Publications by authors named "Saima N Kayani"

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First-in-human high dose AAV9 intrathecal gene therapy for paediatric CLN7 disease: a phase 1, open-label, single ascending dose, non-randomised clinical trial.
Benjamin M Greenberg, Berge Minassian, Souad Messahel, Veronica Bordes Edgar, Andrea Lowden, Saima N Kayani

EBioMedicine· November 2025

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Long-term progression of retinal degeneration in a preclinical model of CLN7 Batten disease as a baseline for testing clinical therapeutics.
Ashley A Rowe, Xin Chen, Emily R Nettesheim, Yacine Issioui, Thomas Dong, Saima N Kayani

EBioMedicine· November 2022

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