV capsid, and a vector genome packaged therein, said vector genome comprising (a) an AAV 5' inverted terminal repeat (IT...

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Main Authors KONDRAGUNTA, BHARGAVI, MELETHIL, SUBHA KARUMUTHIL, GERNER, FRANZ MICHAEL, MARSHALL, TRISTAN JAMES, STADELMAN, ROBERT THOMAS, BEE, JARED, CARRELL, MICHAEL JAMES, BUSS, NICHOLAS ALEXANDER PIERS SASCHA, GUIBERT, MATTHIEU PIERRE, MERCER, ANDREW CHRISTOPHER, DEPAZ, ROBERTO
Format Patent
LanguageEnglish
French
Published 22.05.2020
Subjects
BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
ORGANIC CHEMISTRY
PEPTIDES
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
VINEGAR
WINE
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Summary:Provided herein are methods and compositions for treatment of Batten disease. Such compositions include a recombinant adeno-associated virus (rAAV), said rAAV comprising an AAV capsid, and a vector genome packaged therein, said vector genome comprising (a) an AAV 5' inverted terminal repeat (ITR) sequence; (b) a promoter; (c) a CLN2 coding sequence encoding a human TPP1; (d) an AAV 3' ITR. Also provided herein are methods of treating Batten disease comprising administering to a subject in need thereof the rAAV described herein via more than one route. Also provide herein are pharmaceutical compositions comprising the rAAV described herein and related methods of treating Batten disease. L'invention concerne des procédés et des compositions pour le traitement de la maladie de Batten. De telles compositions comprennent un virus adéno-associé recombinant (VAAr), ledit VAAr comprenant une capside de virus adéno-associé (VAA), et un génome vectoriel encapsidé dans celle-ci, ledit génome vectoriel comprenant (a) une séquence terminale inversée répétée (ITR) de VAA 5'; (b) un promoteur; (c) une séquence de codage de CLN2 codant pour un TPP1 humain; (d) une ITR de VAA 3'. L'invention concerne également des méthodes de traitement de la maladie de Batten comprenant l'administration à un sujet, ayant besoin d'un tel traitement, dudit VAAr par l'intermédiaire de plus d'une voie. L'invention concerne également des compositions pharmaceutiques comprenant le dit VAAr et des méthodes associées de traitement de la maladie de Batten.
Bibliography:Application Number: CA20193117982