Scott Clarkson

A potent epigenetic editor targeting human PCSK9 for durable reduction of low-density lipoprotein cholesterol levels

Frédéric Tremblay, Qiang Xiong, Chih‐Wei Ko, Kenneth A. Kelly et autres

Epigenetic editing holds the promise of durable therapeutic effects by silencing disease-causing genes without changing the underlying DNA sequence. In this study, we designed an epigenetic editor to target human PCSK9 and thereby induce DNA methylation at this locus. A single administration …

us, it (code pays fourni par la source)

OS-090 Novel epigenetic editing technology targets hepatitis B virus in vivo to deeply and durably repress viral markers

Yesseinia I. Angleró-Rodriguez, Sahar Abubucker, John Xiong, Jhon Medina et autres

Durable Multiplex Epigenetic Editing for Generation of Allogeneic CAR T without Chromosomal Rearrangements

Jamie L. Schafer, Justin D. Trombley, Benjamin Hallisey, Kunza Ahmad et autres

CAR T therapies have provided significant breakthroughs for patients with CD19+ malignancies and multiple myeloma, but a major limitation is the need for individualized manufacturing of autologous T cells, increasing the cost and reducing the availability of these life-saving drugs. An allogeneic …

A Genome-wide CRISPR Screen Identifies ZCCHC14 as a Host Factor Required for Hepatitis B Surface Antigen Production

Anastasia Hyrina, Christopher T. Jones, Darlene Chen, Scott Clarkson et autres

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, …

cn, us (code pays fourni par la source)

In-Depth Characterization and Validation in BRG1 -Mutant Lung Cancers Define Novel Catalytic Inhibitors of SWI/SNF Chromatin Remodeling

Zainab Jagani, Gregg Chenail, Kay Xiaoqin Xiang, Geoffrey Bushold et autres

Abstract Members of the ATP-dependent SWI/SNF chromatin remodeling complexes are among the most frequently mutated genes in cancer, suggesting their dysregulation plays a critical role. The synthetic lethality between SWI/SNF catalytic subunits BRM/SMARCA2 and BRG1/SMARCA4 has instigated great interest in targeting BRM. …

us, cn (code pays fourni par la source)

A genome-wide CRISPR screen identifies ZCCHC14 as a host factor required for hepatitis B surface antigen production

Anastasia Hyrina, Christopher T. Jones, Darlene Chen, Scott Clarkson et autres

GRAPHICAL ABSTRACT SUMMARY A hallmark of chronic hepatitis B virus (CHB) 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 …

us (code pays fourni par la source)

A Meta-study on the Thermal Properties of Carbon Nanotubes in Thermal Interface Materials

Scott Clarkson, Asah H Khan, Dipendra Singh

Computer Integrated Circuit (IC) microprocessors are becoming more powerful and densely packed while cooling mechanisms are seeing an equivalent improvement to compensate. A significant limit to cooling performance is thermal transfer between die and heatsink. In this meta study we evaluate carbon …

au (code pays fourni par la source)

TRIAMF: A New Method for Delivery of Cas9 Ribonucleoprotein Complex to Human Hematopoietic Stem Cells

Jonathan S. Yen, Michael Fiorino, Yi Sha Liu, Steve Paula et autres

CRISPR/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 …

us, ch, cn (code pays fourni par la source)

Guide Swap enables genome-scale pooled CRISPR–Cas9 screening in human primary cells

Pamela Y. Ting, Albert E. Parker, J. Scott Lee, Chris Trussell et autres

us (code pays fourni par la source)

CRISPR/Cas9 Gene-Edited Hematopoietic Stem Cell Therapy for Sickle Cell Disease

Vionnie W.C. Yu, Yi Liu, Matthew S. Curran, Pu Zhang et autres

Abstract Sickle cell disease (SCD) is a life-threatening hereditary disorder that affects thirty million individuals worldwide. The disease results from a single amino acid change in the β-globin gene, which causes polymerization of hemoglobin and deformation of red blood cells, leading to …

ch, cn, us, fr (code pays fourni par la source)