M. H. Porteus

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.

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.

Small molecule correctors divert CFTR-F508del from ERAD by stabilizing sequential folding states

The paper identifies molecular machinery underlying CFTR-F508del ERAD and shows correctors stabilize RNF5-resistant folding states.

Genetically Corrected RAG2-SCID Human Hematopoietic Stem CellsRestore V(D)J-Recombinase and Rescue Lymphoid Deficiency

This study corrects the RAG2 gene in human hematopoietic stem cells to restore V(D)J recombination and lymphoid development in RAG2-SCID patients.

Reticular Dysgenesis-associated Adenylate Kinase 2 deficiency causes failure of myelopoiesis through disordered purine metabolism

This study shows AK2 deficiency disrupts mitochondrial metabolism, causing purine metabolism disorder and myelopoiesis failure through AMP accumulation and NAD+ depletion.

Highly Efficient Repair of the ΔF508 Mutation in Airway Stem Cells of Cystic Fibrosis Patients with Functional Rescue of the Differentiated Epithelia

This paper corrects the ΔF508 mutation in CF patient airway stem cells using CRISPR-Cas9 and AAV6, achieving high correction rates and functional rescue in differentiated epithelia.

Identification of Pre-Existing Adaptive Immunity to Cas9 Proteins in Humans

The paper detects pre-existing adaptive immunity to Cas9 proteins in humans, finding antibodies against both SaCas9 and SpCas9 and T-cells specific to SaCas9.