Adeno-associated virus 2-mediated gene therapy decreases autofluorescent storage material and increases brain mass in a murine model of infantile neuronal ceroid lipofuscinosis

Storage Material ◽

Adeno Associated Virus ◽

Brain Mass ◽

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 ◽

Batten Disease ◽

CNS-directed AAV2-mediated gene therapy ameliorates functional deficits in a murine model of infantile neuronal ceroid lipofuscinosis

Molecular Therapy ◽

10.1016/j.ymthe.2005.11.008 ◽

2006 ◽

Vol 13 (3) ◽

pp. 538-547 ◽

Cited By ~ 90

Author(s):

Megan A. Griffey ◽

David Wozniak ◽

Michael Wong ◽

Ellen Bible ◽

Kendra Johnson ◽

...

Keyword(s):

Gene Therapy ◽

Murine Model ◽

Functional Deficits ◽

A murine model of infantile neuronal ceroid lipofuscinosis - Ultrastructural evaluation of storage in the central nervous system and viscera

Pediatric and Developmental Pathology ◽

10.2350/07-03-0242 ◽

2006 ◽

Vol preprint (2007) ◽

pp. 1

Author(s):

Nancy Galvin ◽

Carole Vogler ◽

Beth Levy ◽

Attila Kovacs ◽

Megan Griffey ◽

...

Keyword(s):

Central Nervous System ◽

Nervous System ◽

Murine Model ◽

Ceroid Lipofuscinosis ◽

The Central Nervous System

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 ◽

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 ◽

Behavioral Outcomes ◽

Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis by CNS Administration of a Serotype 2 Adeno-Associated Virus Expressing CLN2 cDNA

Human Gene Therapy ◽

10.1089/hum.2008.022 ◽

2008 ◽

Vol 19 (5) ◽

pp. 463-474 ◽

Cited By ~ 277

Author(s):

Stefan Worgall ◽

Dolan Sondhi ◽

Neil R. Hackett ◽

Barry Kosofsky ◽

Minal V. Kekatpure ◽

...

Keyword(s):

Adeno Associated Virus ◽

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 ◽

Altered glutamate receptor function in the cerebellum of the Ppt1−/− mouse, a murine model of infantile neuronal ceroid lipofuscinosis

Journal of Neuroscience Research ◽

10.1002/jnr.22763 ◽

2011 ◽

Vol 90 (2) ◽

pp. 367-375 ◽

Cited By ~ 14

Author(s):

Rozzy Finn ◽

Attila D. Kovács ◽

David A. Pearce

Keyword(s):

Glutamate Receptor ◽

Murine Model ◽

Receptor Function ◽

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 ◽

Infantile neuronal ceroid lipofuscinosis: Isolation of storage material

Neurology ◽

10.1212/wnl.32.9.1035 ◽

1982 ◽

Vol 32 (9) ◽

pp. 1035-1035 ◽

Cited By ~ 14

Author(s):

J. Palo ◽

I. Elovaara ◽

M. Haltia ◽

N. M. K. Ng Ying Kin ◽

L. S. Wolfe

Keyword(s):

Storage Material ◽