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GENE THERAPY FOR TREATING OR PREVENTING VISUAL EFFECTS IN BATTEN DISEASE
GENE THERAPY FOR TREATING OR PREVENTING VISUAL EFFECTS IN BATTEN DISEASE
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机译:基因疗法治疗或防止视觉在板条疾病影响
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摘要
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 vims (rAAV) delivery of a neuronal ceroid lipofuscinosis neuronal 6 (CLN6) polynucleotide.
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