Reprogramming erythroid cells for lysosomal enzyme production leads to visceral and CNS cross-correction in mice with Hurler syndrome

Restricting transgene expression to maturing erythroid cells can reduce the risk for activating oncogenes in hematopoietic stem cells (HSCs) and their progeny, yet take advantage of their robust protein synthesis machinery for high-level protein production. This study sought to evaluate the feasibil...

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Detaylı Bibliyografya
Asıl Yazarlar: Wang, Daren, Zhang, Wei, Kalfa, Theodosia A., Grabowski, Gregory, Davies, Stella, Malik, Punam, Pan, Dao
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: National Academy of Sciences 2009
Konular:
Biological Sciences
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC2785274/
https://ncbi.nlm.nih.gov/pubmed/19903883
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0908528106
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