Pseudotyped recombinant adeno-associated viral vectors mediate efficient gene transfer into primary human CD34(+) peripheral blood progenitor cells

Cytotherapy
Marlon R VeldwijkFrederik Wenz

Abstract

Because of their pluripotency, human CD34(+) peripheral blood progenitor cells (PBPC) are targets of interest for the treatment of many acquired and inherited disorders using gene therapeutic approaches. Unfortunately, most current vector systems lack either sufficient transduction efficiency or an appropriate safety profile. Standard single-stranded recombinant adeno-associated virus 2 (AAV2)-based vectors offer an advantageous safety profile, yet lack the required efficiency in human PBPC. A panel of pseudotyped AAV vectors (designated AAV2/x, containing the vector genome of serotype 2 and capsid of serotype x, AAV2/1-AAV2/6) was screened on primary human granulocyte-colony-stimulating factor (G-CSF)-mobilized CD34(+) PBPC to determine their gene transfer efficacy. Additionally, double-stranded self-complementary AAV (dsAAV) were used to determine possible second-strand synthesis limitations. AAV2/6 vectors proved to be the most efficient [12.8% (1.8-25.4%) transgene-expressing PBPC after a single transduction], being significantly more efficient (all P<0.005) than the other vectors [AAV2/2, 2.0% (0.2-7.3%); AAV2/1, 1.3% (0.1-2.9%); others, <; 1% transgene-expressing PBPC]. In addition, the relevance of the single-to-double-s...Continue Reading

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Citations

Jun 14, 2014·Molecular Therapy : the Journal of the American Society of Gene Therapy·Laura J SmithSaswati Chatterjee
May 3, 2011·Expert Opinion on Biological Therapy·Patrick MaierFrederik Wenz
May 8, 2010·Experimental Hematology·Natascha K SchuhmannHildegard Büning
Nov 20, 2016·Cellular Microbiology·Ferdy R van Diemen, Robert Jan Lebbink
May 12, 2017·Human Gene Therapy·Nolan BrownMatthew L Hirsch
Sep 13, 2019·The Journal of Allergy and Clinical Immunology·Marie PouzollesValérie S Zimmermann
Apr 25, 2021·Virology Journal·Rossana Colón-ThilletDaniel Stone
May 23, 2021·Journal of Molecular Medicine : Official Organ of the Gesellschaft Deutscher Naturforscher Und Ärzte·Konstantin Kochergin-NikitskySvetlana Smirnikhina

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