In Utero Gene Therapy Treatment of Neuronal Ceroid Lipofuscinosis Type 1 Disease Improves Lifespan and Neurologic Function

Journal of the American College of Surgeons ◽

10.1016/j.jamcollsurg.2021.07.377 ◽

2021 ◽

Vol 233 (5) ◽

pp. S186

Author(s):

Matthew T. Grant ◽

Joshua Dearborn ◽

Mark Sands ◽

Jesse D. Vrecenak

Keyword(s):

Gene Therapy ◽

Neuronal Ceroid Lipofuscinosis ◽

In Utero ◽

Ceroid Lipofuscinosis ◽

Neurologic Function ◽

Therapy Treatment

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1060. Long Term Expression of TPP-I in Rat and Non-Human Primate Brain Following Administration of AAV2CUhCLN2, a Candidate Gene Therapy Treatment for Late Infantile Neuronal Ceroid Lipofuscinosis

Molecular Therapy ◽

10.1016/j.ymthe.2004.06.1005 ◽

2004 ◽

Vol 9 ◽

pp. S406-S407

Keyword(s):

Gene Therapy ◽

Candidate Gene ◽

Neuronal Ceroid Lipofuscinosis ◽

Infantile Neuronal Ceroid Lipofuscinosis ◽

Ceroid Lipofuscinosis ◽

Primate Brain ◽

Therapy Treatment ◽

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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 ◽

Neuronal Ceroid Lipofuscinosis ◽

Infantile Neuronal Ceroid Lipofuscinosis ◽

Enzyme Replacement ◽

Ceroid Lipofuscinosis

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Uniparental disomy of chromosome 1 unmasks recessive mutations of PPT1 in a boy with neuronal ceroid lipofuscinosis type 1

Brain and Development ◽

10.1016/j.braindev.2016.08.010 ◽

2017 ◽

Vol 39 (2) ◽

pp. 182-183 ◽

Author(s):

Lorena Travaglini ◽

Chiara Aiello ◽

Viola Alesi ◽

Sara Loddo ◽

Antonio Novelli ◽

...

Keyword(s):

Neuronal Ceroid Lipofuscinosis ◽

Uniparental Disomy ◽

Chromosome 1 ◽

Ceroid Lipofuscinosis ◽

Recessive Mutations

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

Gene Therapy ◽

10.1038/gt.2017.4 ◽

2017 ◽

Vol 24 (4) ◽

pp. 215-223 ◽

Author(s):

M L Katz ◽

G C Johnson ◽

S B Leach ◽

B G Williamson ◽

J R Coates ◽

...

Keyword(s):

Gene Therapy ◽

Disease Progression ◽

Neurological Disease ◽

Neuronal Ceroid Lipofuscinosis ◽

Canine Model ◽

Ceroid Lipofuscinosis

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

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2018.12.202 ◽

2019 ◽

Vol 126 (2) ◽

pp. S82-S83 ◽

Author(s):

Scott Kerns ◽

M. Arnold ◽

A. Aldrich ◽

R. Fallet ◽

Tammy Kielian ◽

...

Keyword(s):

Gene Therapy ◽

Neuronal Ceroid Lipofuscinosis ◽

Juvenile Neuronal Ceroid Lipofuscinosis ◽

Ceroid Lipofuscinosis

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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

Molecular Genetics and Metabolism ◽

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 ◽

Neuronal Ceroid Lipofuscinosis ◽

Behavioral Outcomes ◽

Infantile Neuronal Ceroid Lipofuscinosis ◽

Ceroid Lipofuscinosis

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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 ◽

Neuronal Ceroid Lipofuscinosis ◽

Infantile Neuronal Ceroid Lipofuscinosis ◽

Ceroid Lipofuscinosis

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Intravenous administration of CLN3 gene therapy for juvenile neuronal ceroid lipofuscinosis

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2017.12.194 ◽

2018 ◽

Vol 123 (2) ◽

pp. S77 ◽

Author(s):

Scott Kerns ◽

J. Daum ◽

M. Bosch ◽

R. Fallet ◽

J. Odvody ◽

...

Keyword(s):

Gene Therapy ◽

Intravenous Administration ◽

Neuronal Ceroid Lipofuscinosis ◽

Juvenile Neuronal Ceroid Lipofuscinosis ◽

Ceroid Lipofuscinosis

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Combination dosing of CLN1 gene therapy extends lifespan in a mouse model of infantile neuronal ceroid lipofuscinosis

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2017.12.338 ◽

2018 ◽

Vol 123 (2) ◽

pp. S124 ◽

Author(s):

Alejandra J. Rozenberg ◽

Erik Lykken ◽

Kaye Spratt ◽

Timothy J. Miller ◽

Steven J. Gray

Keyword(s):

Gene Therapy ◽

Mouse Model ◽

Neuronal Ceroid Lipofuscinosis ◽

Infantile Neuronal Ceroid Lipofuscinosis ◽

Ceroid Lipofuscinosis

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Combination small molecule PPT1 mimetic and CNS-directed gene therapy as a treatment for infantile neuronal ceroid lipofuscinosis

Journal of Inherited Metabolic Disease ◽

10.1007/s10545-011-9446-x ◽

2012 ◽

Vol 35 (5) ◽

pp. 847-857 ◽

Author(s):

Marie S. Roberts ◽

Shannon L. Macauley ◽

Andrew M. Wong ◽

Denis Yilmas ◽

Sarah Hohm ◽

...

Keyword(s):

Gene Therapy ◽

Small Molecule ◽

Neuronal Ceroid Lipofuscinosis ◽

Infantile Neuronal Ceroid Lipofuscinosis ◽

Ceroid Lipofuscinosis

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