Efficacy of dual intracerebroventricular and intravitreal CLN5 gene therapy in sheep prompts the first clinical trial to treat CLN5 Batten disease
Murray, Samantha J., Wellby, Martin P., Barrell, Graham K., Russell, Katharina N., Deane, Ashley R., Wynyard, John R., Gray, Steven J., Palmer, David N., Mitchell, Nadia L.
Published in Frontiers in pharmacology (24.10.2023)
Published in Frontiers in pharmacology (24.10.2023)
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Long-term safety and dose escalation of intracerebroventricular CLN5 gene therapy in sheep supports clinical translation for CLN5 Batten disease
Mitchell, Nadia L, Murray, Samantha J, Wellby, Martin P, Barrell, Graham K, Russell, Katharina N, Deane, Ashley R, Wynyard, John R, Palmer, Madeleine J, Pulickan, Anila, Prendergast, Phillipa M, Casy, Widler, Gray, Steven J, Palmer, David N
Published in Frontiers in genetics (08.08.2023)
Published in Frontiers in genetics (08.08.2023)
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Journal Article