Ocular therapies for neuronal ceroid lipofuscinoses: more than meets the eye

por Murray, Samantha J., Mitchell, Nadia L.
Publicado 2022

The Translational Benefits of Sheep as Large Animal Models of Human Neurological Disorders

por Murray, Samantha J., Mitchell, Nadia L.
Publicado 2022

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

por 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.
Publicado 2023

Progressive MRI brain volume changes in ovine models of CLN5 and CLN6 neuronal ceroid lipofuscinosis

por Murray, Samantha J, Almuqbel, Mustafa M, Felton, Simon A, Palmer, Nickolas J, Myall, Daniel J, Shoorangiz, Reza, Ella, Arsène, Keller, Matthieu, Palmer, David N, Melzer, Tracy R, Mitchell, Nadia L
Publicado 2023

Long-term safety and dose escalation of intracerebroventricular CLN5 gene therapy in sheep supports clinical translation for CLN5 Batten disease

por 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.
Publicado 2023

Intraparenchymal convection enhanced delivery of AAV in sheep to treat Mucopolysaccharidosis IIIC

por O’Leary, Claire, Forte, Gabriella, Mitchell, Nadia L., Youshani, Amir Saam, Dyer, Adam, Wellby, Martin P., Russell, Katharina N., Murray, Samantha J., Jolinon, Nelly, Jones, Simon A, Stacey, Kevin, Davis, Daniel M., Henckaerts, Els, Palmer, David N., Kamaly-Asl, Ian, Bigger, Brian W.
Publicado 2023