{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,1]],"date-time":"2026-05-01T21:18:15Z","timestamp":1777670295129,"version":"3.51.4"},"reference-count":47,"publisher":"SAGE Publications","issue":"23-24","license":[{"start":{"date-parts":[[2022,12,1]],"date-time":"2022-12-01T00:00:00Z","timestamp":1669852800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Human Gene Therapy"],"published-print":{"date-parts":[[2022,12]]},"abstract":"<jats:p>\n                    <jats:italic toggle=\"yes\">Ex vivo<\/jats:italic>\n                    gene therapy procedures targeting hematopoietic stem and progenitor cells (HSPCs) predominantly utilize lentivirus-based vectors for gene transfer. We provide the first pre-clinical evidence of the therapeutic utility of a foamy virus vector (FVV) for the genetic correction of human leukocyte adhesion deficiency type 1 (LAD-1), an inherited primary immunodeficiency resulting from mutation of the \u03b22 integrin common chain, CD18. CD34\n                    <jats:sup>+<\/jats:sup>\n                    HSPCs isolated from a severely affected LAD-1 patient were transduced under a current good manufacturing practice-compatible protocol with FVV harboring a therapeutic CD18 transgene. LAD-1-associated cellular chemotactic defects were ameliorated in transgene-positive, myeloid-differentiated LAD-1 cells assayed in response to a strong neutrophil chemoattractant\n                    <jats:italic toggle=\"yes\">in vitro<\/jats:italic>\n                    . Xenotransplantation of vector-transduced LAD-1 HSPCs in immunodeficient (NSG) mice resulted in long-term (\u223c5 months) human cell engraftment within murine bone marrow. Moreover, engrafted LAD-1 myeloid cells displayed\n                    <jats:italic toggle=\"yes\">in vivo<\/jats:italic>\n                    levels of transgene marking previously reported to ameliorate the LAD-1 phenotype in a large animal model of the disease. Vector insertion site analysis revealed a favorable vector integration profile with no overt evidence of genotoxicity. These results coupled with the unique biological features of wild-type foamy virus support the development of FVVs for\n                    <jats:italic toggle=\"yes\">ex vivo<\/jats:italic>\n                    gene therapy of LAD-1.\n                  <\/jats:p>","DOI":"10.1089\/hum.2022.065","type":"journal-article","created":{"date-parts":[[2022,9,12]],"date-time":"2022-09-12T08:08:03Z","timestamp":1662970083000},"page":"1293-1304","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":2,"title":["Preclinical Evaluation of Foamy Virus Vector-Mediated Gene Addition in Human Hematopoietic Stem\/Progenitor Cells for Correction of Leukocyte Adhesion Deficiency Type 1"],"prefix":"10.1177","volume":"33","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-6581-9653","authenticated-orcid":false,"given":"Richard H.","family":"Smith","sequence":"first","affiliation":[{"name":"Cellular and Molecular Therapeutics Branch, National Heart, Lung, Blood Institute, National Institutes of Health, Bethesda, Maryland, USA."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hanan","family":"Bloomer","sequence":"additional","affiliation":[{"name":"Cellular and Molecular Therapeutics Branch, National Heart, Lung, Blood Institute, National Institutes of Health, Bethesda, Maryland, USA."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Danielle","family":"Fink","sequence":"additional","affiliation":[{"name":"Neutrophil Monitoring Lab, Leidos Biomedical Research, Inc., Frederick National Laboratory for Cancer Research, Frederick, Maryland, USA."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Keyvan","family":"Keyvanfar","sequence":"additional","affiliation":[{"name":"Cellular and Molecular Therapeutics Branch, National Heart, Lung, Blood Institute, National Institutes of Health, Bethesda, Maryland, USA."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Md","family":"Nasimuzzaman","sequence":"additional","affiliation":[{"name":"Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"F\u00e1tima","family":"Sancheznieto","sequence":"additional","affiliation":[{"name":"Cellular and Molecular Therapeutics Branch, National Heart, Lung, Blood Institute, National Institutes of Health, Bethesda, Maryland, USA."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Roop","family":"Dutta","sequence":"additional","affiliation":[{"name":"Cellular and Molecular Therapeutics Branch, National Heart, Lung, Blood Institute, National Institutes of Health, Bethesda, Maryland, USA."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kacey","family":"Guenther Bui","sequence":"additional","affiliation":[{"name":"Cellular and Molecular Therapeutics Branch, National Heart, Lung, Blood Institute, National Institutes of Health, Bethesda, Maryland, USA."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Luigi J.","family":"Alvarado","sequence":"additional","affiliation":[{"name":"Cellular and Molecular Therapeutics Branch, National Heart, Lung, Blood Institute, National Institutes of Health, Bethesda, Maryland, USA."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"suffix":"Jr.","given":"Thomas R.","family":"Bauer","sequence":"additional","affiliation":[{"name":"Immune Deficiency-Cellular Therapy Program, National Cancer Institute, National Institutes of Health, Bethesda, Maryland, USA."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Dennis D.","family":"Hickstein","sequence":"additional","affiliation":[{"name":"Immune Deficiency-Cellular Therapy Program, National Cancer Institute, National Institutes of Health, Bethesda, Maryland, USA."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"David W.","family":"Russell","sequence":"additional","affiliation":[{"name":"Department of Medicine, University of Washington, Seattle, Washington, USA."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Punam","family":"Malik","sequence":"additional","affiliation":[{"name":"Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Johannes C.M.","family":"van der Loo","sequence":"additional","affiliation":[{"name":"Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Steven L.","family":"Highfill","sequence":"additional","affiliation":[{"name":"Center for Cellular Engineering, Department of Transfusion Medicine, Clinical Center, 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