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Nonsense suppressor therapies rescue peroxisome lipid metabolism and assembly in cells from patients with specific PEX gene mutations

Peroxisome biogenesis disorders (PBDs) are multisystemic autosomal recessive disorders resulting from mutations in PEX genes required for normal peroxisome assembly and metabolic activities. Here, we evaluated the potential effectiveness of aminoglycoside G418 (geneticin) and PTC124 (ataluren) nonse...

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Detaylı Bibliyografya
Asıl Yazarlar: Dranchak, Patricia K., Di Pietro, Erminia, Snowden, Ann, Oesch, Nathan, Braverman, Nancy E., Steinberg, Steven J., Hacia, Joseph G.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: 2011
Konular:
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC3136445/
https://ncbi.nlm.nih.gov/pubmed/21465523
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jcb.22979
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