GENE THERAPY VECTORS FOR TREATMENT OF DANON DISEASE

The present disclosure provides gene therapy vectors comprising a polynucleotide sequence encoding a LAMP-2 polypeptide, methods of use thereof, pharmaceutical compositions, and more. In particular, the disclosure provides recombinant AAV vectors having AAVrh74 serotype expressing LAMP-2A, LAMP-2B,...

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Bibliographische Detailangaben
Hauptverfasser:	WONG, RODERICK, YALAMANCHI, NAVEEN, KERAVALA, ANNAHITA, PRATUMSUWAN, PIRATIP, PRABHAKAR, RAJ, SHAH, GAURAV
Format:	Patent
Sprache:	eng ; fre
Schlagworte:	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 SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	The present disclosure provides gene therapy vectors comprising a polynucleotide sequence encoding a LAMP-2 polypeptide, methods of use thereof, pharmaceutical compositions, and more. In particular, the disclosure provides recombinant AAV vectors having AAVrh74 serotype expressing LAMP-2A, LAMP-2B, or LAMP-2C for use as a therapeutic in, for example, Danon Disease. La présente invention concerne des vecteurs de thérapie génique comprenant une séquence polynucléotidique codant pour un polypeptide LAMP-2, leurs procédés d'utilisation, des compositions pharmaceutiques, etc,. En particulier, l'invention concerne des vecteurs AAV recombinés ayant un sérotype AAVrh74 exprimant LAMP-2A, LAMP-2B ou LAMP-2C pour une utilisation comme agent thérapeutique, par exemple, la maladie de Danon.