Matthew Hebden Porteus

End-To-End, Fully Automated Manufacturing of Hematopoietic Stem and Progenitor Cell Therapy Via CCR5-Targeted Integration

This paper demonstrates an automated, high-throughput platform for manufacturing gene-edited hematopoietic stem and progenitor cells (geHSPCs) with preserved function and in vivo engraftment capacity.

Engineering hematopoietic stem and progenitor cells to generate red blood cells as viral traps against HIV-1

This paper engineers hematopoietic stem and progenitor cells to express CD4 on red blood cells, creating viral traps to neutralize HIV-1.

Epigenetic signature and key transcriptional regulators of human antigen-specific type 1 regulatory T cells

This study identifies the epigenetic signature and key transcription factors regulating human antigen-specific Tr1 cells, revealing their unique biology and potential for therapeutic applications.

TFU72 is a novel and potent DNA-PKcs inhibitor for enhancing homology-directed repair gene editing

The paper introduces TFU72, a potent DNA-PKcs inhibitor that significantly enhances HDR gene editing efficiency while mitigating genotoxic side effects.

Discovery of a primed endothelial progenitor that requires VEGF/ERK inhibition to complete vein differentiation

The paper identifies a primed endothelial progenitor that requires VEGF/ERK inhibition to differentiate into vein endothelial cells, revealing a two-step process for vein development.

Immune Composition of the Mononuclear Cell Fraction of Human Umbilical Cord Blood

This study characterizes the immune subset composition of umbilical cord blood mononuclear cells and compares them to adult peripheral blood, revealing distinct immune profiles and developmental trends.

Combining Cell-Intrinsic and -Extrinsic Resistance to HIV-1 By Engineering Hematopoietic Stem Cells for CCR5 Knockout and B Cell Secretion of Therapeutic Antibodies

This paper describes engineering hematopoietic stem cells to produce CCR5 knockout cells and secrete HIV-1 inhibiting antibodies, aiming for a one-time cure for HIV-1.

Durable reconstitution of sinonasal epithelium by transplant of CFTR gene corrected airway stem cells

This study demonstrates that gene-corrected human airway stem cells can durably engraft and restore CFTR function in murine respiratory epithelium, offering a potential therapy for cystic fibrosis.

Human striatal progenitor cells that contain inducible safeguards and overexpress BDNF rescue Huntington's disease phenotypes in R6/2 mice

This study shows that human striatal progenitor cells overexpressing BDNF and with inducible safety switches improve HD symptoms in R6/2 mice.

TET2 regulates early and late transitions in exhausted CD8+ T-cell differentiation and limits CAR T-cell function

This study shows that targeting TET2 in CAR T-cells improves their anti-tumor responses by limiting terminal exhaustion and enhancing therapeutic potential.

Engineering inducible signaling receptors to enable erythropoietin-free erythropoiesis

This paper develops small molecule-inducible EPO receptors to enable erythropoiesis without recombinant EPO, reducing costs and improving scalability of RBC manufacturing.

Dual α-globin and truncated erythropoietin receptor knock-in restores hemoglobin production in α-thalassemia major-derived hematopoietic stem and progenitor cells

This paper describes a genome editing strategy that restores hemoglobin production in α-thalassemia major-derived hematopoietic stem and progenitor cells by integrating a functional α-globin gene and a truncated erythropoietin receptor.

Using human genetics to develop strategies to increase erythropoietic output from genome-edited hematopoietic stem and progenitor cells

The paper uses human genetics and CRISPR/Cas9 to recreate a naturally occurring mutation that increases red blood cell production and shows its combination with a beta-thalassemia correction strategy enhances RBC development.

Combined Lineage Tracing and scRNA-seq Reveals Unexpected First Heart Field Predominance of Human iPSC Differentiation

The paper uses lineage tracing and scRNA-seq to show that human iPSC differentiation predominantly generates left ventricular cardiomyocytes from the first heart field.

Human assembloids reveal the consequences of CACNA1G gene variants in the thalamocortical pathway

This study uses human assembloids to investigate how CACNA1G gene variants affect thalamocortical pathway function in neuropsychiatric disorders.

Comparative analysis of CRISPR off-target activity discovery tools following ex vivo editing of CD34+ hematopoietic stem and progenitor cells

This study compares CRISPR off-target detection tools using ex vivo edited CD34+ HSPCs, finding that HiFi Cas9 reduces off-target sites and some methods outperform others in sensitivity and specificity.

Ultra-deep sequencing reveals no evidence of oncogenic mutations or enrichment by ex vivo CRISPR/Cas9 genome editing in human hematopoietic stem and progenitor cells

The paper uses ultra-deep sequencing to assess oncogenic mutations in human hematopoietic stem and progenitor cells after CRISPR/Cas9 editing, finding no evidence of tumorigenic variants.

Clinically Relevant Gene Editing in Hematopoietic Stem Cells for the Treatment of Pyruvate Kinase Deficiency Hemolytic Anemia

This study uses gene editing in hematopoietic stem cells to treat Pyruvate Kinase Deficiency Hemolytic Anemia, restoring RPK function and ATP levels in erythroid cells.

Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells

This study replaces the HBA1 gene with HBB in β-thalassemia-derived HSPCs using CRISPR, normalizing globin ratios and restoring functional hemoglobin.

Cas9-AAV6 Gene Correction of Beta-Globin in Autologous HSCs Improves Sickle Cell Disease Erythropoiesis in Mice

This study corrects the beta-globin gene in sickle cell disease patient-derived HSCs using CRISPR/Cas9 and shows improved in vivo erythropoiesis in a mouse model.

TRACE-Seq Reveals Clonal Reconstitution Dynamics of Gene Targeted Human Hematopoietic Stem Cells

The paper introduces TRACE-Seq, a method to track clonal reconstitution of gene-targeted human hematopoietic stem cells using barcoded AAV6 donor templates in immunodeficient mice.

Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I

This study uses CRISPR/Cas9 to edit hematopoietic stem cells to express iduronidase, correcting Mucopolysaccharidosis type I in mice.