Magnetic Resonance Imaging as a Readout of CLN5 Gene Therapy Efficacy in Sheep

Magnetic Resonance Imaging as a Readout of CLN5 Gene Therapy Efficacy in Sheep

ABSTRACT Purpose Neuronal ceroid lipofuscinoses (NCL; Batten disease) are a group of rare inherited neurodegenerative disorders caused by mutations in one of 13 ceroid lipofuscinosis neuronal (CLN) genes. The diseases share a common set of symptoms, including motor and cognitive dysfunction, progres...

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Main Authors: Samantha J. Murray, Mustafa M. Almuqbel, Simon A. Felton, Nickolas J. Palmer, Ashley R. Deane, Daniel J. Myall, Reza Shoorangiz, Arsène Ella, Matthieu Keller, David N. Palmer, Tracy R. Melzer, Nadia L. Mitchell
Format: Article
Language:English
Published: Wiley 2025-04-01
Series:Brain and Behavior
Subjects:
batten disease
CLN5
gene therapy
intracerebroventricular
intravitreal
magnetic resonance imaging
Online Access:https://doi.org/10.1002/brb3.70431
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https://doi.org/10.1002/brb3.70431

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