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Current trends in gene therapy to treat inherited disorders of the brain

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How capsid screening could unleash CNS genetic therapies at scale

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AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease

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Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in brain

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Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in brain

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