Publications by authors named "Scott Clarkson"
A hallmark of chronic hepatitis B (CHB) virus infection is the presence of high circulating levels of non-infectious small lipid HBV surface antigen (HBsAg) vesicles. Although rare, sustained HBsAg loss is the idealized endpoint of any CHB therapy. A small molecule, RG7834, has been previously reported to inhibit HBsAg expression by targeting terminal nucleotidyltransferase proteins 4A and 4B (TENT4A and TENT4B).
View Article and Find Full Text PDFCRISPR/Cas9 mediated gene editing of patient-derived hematopoietic stem and progenitor cells (HSPCs) ex vivo followed by autologous transplantation of the edited HSPCs back to the patient can provide a potential cure for monogenic blood disorders such as β-hemoglobinopathies. One challenge for this strategy is efficient delivery of the ribonucleoprotein (RNP) complex, consisting of purified Cas9 protein and guide RNA, into HSPCs. Because β-hemoglobinopathies are most prevalent in developing countries, it is desirable to have a reliable, efficient, easy-to-use and cost effective delivery method.
View Article and Find Full Text PDFArticle Synopsis
- CRISPR-Cas9 screening helps researchers study gene functions, but traditional methods struggle with human primary cells due to the need for stable Cas9 expression.
- The Guide Swap technique overcomes this limitation by allowing efficient genome-scale screening in human primary cells using targeted guide RNAs (gRNAs) and nontargeting gRNAs.
- By validating Guide Swap in CD4 T cells and hematopoiesis models, the researchers discovered new regulators of hematopoietic stem cell expansion and believe this method could be applied to other complex cell types for further biological discoveries.