ArticleMolecular therapy. Advances2026
Antimetabolites synergize with non-genotoxic antibody drug conjugate conditioning in hematopoietic stem cell lentiviral gene therapy.
Article in Molecular therapy. Advances, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Hematopoietic stem cell transplantation (HSCT) of genetically engineered cells is a promising treatment modality for monogenic diseases. However, hematopoietic stem cell (HSC)-directed lentiviral vector (LV) gene therapies remain limited by genotoxicities associated with standard non-targeted conditioning using irradiation or alkylating chemotherapy. We and others developed an antibody drug conjugate (ADC) with anti-CD117 and saporin (sap), which selectively depletes CD117-expressing HSC and progenitor cells (HSPCs). Since anti-CD117-sap does not generally provide engraftment comparable to conventional conditioning, we investigated the hypothesis anti-CD117-sap is not effective as a single conditioning agent due to insufficient HSPC depletion. We show anti-CD117-sap induces nearly complete residual HSPC entry into a non-G
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