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publicationDate 2020-10-22^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber WO-2020214737-A1
titleOfInvention Gene therapy for treating or preventing visual effects in batten disease
abstract The present disclosure relates to gene therapy methods of preserving photoreceptors and/or inhibiting or preventing retinal degeneration in Batten disease patients, including recombinant adeno-associated virus (rAAV) delivery of a neuronal ceroid lipofuscinosis neuronal 6 (CLN6) polynucleotide.
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