scholarly journals 198. Adeno-Associated Viral Gene Therapy To Treat Niemann-Pick Disease, Type C1

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

Gene therapy for the treatment of Niemann-Pick disease type C1: Comparison of AAV9 to a novel serotype, AAV-PHP.B

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

scholarly journals Gene therapy in a mouse model of Niemann-Pick disease type C1

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

scholarly journals Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1

2016 ◽  
pp. ddw367 ◽  
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

ANALYSIS OF CHOLESTEROL AND OXYSTEROL LEVELS IN NIEMANN PICK DISEASE TYPE C MICE AND PATIENTS

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
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