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
doaj +2 more sources
Breaking Entry-and Species Barriers: LentiBOOST® Plus Polybrene Enhances Transduction Efficacy of Dendritic Cells and Monocytes by Adenovirus 5 [PDF]
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
doaj +2 more sources
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
doaj +2 more sources
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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Identification of a small molecule for enhancing lentiviral transduction of T cells
Molecular Therapy: Methods & 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
doaj +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
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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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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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Dasatinib is a potent enhancer for CAR T cell generation by CD3-targeted lentiviral vectors
Molecular Therapy: Methods & 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.
Angela H. Braun +3 more
doaj +1 more source
Advanced Science, 2023 Lentiviral vectors (LV) have become the dominant tool for stable gene transfer into lymphocytes including chimeric antigen receptor (CAR) gene delivery to T cells, a major breakthrough in cancer therapy.
Filippos T Charitidis +10 more
doaj +1 more source