Gene-edited pseudogene resurrection corrects p47(phox)-deficient chronic granulomatous disease

Pseudogenes are duplicated genes with mutations rendering them nonfunctional. For single-gene disorders with homologous pseudogenes, the pseudogene might be a target for genetic correction. Autosomal-recessive p47(phox)-deficient chronic granulomatous disease (p47-CGD) is a life-threatening immune d...

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發表在:Blood Adv
Main Authors: Merling, Randall K., Kuhns, Douglas B., Sweeney, Colin L., Wu, Xiaolin, Burkett, Sandra, Chu, Jessica, Lee, Janet, Koontz, Sherry, Di Pasquale, Giovanni, Afione, Sandra A., Chiorini, John A., Kang, Elizabeth M., Choi, Uimook, De Ravin, Suk See, Malech, Harry L.
格式: Artigo
語言:Inglês
出版: American Society of Hematology 2016
主題:
Hematopoiesis and Stem Cells
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC5727772/
https://ncbi.nlm.nih.gov/pubmed/29296942
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/bloodadvances.2016001214
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