scholarly journals 611. Promoter Evaluation of AAV Gene Therapy in the Central Nervous System for Feline Niemann-Pick Type C Disease

2016 ◽  
Vol 24 ◽  
pp. S242
Author(s):  
Brittney L. Gurda ◽  
Gary Swain ◽  
Jessica H. Bagel ◽  
Maria Prociuk ◽  
Steven J. Gray ◽  
...  
2012 ◽  
Vol 2012 ◽  
pp. 1-10 ◽  
Author(s):  
Jerusha Naidoo ◽  
Deborah Young

Substantial progress has been made in the development of novel gene therapy strategies for central nervous system (CNS) disorders in recent years. However, unregulated transgene expression is a significant issue limiting human applications due to the potential side effects from excessive levels of transgenic protein that indiscriminately affect both diseased and nondiseased cells. Gene regulation systems are a tool by which tight tissue-specific and temporal regulation of transgene expression may be achieved. This review covers the features of ideal regulatory systems and summarises the mechanics of current exogenous and endogenous gene regulation systems and their utility in the CNS.


2000 ◽  
Vol 107 (2) ◽  
pp. 184-190 ◽  
Author(s):  
Gianvito Martino ◽  
Pietro L Poliani ◽  
Roberto Furlan ◽  
Peggy Marconi ◽  
Joseph C Glorioso ◽  
...  

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