Allele-specific silencing of the dominant disease allele in sialuria by RNA interference

Dominant disease alleles are attractive therapeutic targets for allele-specific gene silencing by small interfering RNA (siRNA). Sialuria is a dominant disorder caused by missense mutations in the allosteric site of GNE, coding for the rate-limiting enzyme of sialic acid biosynthesis, UDP-GlcNAc 2-e...

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Main Authors: Klootwijk, Riko D., Savelkoul, Paul J. M., Ciccone, Carla, Manoli, Irini, Caplen, Natasha J., Krasnewich, Donna M., Gahl, William A., Huizing, Marjan
Formáid: Artigo
Teanga:Inglês
Foilsithe: The Federation of American Societies for Experimental Biology 2008
Ábhair:
Research Communications
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC2574030/
https://ncbi.nlm.nih.gov/pubmed/18653764
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1096/fj.08-110890
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