Self-Complementary AAV9 Gene Delivery Partially Corrects Pathology Associated with Juvenile Neuronal Ceroid Lipofuscinosis (CLN3)

Juvenile neuronal ceroid lipofuscinosis (JNCL) is a fatal lysosomal storage disease caused by autosomal-recessive mutations in CLN3 for which no treatment exists. Symptoms appear between 5 and 10 years of age, beginning with blindness and seizures, followed by progressive cognitive and motor decline...

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發表在:J Neurosci
Main Authors: Bosch, Megan E., Aldrich, Amy, Fallet, Rachel, Odvody, Jessica, Burkovetskaya, Maria, Schuberth, Kaitlyn, Fitzgerald, Julie A., Foust, Kevin D., Kielian, Tammy
格式: Artigo
語言:Inglês
出版: Society for Neuroscience 2016
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Articles
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC6601943/
https://ncbi.nlm.nih.gov/pubmed/27629717
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1523/JNEUROSCI.1635-16.2016
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