scholarly journals AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington’s disease

Gene Therapy ◽  
2017 ◽  
Vol 24 (10) ◽  
pp. 630-639 ◽  
Author(s):  
J Miniarikova ◽  
V Zimmer ◽  
R Martier ◽  
C C Brouwers ◽  
C Pythoud ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Huntington's Disease ◽  
Huntington’S Disease ◽  
Rat Model ◽  
Neuronal Dysfunction ◽  
Mutant Huntingtin

scholarly journals AAV1/2-mediated BDNF gene therapy in a transgenic rat model of Huntington’s disease

Gene Therapy ◽  
2015 ◽  
Vol 23 (3) ◽  
pp. 283-295 ◽  
Author(s):  
B Connor ◽  
Y Sun ◽  
D von Hieber ◽  
S K Tang ◽  
K S Jones ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Huntington's Disease ◽  
Huntington’S Disease ◽  
Rat Model ◽  
Transgenic Rat ◽  
Bdnf Gene ◽  
Transgenic Rat Model

Neurturin gene therapy improves motor function and prevents death of striatal neurons in a 3-nitropropionic acid rat model of Huntington's disease

2007 ◽  
Vol 26 (2) ◽  
pp. 375-384 ◽  
Author(s):  
Shilpa Ramaswamy ◽  
Jodi L. McBride ◽  
Christopher D. Herzog ◽  
Eugene Brandon ◽  
Mehdi Gasmi ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Huntington's Disease ◽  
Huntington’S Disease ◽  
Motor Function ◽  
Rat Model ◽  
Striatal Neurons ◽  
Nitropropionic Acid ◽  
3 Nitropropionic Acid

scholarly journals AAV Vector–mediated RNAi of Mutant Huntingtin Expression Is Neuroprotective in a Novel Genetic Rat Model of Huntington's Disease

2008 ◽  
Vol 16 (5) ◽  
pp. 947-956 ◽  
Author(s):  
Nicholas R Franich ◽  
Helen L Fitzsimons ◽  
Dahna M Fong ◽  
Matthias Klugmann ◽  
Matthew J During ◽  
...  
Keyword(s):  
Huntington's Disease ◽  
Huntington’S Disease ◽  
Rat Model ◽  
Mutant Huntingtin ◽  
Aav Vector

scholarly journals Nucleic Acid-Based Therapy Approaches for Huntington's Disease

2012 ◽  
Vol 2012 ◽  
pp. 1-9 ◽  
Author(s):  
Tatyana Vagner ◽  
Deborah Young ◽  
Alexandre Mouravlev
Keyword(s):  
Gene Therapy ◽  
Nucleic Acid ◽  
Huntington's Disease ◽  
Huntington’S Disease ◽  
Clinical Symptoms ◽  
Cell Metabolism ◽  
Therapeutic Strategies ◽  
Cag Repeat ◽  
Mutant Huntingtin ◽  
Dominant Mutation

Huntington's disease (HD) is caused by a dominant mutation that results in an unstable expansion of a CAG repeat in the huntingtin gene leading to a toxic gain of function in huntingtin protein which causes massive neurodegeneration mainly in the striatum and clinical symptoms associated with the disease. Since the mutation has multiple effects in the cell and the precise mechanism of the disease remains to be elucidated, gene therapy approaches have been developed that intervene in different aspects of the condition. These approaches include increasing expression of growth factors, decreasing levels of mutant huntingtin, and restoring cell metabolism and transcriptional balance. The aim of this paper is to outline the nucleic acid-based therapeutic strategies that have been tested to date.

scholarly journals AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington’s Disease Minipig Model

2018 ◽  
Vol 26 (9) ◽  
pp. 2163-2177 ◽  
Author(s):  
Melvin M. Evers ◽  
Jana Miniarikova ◽  
Stefan Juhas ◽  
Astrid Vallès ◽  
Bozena Bohuslavova ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Huntington's Disease ◽  
Huntington’S Disease ◽  
Mutant Huntingtin ◽  
Broad Distribution
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