Publications by authors named "Kah Mun Siow"
Article Synopsis
- CRISPR genome editing in disorders like p47-deficient chronic granulomatous disease (CGD) faces challenges due to chromosomal rearrangements caused by multiple similar gene targets on the same chromosome.
- Research identified that interactions between homologous gene sequences led to significant rearrangements after editing the NCF1 gene and its related pseudogenes in human cell models.
- The study emphasized the importance of understanding the genomic context where editing occurs, as the presence of homologous regions can increase the risk of unintended chromosomal changes during the editing process.
p47 -deficient chronic granulomatous disease (p47-CGD) is a primary immunodeficiency caused by mutations in the neutrophil cytosolic factor 1 () gene, resulting in defective NADPH oxidase function in phagocytes. Due to its complex genomic context, the locus is not suited for safe gene editing with current genome editing technologies. Therefore, we developed a targeted coding sequence knock-in by CRISPR-Cas9 ribonucleoprotein and viral vector template delivery, to restore p47 expression under the control of the endogenous locus.
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