A Nontoxic Transduction Enhancer Enables Highly Efficient Lentiviral Transduction of Primary Murine T Cells and Hematopoietic Stem Cells [PDF]
Molecular Therapy: Methods & 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 ...
Marianne Delville +8 more
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Prostaglandin E2 as transduction enhancer affects competitive engraftment of human hematopoietic stem and progenitor cells [PDF]
Molecular Therapy: Methods & 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 ...
Valentina Poletti +3 more
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Viruses, 2022 Due to their ability to trigger strong immune responses, adenoviruses (HAdVs) in general and the serotype5 (HAdV-5) in particular are amongst the most popular viral vectors in research and clinical application.
Astrid Strack +7 more
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Creating New β-Globin-Expressing Lentiviral Vectors by High-Resolution Mapping of Locus Control Region Enhancer Sequences [PDF]
Molecular Therapy: Methods & Clinical Development, 2020 Hematopoietic stem cell gene therapy is a promising approach for treating disorders of the hematopoietic system. Identifying combinations of cis-regulatory elements that do not impede packaging or transduction efficiency when included in lentiviral ...
Richard A. Morgan +14 more
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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.
David Fenard +7 more
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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
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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
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An in vivo systemic massively parallel platform for deciphering animal tissue-specific regulatory function [PDF]
Frontiers in GeneticsIntroduction: Transcriptional regulation is an important process wherein non-protein coding enhancer sequences play a key role in determining cell type identity and phenotypic diversity.
Ashley R. Brown +27 more
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Enhanced Transgene Expression in Cord Blood CD34+-Derived Hematopoietic Cells, Including Developing T Cells and NOD/SCID Mouse Repopulating Cells, Following Transduction with Modified TRIP Lentiviral Vectors [PDF]
Molecular Therapy, 2001 The recent development of lentivirus-derived vectors is an important breakthrough in gene transfer technology because these vectors allow transduction of nondividing cells such as hematopoietic stem cells (HSC), due to an active nuclear import of reverse-
Aude Sirven +7 more
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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
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