Prostaglandin E2 as transduction enhancer affects competitive engraftment of human hematopoietic stem and progenitor cells [PDF]
Molecular Therapy - Methods and Clinical Development, 2023 Ex vivo gene therapy (GT) is a promising treatment for inherited genetic diseases. An ideal transduction protocol should determine high gene marking in long-term self-renewing hematopoietic stem cells (HSCs), preserving their repopulation potential ...
Danilo Pellín +2 more
exaly +4 more sources
A Nontoxic Transduction Enhancer Enables Highly Efficient Lentiviral Transduction of Primary Murine T Cells and Hematopoietic Stem Cells [PDF]
Molecular Therapy - Methods and Clinical Development, 2018 Lentiviral vectors have emerged as an efficient, safe therapeutic tool for gene therapy based on hematopoietic stem cells (HSCs) or T cells. However, the monitoring of transduced cells in preclinical models remains challenging because of the inefficient ...
Emmanuelle Six +2 more
exaly +4 more sources
Enhancing the transduction efficiency of lentiviral vectors in CAR-T cell therapy through an optimization workflow [PDF]
Frontiers in MedicineEfficient lentiviral (LV) transduction is a cornerstone of CAR-T manufacturing, yet performance is often construct-specific and highly sensitive to production and delivery parameters. We developed a stepwise optimization workflow using an underperforming
Rita Ferreira +3 more
doaj +2 more sources
Identification of a small molecule for enhancing lentiviral transduction of T cells
Molecular Therapy - Methods and Clinical Development, 2023 Genetic modification of cells using viral vectors has shown huge therapeutic benefit in multiple diseases. However, inefficient transduction contributes to the high cost of these therapies.
Paulina Malach +10 more
exaly +3 more sources
Dasatinib is a potent enhancer for CAR T cell generation by CD3-targeted lentiviral vectors
Molecular Therapy - Methods and Clinical Development, 2023 CD3-targeted lentiviral vectors (CD3-LVs) mediate selective transduction of human T lymphocytes in vitro and in vivo while simultaneously activating the targeted cells.
Christian J Buchholz, Naphang Ho
exaly +3 more sources
Comparative analysis of CRISPR-Cas9, lentiviral transduction, and base editing for sickle cell disease in a murine model [PDF]
Blood Advances: Sickle cell disease (SCD) is a red blood cell disorder caused by a mutation in the β-globin gene, leading to sickle hemoglobin polymerization under low oxygen conditions.
Henna Butt +17 more
doaj +2 more sources
Development of a gene-activated matrix for enhanced AAV gene delivery in vitro [PDF]
Frontiers in Bioengineering and BiotechnologyRecombinant AAV vectors are among the most extensively studied vectors for viral gene delivery due to their unique safety profile and their ability to mediate efficient, long-term transgene expression by persisting episomally in the nucleus.
Ahmed Musoski +17 more
doaj +2 more sources
Molecular Therapy - Nucleic Acids, 2013 Gene transfer into hCD34+ hematopoietic stem/progenitor cells (HSCs) using human immunodeficiency virus type 1 (HIV-1)-based lentiviral vectors (LVs) has several promising therapeutic applications.
Anne Galy +2 more
exaly +3 more sources
Spatial and longitudinal tracking of enhancer-AAV vectors that target transgene expression to injured mouse myocardium [PDF]
eLifeTissue regeneration enhancer elements (TREEs) direct expression of target genes in injured and regenerating tissues. Additionally, TREEs of zebrafish origin were shown to direct expression of transgenes in border zone regions after cardiac injury when ...
David W Wolfson +11 more
doaj +2 more sources
Viruses, 2021 Human multipotent mesenchymal stromal cells (hMSCs) are currently developed as cell therapeutics for different applications, including regenerative medicine, immune modulation, and cancer treatment.
Robin Nilson +11 more
doaj +1 more source