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Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair
X-linked chronic granulomatous disease is an immunodeficiency characterized by defective production of microbicidal reactive oxygen species (ROS) by phagocytes. Causative mutations occur throughout the 13 exons and splice sites of the CYBB gene, resulting in loss of gp91(phox) protein. Here we repor...
Αποθηκεύτηκε σε:
| Τόπος έκδοσης: | Gene Ther |
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| Κύριοι συγγραφείς: | , , , , , , , , , , , , , , , |
| Μορφή: | Artigo |
| Γλώσσα: | Inglês |
| Έκδοση: |
2021
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| Θέματα: | |
| Διαθέσιμο Online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC8232036/ https://ncbi.nlm.nih.gov/pubmed/33712802 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41434-021-00251-z |
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