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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Dades bibliogràfiques
Publicat a:Blood Adv
Autors principals: 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.
Format: Artigo
Idioma:Inglês
Publicat: American Society of Hematology 2016
Matèries:
Hematopoiesis and Stem Cells
Accés en línia: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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