Efficacy of dual intracerebroventricular and intravitreal <i>CLN5</i> gene therapy in sheep prompts the first clinical trial to treat CLN5 Batten disease.

Front Pharmacol · 2023 · PMC10628725 · PMID 37942487

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did not reach statistical significanceno p-value reported

Storage accumulation was lower in the retina of the treated eye compared with the untreated eye for all early symptomatic treated sheep although this did not reach statistical significance ( Supplementary Figure S8B ).

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