Search Results

Therapeutic adenine base editing of human hematopoietic stem cells

In ß-thalassemia, either -globin induction to form fetal hemoglobin (a22) or ß-globin repair to restore adult hemoglobin (a2ß2) could be therapeutic. ABE8e, a recently evolved adenine base editor variant, can achieve efficient adenine conversion, yet...

Authors: Liao J, Chen S, Hsiao S, Jiang Y, Yang Y, Zhang Y, Wang X, Lai Y, Bauer DE, Wu Y

SST Peer Reviewed

Naturally occurring T cell mutations enhance engineered T cell therapies

Adoptive T cell therapies have produced exceptional responses in a subset of patients with cancer. However, therapeutic efficacy can be hindered by poor T cell persistence and function1. In human T cell cancers, evolution of the disease positively...

Authors: Garcia J, Daniels J, Lee Y, Zhu I, Cheng K, Liu Q, Goodman D, Burnett C, Law C, Thienpont C, Alavi...

SST Peer Reviewed

Improved cytosine base editors generated from TadA variants

Cytosine base editors (CBEs) enable programmable genomic C·G-to-T·A transition mutations and typically comprise a modifed CRISPR–Cas enzyme, a naturally occurring cytidine deaminase, and an inhibitor of uracil repair. Previous studies have shown that...

Authors: Lam DK, Feliciano PR, Arif A, Bohnuud T, Fernandez TP, Gehrke JM, Grayson P, Lee KD, Ortega MA,...

SST Peer Reviewed

Highly scalable arrayed CRISPR mediated gene silencing in primary lungsmall airway epithelial cells

Small airway epithelial cells (SAECs) play a central role in the pathogenesis of lung diseases and are now becoming a crucial cellular model for target identification and validation in drug discovery. However, primary cell lines such as SAECs are...

Authors: Dickson A, Mullooly N, Serrano A, Escudero-Ibarz L, Wiggins C, Gianni D

SST Peer Reviewed

Molecular and Physiological Effects of Browning Agents on White Adipocytes from Bone Marrow Mesenchymal Stromal Cells

Two different types of adipose depots can be observed in mammals: white adipose tissue (WAT) and brown adipose tissue (BAT). The primary role of WAT is to deposit surplus energy in the form of triglycerides, along with many metabolic and hormonal...

Authors: Girolamo Di Maio, Nicola Alessio, Gianfranco Peluso, Silverio Perrotta, Marcellino Monda, Giovanni...

Neoantigen-targeted CD8+ T cell responses with PD-1 blockade therapy

Neoantigens are peptides derived from non-synonymous mutations presented by human leukocyte antigens (HLAs), which are recognized by antitumour T cells1-14. The large HLA allele diversity and limiting clinical samples have restricted the study of the...

Authors: Cristina Puig-Saus 1 2 3 4, Barbara Sennino 5, Songming Peng 5, Clifford L Wang 5, Zheng...

SST Peer Reviewed

Protocol to create phenotypic primary human hepatocyte cultures using the RASTRUM 3D cell model platform

To improve human hepatotoxicity prediction, in vitro liver cell models replicating hepatocyte function, drug metabolism, and toxicity are required. Here, we present a protocol for creating 3D primary human hepatocyte (PHH) cell models using the...

Authors: Christine Yee , Yik Lung Chan , Robert Utama , Marie Besnier , Martin Engel , Lisa Belfiore

Ultra-deep sequencing validates safety of CRISPR/Cas9 genome editing in human hematopoietic stem and progenitor cells

As CRISPR-based therapies enter the clinic, evaluation of safety remains a critical and active area of study. Here, we employ a clinical next generation sequencing (NGS) workflow to achieve high sequencing depth and detect ultra-low frequency...

Authors: Cromer MK, Barsan VV, Jaeger E, Wang M, Hampton JP, Chen F, Kennedy D, Xiao J, Khrebtukova I, Granat...

SST Peer Reviewed

A High-Throughput Microphysiological Liver Chip System to Model Drug-Induced Liver Injury Using Human Liver Organoids

Background and AimsDrug-induced liver injury (DILI) is a major failure mode in pharmaceutical development. This study aims to address the limitations of existing preclinical models by assessing a high-throughput, microfluidic liver-on-a-chip system,...

Authors: Sophia R. Meyer , Charles J. Zhang , Max A. Garcia, Megan C. Procario , Sanghee Yoo , Amber...

Programmable C:G to G:C genome editing with CRISPR-Cas9-directed base excision repair proteins

Many genetic diseases are caused by single-nucleotide polymorphisms. Base editors can correct these mutations at single-nucleotide resolution, but until recently, only allowed for transition edits, addressing four out of twelve possible DNA base...

Authors: Chen L, Park JE, Paa P, Rajakumar PD, Prekop HT, Chew YT, Manivannan SN, Chew WL.

SST Peer Reviewed

Data Type

Category

Research Area

Platform

Topic

Species

Characteristic

Format

Origin

Therapeutic adenine base editing of human hematopoietic stem cells

Naturally occurring T cell mutations enhance engineered T cell therapies

Improved cytosine base editors generated from TadA variants

Highly scalable arrayed CRISPR mediated gene silencing in primary lungsmall airway epithelial cells

Molecular and Physiological Effects of Browning Agents on White Adipocytes from Bone Marrow Mesenchymal Stromal Cells

Neoantigen-targeted CD8+ T cell responses with PD-1 blockade therapy

Protocol to create phenotypic primary human hepatocyte cultures using the RASTRUM 3D cell model platform

Ultra-deep sequencing validates safety of CRISPR/Cas9 genome editing in human hematopoietic stem and progenitor cells

A High-Throughput Microphysiological Liver Chip System to Model Drug-Induced Liver Injury Using Human Liver Organoids

Programmable C:G to G:C genome editing with CRISPR-Cas9-directed base excision repair proteins