Vectofusin-1 is a histidine-rich cationic amphipathic peptide derived from the LAH4 peptide family, and also acts as a Viral entry enhancer. Vectofusin-1 promotes the adhesion and fusion of retroviral/lentiviral vectors with cell membranes during viral entry, thereby improving transduction efficiency. Vectofusin-1 potently enhances lentiviral transduction of cells [1] [2].
In Vivo, Vectofusin-1 (12 μg/mL; ex vivo; 6-hour treatment) exhibits no deleterious effects on the transduction, viability, differentiation, or sustained engraftment of human hCD34+ cells in BALB-Rag/γC mice, supporting a safe profile for ex vivo HSC transduction applications[1].
In Vitro, Vectofusin-1 (12 μg/mL; 6 h) potently enhances the transduction efficiency of multiple lentiviral and retroviral pseudotypes in umbilical cord blood-derived hCD34+ hematopoietic stem/progenitor cells, resulting in a GFP+ cell proportion ranging from 57.5% to 79.4%, with no detectable cytotoxicity[1]. Vectofusin-1 (0-35 μg/mL; 5 days) enhances the transduction efficiency of GALVTR-LV in umbilical cord blood-derived hCD34+ hematopoietic stem/progenitor cells, with an EC50 of 6 μg/mL and a maximum average transduction efficiency of up to 67%[1]. Vectofusin-1 (0-100 μg/mL; overnight) has a median toxic concentration (TC50) of 60 μg/mL against umbilical cord blood-derived hCD34+ hematopoietic stem/progenitor cells, resulting in a 1-log safety index (TC50/EC50 = 10)[1]. Vectofusin-1 (12 μg/mL) effectively enhances the transduction efficiency of VSV-G-pseudotyped lentiviral vectors in umbilical cord blood-derived hCD34+ hematopoietic stem and progenitor cells (HSPCs), with the proportion of GFP-positive cells ranging from 76.2% to 82.7% in both total cell and subset analyses, which is comparable to the efficacy of Retronectin[2]. Vectofusin-1 (12 μg/mL) effectively enhances the transduction efficiency of VSV-G-pseudotyped lentiviral vectors in bone marrow-derived hCD34+ hematopoietic stem and progenitor cells (HSPCs); in analyses of total cells and subpopulations, the proportion of GFP-positive cells ranges from 69.8% to 74.8%, with a mean vector copy number (VCN) of 6.0 per cell, which is comparable to the efficacy of Retronectin[2].
References:
[1]. Fenard D, et al. Vectofusin-1, a new viral entry enhancer, strongly promotes lentiviral transduction of human hematopoietic stem cells. Mol Ther Nucleic Acids. 2013;2(5):e90. Published 2013 May 7.
[2]. Piovan C, et al. Vectofusin-1 Promotes RD114-TR-Pseudotyped Lentiviral Vector Transduction of Human HSPCs and T Lymphocytes. Mol Ther Methods Clin Dev. 2017;5:22-30. Published 2017 Mar 8.
In Vivo, Vectofusin-1 (12 μg/mL; ex vivo; 6-hour treatment) exhibits no deleterious effects on the transduction, viability, differentiation, or sustained engraftment of human hCD34+ cells in BALB-Rag/γC mice, supporting a safe profile for ex vivo HSC transduction applications[1].
In Vitro, Vectofusin-1 (12 μg/mL; 6 h) potently enhances the transduction efficiency of multiple lentiviral and retroviral pseudotypes in umbilical cord blood-derived hCD34+ hematopoietic stem/progenitor cells, resulting in a GFP+ cell proportion ranging from 57.5% to 79.4%, with no detectable cytotoxicity[1]. Vectofusin-1 (0-35 μg/mL; 5 days) enhances the transduction efficiency of GALVTR-LV in umbilical cord blood-derived hCD34+ hematopoietic stem/progenitor cells, with an EC50 of 6 μg/mL and a maximum average transduction efficiency of up to 67%[1]. Vectofusin-1 (0-100 μg/mL; overnight) has a median toxic concentration (TC50) of 60 μg/mL against umbilical cord blood-derived hCD34+ hematopoietic stem/progenitor cells, resulting in a 1-log safety index (TC50/EC50 = 10)[1]. Vectofusin-1 (12 μg/mL) effectively enhances the transduction efficiency of VSV-G-pseudotyped lentiviral vectors in umbilical cord blood-derived hCD34+ hematopoietic stem and progenitor cells (HSPCs), with the proportion of GFP-positive cells ranging from 76.2% to 82.7% in both total cell and subset analyses, which is comparable to the efficacy of Retronectin[2]. Vectofusin-1 (12 μg/mL) effectively enhances the transduction efficiency of VSV-G-pseudotyped lentiviral vectors in bone marrow-derived hCD34+ hematopoietic stem and progenitor cells (HSPCs); in analyses of total cells and subpopulations, the proportion of GFP-positive cells ranges from 69.8% to 74.8%, with a mean vector copy number (VCN) of 6.0 per cell, which is comparable to the efficacy of Retronectin[2].
References:
[1]. Fenard D, et al. Vectofusin-1, a new viral entry enhancer, strongly promotes lentiviral transduction of human hematopoietic stem cells. Mol Ther Nucleic Acids. 2013;2(5):e90. Published 2013 May 7.
[2]. Piovan C, et al. Vectofusin-1 Promotes RD114-TR-Pseudotyped Lentiviral Vector Transduction of Human HSPCs and T Lymphocytes. Mol Ther Methods Clin Dev. 2017;5:22-30. Published 2017 Mar 8.