Trem-cel, a CRISPR/Cas9 gene-edited allograft lacking CD33, shows rapid primary engraftment with CD33-negative hematopoiesis in patients with high-risk AML and avoids hematopoietic toxicity during gemtuzumab ozogamicin (GO) post-hematopoietic cell transplant (HCT) maintenance
Novel CLL-1-directed CAR-T cells Mediate Potent Antigen-specific Cytolytic Activity in
Mouse Models of Acute Myeloid Leukemia
Clinical Holds for Cell and Gene Therapy Trials: Risks, Impact, and Lessons Learned
GUMM: A Purpose-Built Computational Workflow for Single Cell Genotyping of Gene Editing Experiments
Development of a Gene Edited Next-Generation Hematopoietic Cell Transplant to Enable Acute Myeloid Leukemia Treatment by Solving Off-Tumor Toxicity
A Single Cell DNA Sequencing Resource and Computational Approach to Quantify CRISPR-Cas9 Gene Editing Allelism
CD33-Deleted Hematopoietic Stem and Progenitor Cells Display Normal Engraftment after Hematopoietic Cell Transplant (HCT) and Tolerate Post-HCT GemtuzumabOzogamicin(GO) Without Cytopenias
Gene Editing and Immunotherapeutic Targeting of ADGRE2/EMR2 to Enable Combinatorial Targeting in Acute Myeloid Leukemia (AML)
Initial First-In-Human Results: CD33-Deleted Hematopoietic Stem and Progenitor Cells Display Normal Engraftment after Hematopoietic Cell Transplantation (HCT) and Tolerate Post-HCT Gemtuzumab Ozogamicin (GO) without Cytopenias
