Extraneuronal pathology in a canine model of CLN2 neuronal ceroid lipofuscinosis after intracerebroventricular gene therapy that delays neurological disease progression

Canine Model ◽

Intravitreal enzyme replacement therapy attenuates retinal disease progression in a canine model of neuronal ceroid lipofuscinosis type 2 (CLN2)

10.1016/j.ymgme.2017.12.360 ◽

2018 ◽

Vol 123 (2) ◽

pp. S132 ◽

Cited By ~ 2

Author(s):

John Sinclair ◽

Rebecca Whiting ◽

Grace Robinson ◽

Katherine Bibi ◽

Annailsa Nguyen ◽

...

Keyword(s):

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Disease Progression ◽

Retinal Disease ◽

Canine Model ◽

Enzyme Replacement ◽

Intracerebroventricular gene therapy that delays neurological disease progression is associated with selective preservation of retinal ganglion cells in a canine model of CLN2 disease

Experimental Eye Research ◽

10.1016/j.exer.2016.03.023 ◽

2016 ◽

Vol 146 ◽

pp. 276-282 ◽

Cited By ~ 17

Author(s):

Rebecca E.H. Whiting ◽

Cheryl A. Jensen ◽

Jacqueline W. Pearce ◽

Lauren E. Gillespie ◽

Daniel E. Bristow ◽

...

Keyword(s):

Gene Therapy ◽

Disease Progression ◽

Retinal Ganglion Cells ◽

Neurological Disease ◽

Ganglion Cells ◽

Canine Model ◽

Retinal Ganglion ◽

Selective Preservation

Enzyme replacement therapy attenuates disease progression in a canine model of late‐infantile neuronal ceroid lipofuscinosis ( CLN 2 disease)

Journal of Neuroscience Research ◽

10.1002/jnr.23423 ◽

2014 ◽

Vol 92 (11) ◽

pp. 1591-1598 ◽

Cited By ~ 64

Author(s):

Martin L. Katz ◽

Joan R. Coates ◽

Christine M. Sibigtroth ◽

Jacob D. Taylor ◽

Melissa Carpentier ◽

...

Keyword(s):

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Disease Progression ◽

Canine Model ◽

Enzyme Replacement ◽

A canine model for neuronal ceroid lipofuscinosis highlights the promise of gene therapy for lysosomal storage diseases

Annals of Translational Medicine ◽

10.21037/atm.2016.10.16 ◽

2016 ◽

Vol 4 (S1) ◽

pp. S20-S20

Author(s):

Jonathan E. Phillips ◽

Richard H. Gomer

Keyword(s):

Gene Therapy ◽

Lysosomal Storage Diseases ◽

Canine Model ◽

Lysosomal Storage ◽

Storage Diseases ◽

Intracerebroventricular (ICV) Recombinant Human Tripeptidyl Peptidase-1 (rhTPP1) Enzyme Replacement Attenuates Disease Progression in a Canine Model of Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)

10.1016/j.ymgme.2011.11.170 ◽

2012 ◽

Vol 105 (2) ◽

pp. S63

Author(s):

Brian Vuillemenot ◽

Martin Katz ◽

Joan Coates ◽

Derek Kennedy ◽

Camille Flournoy ◽

...

Keyword(s):

Disease Progression ◽

Canine Model ◽

Enzyme Replacement ◽

Ceroid Lipofuscinosis ◽

Tripeptidyl Peptidase

218. Gene Therapy and Enzyme Replacement in a Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinosis

Molecular Therapy ◽

10.1016/j.ymthe.2006.08.243 ◽

2006 ◽

Vol 13 ◽

pp. S84

Author(s):

Michael Chang ◽

David Sleat ◽

Seng Cheng ◽

Marco Passini ◽

Peter Lobel ◽

...

Keyword(s):

Gene Therapy ◽

Mouse Model ◽

Enzyme Replacement ◽

Ex vivo gene therapy for the preservation of retinal and central nervous system structure and function in a canine model of CLN2 neuronal ceroid lipofuscinosis

10.32469/10355/56993 ◽

2016 ◽

Author(s):

Christopher J. Tracy

Keyword(s):

Gene Therapy ◽

Central Nervous System ◽

Nervous System ◽

Ex Vivo ◽

Canine Model ◽

Structure And Function ◽

System Structure ◽

Ex Vivo Gene Therapy ◽

And Function

An improved, novel, systemically administered AAV gene therapy for treatment of CLN3 juvenile neuronal ceroid lipofuscinosis

10.1016/j.ymgme.2018.12.202 ◽

2019 ◽

Vol 126 (2) ◽

pp. S82-S83 ◽

Cited By ~ 1

Author(s):

Scott Kerns ◽

M. Arnold ◽

A. Aldrich ◽

R. Fallet ◽

Tammy Kielian ◽

...

Keyword(s):

Gene Therapy ◽

Juvenile Neuronal Ceroid Lipofuscinosis ◽

Intrathecal and intravenous combination gene therapy in the mouse model of infantile neuronal ceroid lipofuscinosis extends lifespan and improves behavioral outcomes in moderately affected mice

10.1016/j.ymgme.2018.12.149 ◽

2019 ◽

Vol 126 (2) ◽

pp. S63-S64

Author(s):

Steven J. Gray ◽

Alejandra Rozenberg ◽

Erik Lykken ◽

Linas Padegimas ◽

Timothy Miller

Keyword(s):

Gene Therapy ◽

Mouse Model ◽

Behavioral Outcomes ◽

224. Comparison of AAV Serotypes 2 and 5 Based Vectors for Corrective Gene Therapy for Late Infantile Neuronal Ceroid Lipofuscinosis

Molecular Therapy ◽

10.1016/s1525-0016(16)40666-0 ◽

2003 ◽

Vol 7 (5) ◽

pp. S89

Keyword(s):

Gene Therapy ◽