Results 11 to 20 of about 289,359 (292)

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, Tayebeh Soheili, Florence Bellier, Amandine Durand, Adeline Denis, Chantal Lagresle-Peyrou, Marina Cavazzana, Isabelle Andre-Schmutz, Emmanuelle Six   +8 more
doaj   +5 more sources

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, Annita Montepeloso, Danilo Pellin, Alessandra Biffi   +3 more
doaj   +6 more sources

Vectofusin-1, a New Viral Entry Enhancer, Strongly Promotes Lentiviral Transduction of Human Hematopoietic Stem Cells

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, Dina Ingrao, Ababacar Seye, Julien Buisset, Sandrine Genries, Samia Martin, Antoine Kichler, Anne Galy   +7 more
doaj   +4 more sources

Enhancing the transduction efficiency of lentiviral vectors in CAR-T cell therapy through an optimization workflow [PDF]

Frontiers in Medicine
Efficient 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, Jaciara Fernanda Gomes Gama, Ana Godinho-Santos, Joao Goncalves   +3 more
doaj   +2 more sources

Optimized peptide nanofibrils as efficient transduction enhancers for in vitro and ex vivo gene transfer

Frontiers in Immunology, 2023
Chimeric antigen receptor (CAR)-T cell therapy is a groundbreaking immunotherapy for cancer. However, the intricate and costly manufacturing process remains a hurdle.
Lena Rauch-Wirth, Alexander Renner, Kübra Kaygisiz, Tatjana Weil, Laura Zimmermann, Armando A. Rodriguez-Alfonso, Armando A. Rodriguez-Alfonso, Desiree Schütz, Sebastian Wiese, Ludger Ständker, Tanja Weil, Dominik Schmiedel, Jan Münch, Jan Münch   +13 more
doaj   +3 more sources

Development of a gene-activated matrix for enhanced AAV gene delivery in vitro [PDF]

Frontiers in Bioengineering and Biotechnology
Recombinant 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, Florent Poulhès, Cedric Sapet, Neelam Iqbal, Payal Ganguly, Payal Ganguly, Maximilian Kampick, Bastian Hoechst, Mario Marotta, Katja Dumler, Animesh Jha, Elena A. Jones, Peter V. Giannoudis, Peter V. Giannoudis, Marilys Blanchy, Percy Knolle, Olivier Zelphati, Martina Anton   +17 more
doaj   +2 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, Shruti Sathish, Evan London, Anh Le, Quan Li, Bjorg Gudmundsdottir, Duck-Yeon Lee, Emma V. Burke, Bradley P. Yates, David R. Liu, Matthew Hsieh, Alexis Leonard, William A. Eaton, Naoya Uchida, Francis J. Pierciey, Jr., Gregory A. Newby, John F. Tisdale, Selami Demirci   +17 more
doaj   +2 more sources

JSRV Intragenic Enhancer Element Increases Expression from a Heterologous Promoter and Promotes High Level AAV-Mediated Transgene Expression in the Lung and Liver of Mice

Viruses, 2020
Jaagsiekte sheep retrovirus (JSRV) induces tumors in the distal airways of sheep and goats. A putative intragenic enhancer, termed JE, localized to the 3′ end of the JSRV env gene, has been previously described.
Darrick L. Yu, Natalie Chow, Sarah K. Wootton   +2 more
doaj   +3 more sources

Transduction Enhancers Enable Efficient Human Adenovirus Type 5-Mediated Gene Transfer into Human Multipotent Mesenchymal Stromal Cells

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, Olivia Lübbers, Linus Weiß, Karmveer Singh, Karin Scharffetter-Kochanek, Markus Rojewski, Hubert Schrezenmeier, Philip Helge Zeplin, Wolfgang Funk, Lea Krutzke, Stefan Kochanek, Astrid Kritzinger   +11 more
doaj   +1 more source

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, Annika M. Frank, Naphang Ho, Christian J. Buchholz   +3 more
doaj   +1 more source