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

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 ◽

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 ◽

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 ◽

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 ◽

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 ◽

Ceroid Lipofuscinosis ◽

Primate Brain ◽

Therapy Treatment ◽

Human Primate

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 ◽

Gene Therapy Vector ◽

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 ◽

Resonance Spectroscopy ◽

Non Invasive ◽

Ceroid Lipofuscinosis ◽

The Impact

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 ◽

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 ◽

Batten Disease ◽