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Gene therapy for the treatment of Niemann-Pick disease type C1: Comparison of AAV9 to a novel serotype, AAV-PHP.B
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2017.12.075
◽
2018
◽
Vol 123
(2)
◽
pp. S36-S37
Author(s):
Cristin Davidson
◽
Alana Gibson
◽
Tansy Gu
◽
Randy Chandler
◽
Benjamin Deverman
◽
...
Keyword(s):
Gene Therapy
◽
Disease Type
◽
Niemann Pick Disease
◽
Niemann Pick
◽
Pick Disease
Download Full-text
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Cited By
References
Faculty Opinions recommendation of Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1.
Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
◽
10.3410/f.726883447.793542559
◽
2018
◽
Author(s):
Barbara Borroni
Keyword(s):
Gene Therapy
◽
Disease Type
◽
Niemann Pick Disease
◽
Niemann Pick
◽
Pick Disease
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198. Adeno-Associated Viral Gene Therapy To Treat Niemann-Pick Disease, Type C1
Molecular Therapy
◽
10.1016/s1525-0016(16)33803-5
◽
2015
◽
Vol 23
◽
pp. S79
Author(s):
Randy J. Chandler
◽
Ian M. Williams
◽
Arturo A. Incao
◽
Forbes D. Porter
◽
William J. Pavan
◽
...
Keyword(s):
Gene Therapy
◽
Disease Type
◽
Viral Gene
◽
Niemann Pick Disease
◽
Viral Gene Therapy
◽
Niemann Pick
◽
Pick Disease
Download Full-text
Gene therapy in a mouse model of Niemann-Pick disease type C1
Human Gene Therapy
◽
10.1089/hum.2020.175
◽
2020
◽
Author(s):
Yoshie Kurokawa
◽
Hitoshi Osaka
◽
Takeshi Kouga
◽
Eriko Jimbo
◽
Kazuhiro Muramatsu
◽
...
Keyword(s):
Gene Therapy
◽
Mouse Model
◽
Disease Type
◽
Niemann Pick Disease
◽
Niemann Pick
◽
Pick Disease
Download Full-text
Gene Therapy Moves Forward for Niemann–Pick Disease Type C1
Human Gene Therapy
◽
10.1089/hum.2021.29163.rsm
◽
2021
◽
Vol 32
(11-12)
◽
pp. 540-540
Author(s):
R. Scott McIvor
Keyword(s):
Gene Therapy
◽
Disease Type
◽
Niemann Pick Disease
◽
Niemann Pick
◽
Pick Disease
Download Full-text
Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1
Human Molecular Genetics
◽
10.1093/hmg/ddw367
◽
2016
◽
pp. ddw367
◽
Cited By ~ 13
Author(s):
Randy J. Chandler
◽
Ian M. Williams
◽
Alana L. Gibson
◽
Cristin D. Davidson
◽
Arturo A. Incao
◽
...
Keyword(s):
Gene Therapy
◽
Disease Type
◽
Niemann Pick Disease
◽
Niemann Pick
◽
Pick Disease
Download Full-text
Niemann-Pick Disease Type C Quality-of-Life Scale for Adults
PsycTESTS Dataset
◽
10.1037/t75153-000
◽
2019
◽
Author(s):
Lydia Aston
◽
Rachel Shaw
◽
Rebecca Knibb
Keyword(s):
Quality Of Life
◽
Disease Type
◽
Niemann Pick Disease
◽
Quality Of Life Scale
◽
Life Scale
◽
Type C
◽
Niemann Pick
◽
Pick Disease
Download Full-text
ANALYSIS OF CHOLESTEROL AND OXYSTEROL LEVELS IN NIEMANN PICK DISEASE TYPE C MICE AND PATIENTS
Neuropediatrics
◽
10.1055/s-2006-945720
◽
2006
◽
Vol 37
(S 1)
◽
Author(s):
S Tay
◽
X He
◽
AM Jenner
◽
BS Wong
◽
WY Ong
Keyword(s):
Disease Type
◽
Niemann Pick Disease
◽
Type C
◽
Niemann Pick
◽
Pick Disease
Download Full-text
Orphan designation: Hydroxy-propyl-beta-cyclodextrin, Treatment of Niemann-Pick disease, type C
Case Medical Research
◽
10.31525/cmr-399673
◽
2019
◽
Author(s):
Keyword(s):
Disease Type
◽
Orphan Designation
◽
Beta Cyclodextrin
◽
Niemann Pick Disease
◽
Type C
◽
Niemann Pick
◽
Pick Disease
Download Full-text
Orphan designation: miglustat, Treatment of Niemann-Pick disease, type C
Case Medical Research
◽
10.31525/cmr-bc1108
◽
2019
◽
Author(s):
Keyword(s):
Disease Type
◽
Orphan Designation
◽
Niemann Pick Disease
◽
Type C
◽
Niemann Pick
◽
Pick Disease
Download Full-text
Faculty Opinions recommendation of The adult form of Niemann-Pick disease type C.
Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
◽
10.3410/f.1085930.538703
◽
2007
◽
Author(s):
Peter Brown
Keyword(s):
Disease Type
◽
Adult Form
◽
Niemann Pick Disease
◽
Type C
◽
Niemann Pick
◽
Pick Disease
Download Full-text
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