decarboxylase deficiency
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2021 ◽  
Vol 21 ◽  
Author(s):  
Wuh-Liang Hwu ◽  
Shin-Ichi Muramatsu ◽  
Bruria Gidoni-Ben-Zeev

: Preexisting immunity to adeno-associated virus (AAV) poses a concern in AAV vector–mediated gene therapy. Localized administration of low doses of carefully chosen AAV serotypes can mitigate the risk of an immune response. This article will illustrate the low risk of immune response to AAV serotype 2 vector–mediated gene therapy to the brain with support from clinical trial data in aromatic L-amino acid decarboxylase deficiency and Parkinson disease.


Author(s):  
Yuiko Hasegawa ◽  
Eriko Nishi ◽  
Yuko Mishima ◽  
Tomohiro Sakaguchi ◽  
Futoshi Sekiguchi ◽  
...  

2021 ◽  
Vol 24 ◽  
pp. S206-S207
Author(s):  
K. Williams ◽  
H. Skrobanski ◽  
C. Werner ◽  
S. O’Neill ◽  
K. Buesch ◽  
...  

2021 ◽  
Vol 27 ◽  
pp. 100744
Author(s):  
Francyne Kubaski ◽  
Zackary M. Herbst ◽  
Danilo A.A. Pereira ◽  
Camilo Silva ◽  
Christine Chen ◽  
...  

2021 ◽  
Vol 24 ◽  
pp. S206
Author(s):  
H. Skrobanski ◽  
K. Williams ◽  
C. Werner ◽  
S. O’Neill ◽  
K. Buesch ◽  
...  

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