GENE THERAPY FOR NEURONAL CEROID LIPOFUSCINOSES

• 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
• Language: English; 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

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...