Advancements in Hematopoietic Stem Cell Gene Therapy: A Journey of Progress for Viral Transduction

被引：1
作者：
Giommetti, Aurora [1 ,2 ]
Papanikolaou, Eleni [1 ,3 ]
机构：
[1] Miltenyi Botec BV & Co KG, D-51429 Bergisch Gladbach, Germany
[2] Univ Freiburg, Fac Biol, D-79104 Freiburg, Germany
[3] Natl & Kapodistrian Univ Athens, Sch Med, Lab Biol, Athens 11527, Greece
来源：
CELLS | 2024年 / 13卷 / 12期
关键词：
gene therapy; hematopoietic stem cells (HSC); transduction; viral vectors; transduction enhancers; rare diseases; LENTIVIRAL VECTOR TRANSDUCTION; VSV-G-LVS; ALLOW EFFICIENT; IN-VITRO; T-CELLS; TRANSPLANTATION; RECONSTITUTION; ENHANCER; VIRUS; TRIAL;
D O I：
10.3390/cells13121039
中图分类号：
Q2 [细胞生物学];
学科分类号：
071009 ; 090102 ;
摘要：
Hematopoietic stem cell (HSC) transduction has undergone remarkable advancements in recent years, revolutionizing the landscape of gene therapy specifically for inherited hematologic disorders. The evolution of viral vector-based transduction technologies, including retroviral and lentiviral vectors, has significantly enhanced the efficiency and specificity of gene delivery to HSCs. Additionally, the emergence of small molecules acting as transduction enhancers has addressed critical barriers in HSC transduction, unlocking new possibilities for therapeutic intervention. Furthermore, the advent of gene editing technologies, notably CRISPR-Cas9, has empowered precise genome modification in HSCs, paving the way for targeted gene correction. These striking progresses have led to the clinical approval of medicinal products based on engineered HSCs with impressive therapeutic benefits for patients. This review provides a comprehensive overview of the collective progress in HSC transduction via viral vectors for gene therapy with a specific focus on transduction enhancers, highlighting the latest key developments, challenges, and future directions towards personalized and curative treatments.
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页数：15
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共 64 条
  • [1] Mystery solved: VSV-G-LVs do not allow efficient gene transfer into unstimulated T cells, B cells, and HSCs because they lack the LDL receptor
    Amirache, Fouzia
    Levy, Camille
    Costa, Caroline
    Mangeot, Philippe-Emmanuel
    Torbett, Bruce E.
    Wang, Cathy X.
    Negre, Didier
    Cosset, Francois-Loic
    Verhoeyen, Els
    [J]. BLOOD, 2014, 123 (09) : 1422 - 1424
  • [2] Anastasov N, 2016, METHODS MOL BIOL, V1448, P49, DOI 10.1007/978-1-4939-3753-0_4
  • [3] BACH FH, 1968, LANCET, V2, P1364
  • [4] Development of an in vitro genotoxicity assay to detect retroviral vector-induced lymphoid insertional mutants
    Bastone, Antonella L.
    Dziadek, Violetta
    John-Neek, Philipp
    Mansel, Friederike
    Fleischauer, Jenni
    Agyeman-Duah, Eric
    Schaudien, Dirk
    Dittrich-Breiholz, Oliver
    Schwarzer, Adrian
    Schambach, Axel
    Rothe, Michael
    [J]. MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT, 2023, 30 : 515 - 533
  • [5] Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection
    Biffi, Alessandra
    Bartholomae, Cynthia C.
    Cesana, Daniela
    Cartier, Natalie
    Aubourg, Patrik
    Ranzani, Marco
    Cesani, Martina
    Benedicenti, Fabrizio
    Plati, Tiziana
    Rubagotti, Enrico
    Merella, Stefania
    Capotondo, Alessia
    Sgualdino, Jacopo
    Zanetti, Gianluigi
    von Kalle, Christof
    Schmidt, Manfred
    Naldini, Luigi
    Montini, Eugenio
    [J]. BLOOD, 2011, 117 (20) : 5332 - 5339
  • [6] Stem-Cell Gene Therapy for the Wiskott-Aldrich Syndrome
    Boztug, Kaan
    Schmidt, Manfred
    Schwarzer, Adrian
    Banerjee, Pinaki P.
    Diez, Ines Avedillo
    Dewey, Ricardo A.
    Boehm, Marie
    Nowrouzi, Ali
    Ball, Claudia R.
    Glimm, Hanno
    Naundorf, Sonja
    Kuehlcke, Klaus
    Blasczyk, Rainer
    Kondratenko, Irina
    Marodi, Laszlo
    Orange, Jordan S.
    von Kalle, Christof
    Klein, Christoph
    [J]. NEW ENGLAND JOURNAL OF MEDICINE, 2010, 363 (20) : 1918 - 1927
  • [7] Gene Therapy for Wiskott-Aldrich Syndrome-Long-Term Efficacy and Genotoxicity
    Braun, Christian Joerg
    Boztug, Kaan
    Paruzynski, Anna
    Witzel, Maximilian
    Schwarzer, Adrian
    Rothe, Michael
    Modlich, Ute
    Beier, Rita
    Goehring, Gudrun
    Steinemann, Doris
    Fronza, Raffaele
    Ball, Claudia Regina
    Haemmerle, Reinhard
    Naundorf, Sonja
    Kuelcke, Klaus
    Rose, Martina
    Fraser, Chris
    Mathias, Liesl
    Ferrari, Rudolf
    Abboud, Miguel R.
    Al-Herz, Waleed
    Kondratenko, Irina
    Marodi, Laszlo
    Glimm, Hanno
    Schlegelberger, Brigitte
    Schambach, Axel
    Albert, Michael Heinrich
    Schmidt, Manfred
    von Kalle, Christof
    Klein, Christoph
    [J]. SCIENCE TRANSLATIONAL MEDICINE, 2014, 6 (227)
  • [8] Immunological barriers to haematopoietic stem cell gene therapy
    Charlesworth, Carsten T.
    Hsu, Ian
    Wilkinson, Adam C.
    Nakauchi, Hiromitsu
    [J]. NATURE REVIEWS IMMUNOLOGY, 2022, 22 (12) : 719 - 733
  • [9] Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency
    Cicalese, Maria Pia
    Ferrua, Francesca
    Castagnaro, Laura
    Pajno, Roberta
    Barzaghi, Federica
    Giannelli, Stefania
    Dionisio, Francesca
    Brigida, Immacolata
    Bonopane, Marco
    Casiraghi, Miriam
    Tabucchi, Antonella
    Carlucci, Filippo
    Grunebaum, Eyal
    Adeli, Mehdi
    Bredius, Robbert G.
    Puck, Jennifer M.
    Stepensky, Polina
    Tezcan, Ilhan
    Rolfe, Katie
    De Boever, Erika
    Reinhardt, Rickey R.
    Appleby, Jonathan
    Ciceri, Fabio
    Roncarolo, Maria Grazia
    Aiuti, Alessandro
    [J]. BLOOD, 2016, 128 (01) : 45 - 54
  • [10] PROTAMINE SULFATE AS AN EFFECTIVE ALTERNATIVE TO POLYBRENE IN RETROVIRAL-MEDIATED GENE-TRANSFER - IMPLICATIONS FOR HUMAN-GENE THERAPY
    CORNETTA, K
    ANDERSON, WF
    [J]. JOURNAL OF VIROLOGICAL METHODS, 1989, 23 (02) : 187 - 194
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