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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
Download Full-text
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218. Gene Therapy and Enzyme Replacement in a Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinosis
Molecular Therapy
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10.1016/j.ymthe.2006.08.243
◽
2006
◽
Vol 13
◽
pp. S84
Author(s):
Michael Chang
◽
David Sleat
◽
Seng Cheng
◽
Marco Passini
◽
Peter Lobel
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...
Keyword(s):
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Mouse Model
◽
Neuronal Ceroid Lipofuscinosis
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Infantile Neuronal Ceroid Lipofuscinosis
◽
Enzyme Replacement
◽
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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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
◽
Cited By ~ 2
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
Download Full-text
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
◽
Cited By ~ 36
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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42. AAV-mediated gene therapy causes functional improvements in a murine model of infantile neuronal ceroid lipofuscinosis (INCL, Batten Disease)
Molecular Therapy
◽
10.1016/j.ymthe.2004.05.068
◽
2004
◽
Vol 9
◽
pp. S18
Keyword(s):
Gene Therapy
◽
Murine Model
◽
Neuronal Ceroid Lipofuscinosis
◽
Batten Disease
◽
Infantile Neuronal Ceroid Lipofuscinosis
◽
Ceroid Lipofuscinosis
Download Full-text
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
◽
Human Primate
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893. Second Generation Gene Therapy Vector for Classical Late Infantile Neuronal Ceroid Lipofuscinosis
Molecular Therapy
◽
10.1016/j.ymthe.2006.08.982
◽
2006
◽
Vol 13
◽
pp. S344
Author(s):
Mario A. Cabrera-Salazar
◽
Bradley L. Hodges
◽
Nelson S. Yew
◽
Jie Bu
◽
Jonathan A. Fidler
◽
...
Keyword(s):
Gene Therapy
◽
Second Generation
◽
Neuronal Ceroid Lipofuscinosis
◽
Gene Therapy Vector
◽
Infantile Neuronal Ceroid Lipofuscinosis
◽
Ceroid Lipofuscinosis
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969. Development of Non-Invasive Imaging Strategies to Assess the Impact of Gene Therapy: Phenotypic Assessment of the Neurodegenerative CNS Disorder Late Infantile Neuronal Ceroid Lipofuscinosis Using Magnetic Resonance Spectroscopy
Molecular Therapy
◽
10.1016/j.ymthe.2005.07.512
◽
2005
◽
Vol 11
◽
pp. S374
Keyword(s):
Gene Therapy
◽
Magnetic Resonance
◽
Magnetic Resonance Spectroscopy
◽
Neuronal Ceroid Lipofuscinosis
◽
Resonance Spectroscopy
◽
Infantile Neuronal Ceroid Lipofuscinosis
◽
Non Invasive
◽
Ceroid Lipofuscinosis
◽
The Impact
Download Full-text
197. AAV2/5-Mediated Gene Therapy Synergizes with Bone Marrow Transplantation in the Treatment of Infantile Neuronal Ceroid Lipofuscinosis
Molecular Therapy
◽
10.1016/s1525-0016(16)36769-7
◽
2011
◽
Vol 19
◽
pp. S77
Keyword(s):
Gene Therapy
◽
Bone Marrow
◽
Bone Marrow Transplantation
◽
Marrow Transplantation
◽
Neuronal Ceroid Lipofuscinosis
◽
Infantile Neuronal Ceroid Lipofuscinosis
◽
Ceroid Lipofuscinosis
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824. AAV-Mediated Gene Therapy for Murine Infantile Neuronal Ceroid Lipofuscinosis (INCL, Batten Disease)
Molecular Therapy
◽
10.1016/s1525-0016(16)41266-9
◽
2003
◽
Vol 7
(5)
◽
pp. S317-S318
Keyword(s):
Gene Therapy
◽
Neuronal Ceroid Lipofuscinosis
◽
Batten Disease
◽
Infantile Neuronal Ceroid Lipofuscinosis
◽
Ceroid Lipofuscinosis
Download Full-text
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