AAV9/MFSD8 gene therapy is effective in preclinical models of neuronal ceroid lipofuscinosis type 7 disease

Preclinical Models ◽

Ceroid Lipofuscinosis ◽

Mfsd8 Gene

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 ◽

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 ◽

Cited By ~ 22

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 ◽

Canine Model ◽

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 ◽

Intravenous administration of CLN3 gene therapy for juvenile neuronal ceroid lipofuscinosis

10.1016/j.ymgme.2017.12.194 ◽

2018 ◽

Vol 123 (2) ◽

pp. S77 ◽

Cited By ~ 1

Author(s):

Scott Kerns ◽

J. Daum ◽

M. Bosch ◽

R. Fallet ◽

J. Odvody ◽

...

Keyword(s):

Gene Therapy ◽

Intravenous Administration ◽

Juvenile Neuronal Ceroid Lipofuscinosis ◽

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

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 ◽

Novel in-frame deletion in MFSD8 gene revealed by trio whole exome sequencing in an Iranian affected with neuronal ceroid lipofuscinosis type 7: a case report

Journal of Medical Case Reports ◽

10.1186/s13256-018-1788-7 ◽

2018 ◽

Vol 12 (1) ◽

Cited By ~ 3

Author(s):

Ali Hosseini Bereshneh ◽

Masoud Garshasbi

Keyword(s):

Case Report ◽

Exome Sequencing ◽

Whole Exome Sequencing ◽

Ceroid Lipofuscinosis ◽

Whole Exome ◽

Mfsd8 Gene

A novel mutation in the MFSD8 gene in late infantile neuronal ceroid lipofuscinosis

Neurogenetics ◽

10.1007/s10048-008-0153-1 ◽

2008 ◽

Vol 10 (1) ◽

pp. 73-77 ◽

Cited By ~ 16

Author(s):

E. Stogmann ◽

S. El Tawil ◽

J. Wagenstaller ◽

A. Gaber ◽

S. Edris ◽

...

Keyword(s):

Novel Mutation ◽

Ceroid Lipofuscinosis ◽

Mfsd8 Gene

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 ◽