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...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Julkaisussa:Blood Adv
Päätekijät: 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.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: American Society of Hematology 2016
Aiheet:
Hematopoiesis and Stem Cells
Linkit: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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