B. R. Webber

Engineering Memory T Cells as a platform for Long-Term Enzyme Replacement Therapy in Lysosomal Storage Disorders

This study demonstrates that genetically engineered memory T cells can deliver enzyme replacement therapy for mucopolysaccharidosis type I, reducing GAG levels in multiple tissues for up to 22 weeks.

Precision Enhancement of CAR-NK Cells through Non-Viral Engineering and Highly Multiplexed Base Editing

This study enhances CAR-NK cell functionality using non-viral base editing and transposon integration, showing improved therapeutic potential in Burkitt's lymphoma models.

Ablation of SYK kinase from primary human Natural Killer cells via CRISPR/Cas9 enhances cytotoxicity and cytokine production

This study ablates SYK kinase in primary human NK cells using CRISPR/Cas9, finding it enhances cytotoxicity and cytokine production compared to other gene ablations.

Myosin Heavy Chain Converter Domain Mutations Drive Early-Stage Changes in Extracellular Matrix Dynamics in Hypertrophic Cardiomyopathy

This study identifies early-stage ECM dysregulation in HCM caused by MYH7/MYH6 mutations using hiPSC-derived cardiomyocytes.

Homology mediated end joining enables efficient non-viral targeted integration of large DNA templates in primary human T cells

This paper describes a non-viral method for efficiently integrating large DNA into primary human T cells using CRISPR-Cas9 and homology-mediated end joining, achieving high functionality and anti-tumor activity.

Genetic editing of CISH enhances T cell effector programs independently of immune checkpoint cell surface ligand expression

This study shows that genetic editing of CISH enhances T cell anti-tumor activity independently of PD-L1 expression, outperforming PD-1 and TIGIT disruption in certain contexts.

Non-Viral Engineering of CAR-NK and CAR-T cells using the Tc Buster Transposon System™

This paper demonstrates a non-viral method using the Tc Buster transposon system to deliver CAR constructs to primary human NK and T cells, achieving efficient gene expression and functional activity.

Internal checkpoint regulates T cell neoantigen reactivity and susceptibility to PD1 blockade

This study shows that CISH knockout in TIL enhances neoantigen reactivity and synergizes with PD1 blockade to improve tumor regression.

Engineering T cells to enhance 3D migration through structurally and mechanically complex tumor microenvironments

This study engineers T cells to improve 3D migration through tumor microenvironments by manipulating microtubule stability and contractility, showing enhanced migration in 3D matrices and tumors.

CRISPR-Cas9 cytidine and adenosine base editing of splice-sites mediates highly-efficient disruption of proteins in primary cells

The paper compares CBE and ABE base editors for splice-site disruption in T-cells, finding CBEs more reliable and splice-donor targeting most effective.

A Genetically Engineered Primary Human Natural Killer Cell Platform for Cancer Immunotherapy

This paper develops a CRISPR/Cas9-based method to edit human NK cells, enhancing their anti-cancer activity by knocking out ADAM17 and PD1.

Highly Multiplexed Genome Engineering Using CRISPR/Cas9 gRNA Arrays

This paper develops a CRISPR/Cas9 gRNA array system to enable efficient multiplex genome engineering by delivering up to 10 gRNAs as a single transcript.

Rapid DNA Replication Origin Licensing Protects Stem Cell Pluripotency

This study shows that rapid DNA replication origin licensing in pluripotent stem cells maintains pluripotency by enabling fast MCM loading despite short G1 phases.