{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,1]],"date-time":"2026-05-01T21:19:26Z","timestamp":1777670366087,"version":"3.51.4"},"reference-count":63,"publisher":"SAGE Publications","issue":"1-2","license":[{"start":{"date-parts":[[2023,1,1]],"date-time":"2023-01-01T00:00:00Z","timestamp":1672531200000},"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":[[2023,1]]},"abstract":"<jats:p>\n                    Chronic granulomatous disease (CGD) is a rare congenital immunodeficiency characterized by a defect in nicotinamide adenine dinucleotide phosphate oxidase required for phagocytosis. Hematopoietic stem cell (HSC) transplantation is currently the only curative treatment, but it is ladened with morbidities and mortality. Gene therapy is a promising treatment for CGD. However, if not properly designed, the gene therapy approach may not be successful. We engineered lentiviral vectors (LVs) carrying a universal promoter (\n                    <jats:italic toggle=\"yes\">EF1a<\/jats:italic>\n                    ) and two myeloid-specific promoters (\n                    <jats:italic toggle=\"yes\">miR223<\/jats:italic>\n                    and\n                    <jats:italic toggle=\"yes\">CD68<\/jats:italic>\n                    ) to drive the expression of green fluorescence protein (GFP) or\n                    <jats:italic toggle=\"yes\">CYBB<\/jats:italic>\n                    , one of the key defective genes causing CGD. Tissue-specific LV expression was investigated\n                    <jats:italic toggle=\"yes\">in vitro<\/jats:italic>\n                    and in a CGD mouse model. We compared GFP expression in both myeloid differentiated and undifferentiated HSCs. The CGD mice were transplanted with LV-modified mouse HSCs to investigate expression of\n                    <jats:italic toggle=\"yes\">CYBB<\/jats:italic>\n                    and restoration of reactive oxygen species. The LV promoters were further compared under low and high-transgenic conditions to assess safety and therapeutic efficacy. A pneumonia disease model based on pathogenic\n                    <jats:italic toggle=\"yes\">Staphylococcus aureus<\/jats:italic>\n                    challenge was established to assess the survival rate and body weight change. All three promoters demonstrated ectopic\n                    <jats:italic toggle=\"yes\">CYBB<\/jats:italic>\n                    expression\n                    <jats:italic toggle=\"yes\">in vitro<\/jats:italic>\n                    and\n                    <jats:italic toggle=\"yes\">in vivo<\/jats:italic>\n                    . The\n                    <jats:italic toggle=\"yes\">EF1a<\/jats:italic>\n                    promoter showed the highest expression of GFP or\n                    <jats:italic toggle=\"yes\">CYBB<\/jats:italic>\n                    in transduced cells, including HSCs without cytotoxicity, whereas the LV-\n                    <jats:italic toggle=\"yes\">miR223<\/jats:italic>\n                    showed the highest transgene delivery efficiency with high myeloid specificity. Importantly, under low-transgenic condition, only the LV\n                    <jats:italic toggle=\"yes\">-EF1a-CYBB<\/jats:italic>\n                    showed high antibacterial activity\n                    <jats:italic toggle=\"yes\">in vivo<\/jats:italic>\n                    .\n                  <\/jats:p>","DOI":"10.1089\/hum.2022.140","type":"journal-article","created":{"date-parts":[[2022,10,24]],"date-time":"2022-10-24T01:37:10Z","timestamp":1666575430000},"page":"19-29","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":2,"title":["Lentiviral Gene Therapy of Chronic Granulomatous Disease: Functional Assessment of Universal and Tissue-Specific Promoters"],"prefix":"10.1177","volume":"34","author":[{"given":"Haokun","family":"Yuan","sequence":"first","affiliation":[{"name":"School of Medicine, University of Electronic Science and Technology of China, Chengdu, China."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Xiaomei","family":"Wu","sequence":"additional","affiliation":[{"name":"School of Medicine, University of Electronic Science and Technology of China, Chengdu, China."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hongwei","family":"Liu","sequence":"additional","affiliation":[{"name":"School of Medicine, University of Electronic Science and Technology of China, Chengdu, China."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2395-5216","authenticated-orcid":false,"given":"Lung-Ji","family":"Chang","sequence":"additional","affiliation":[{"name":"School of Medicine, University of Electronic Science and Technology of China, Chengdu, China."},{"name":"Shenzhen Geno-Immune Medical Institute, Shenzhen, China."}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"179","published-online":{"date-parts":[[2023,1,17]]},"reference":[{"key":"e_1_3_7_2_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.clim.2007.09.008"},{"key":"e_1_3_7_3_2","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.24573"},{"key":"e_1_3_7_4_2","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0005234"},{"key":"e_1_3_7_5_2","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2249.2008.03644.x"},{"key":"e_1_3_7_6_2","doi-asserted-by":"publisher","DOI":"10.1093\/cid\/ciu1154"},{"key":"e_1_3_7_7_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaci.2015.11.041"},{"key":"e_1_3_7_8_2","doi-asserted-by":"publisher","DOI":"10.1007\/s13765-012-2098-1"},{"key":"e_1_3_7_9_2","doi-asserted-by":"publisher","DOI":"10.1007\/s12325-017-0636-2"},{"key":"e_1_3_7_10_2","doi-asserted-by":"publisher","DOI":"10.3389\/fped.2020.00327"},{"key":"e_1_3_7_11_2","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2011.166"},{"key":"e_1_3_7_12_2","doi-asserted-by":"publisher","DOI":"10.1007\/s10038-006-0039-8"},{"key":"e_1_3_7_13_2","doi-asserted-by":"publisher","DOI":"10.1002\/humu.20820"},{"key":"e_1_3_7_14_2","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2016.126"},{"key":"e_1_3_7_15_2","doi-asserted-by":"publisher","DOI":"10.1038\/sj.mt.6300355"},{"key":"e_1_3_7_16_2","doi-asserted-by":"publisher","DOI":"10.1186\/s40425-017-0246-1"},{"key":"e_1_3_7_17_2","doi-asserted-by":"publisher","DOI":"10.1002\/cpim.93"},{"key":"e_1_3_7_18_2","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2020.01941"},{"key":"e_1_3_7_19_2","doi-asserted-by":"publisher","DOI":"10.1080\/2162402X.2019.1671761"},{"key":"e_1_3_7_20_2","doi-asserted-by":"publisher","DOI":"10.1136\/jitc-2020-000990"},{"key":"e_1_3_7_21_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41375-018-0106-0"},{"key":"e_1_3_7_22_2","doi-asserted-by":"publisher","DOI":"10.1186\/s13045-018-0572-x"},{"key":"e_1_3_7_23_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2016.12.006"},{"key":"e_1_3_7_24_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.htct.2019.06.007"},{"key":"e_1_3_7_25_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41598-019-46938-7"},{"key":"e_1_3_7_26_2","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2019.02361"},{"key":"e_1_3_7_27_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2020.09.030"},{"key":"e_1_3_7_28_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.freeradbiomed.2019.10.005"},{"key":"e_1_3_7_29_2","doi-asserted-by":"publisher","DOI":"10.1038\/nm1388"},{"key":"e_1_3_7_30_2","doi-asserted-by":"publisher","DOI":"10.1523\/JNEUROSCI.1483-16.2018"},{"key":"e_1_3_7_31_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2007-05-087759"},{"key":"e_1_3_7_32_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.celrep.2019.03.009"},{"key":"e_1_3_7_33_2","doi-asserted-by":"publisher","DOI":"10.1242\/dev.107086"},{"key":"e_1_3_7_34_2","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2010.226"},{"key":"e_1_3_7_35_2","doi-asserted-by":"publisher","DOI":"10.1089\/humc.2017.245"},{"key":"e_1_3_7_36_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2001-12-0165"},{"key":"e_1_3_7_37_2","doi-asserted-by":"publisher","DOI":"10.1038\/gt.2011.65"},{"key":"e_1_3_7_38_2","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2014.87"},{"key":"e_1_3_7_39_2","doi-asserted-by":"publisher","DOI":"10.19185\/matters.201805000005"},{"key":"e_1_3_7_40_2","doi-asserted-by":"publisher","DOI":"10.1038\/nm.2088"},{"key":"e_1_3_7_41_2","doi-asserted-by":"publisher","DOI":"10.1089\/hgtb.2012.157"},{"key":"e_1_3_7_42_2","doi-asserted-by":"publisher","DOI":"10.1038\/gt.2010.138"},{"key":"e_1_3_7_43_2","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2009.134"},{"key":"e_1_3_7_44_2","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2008.5"},{"key":"e_1_3_7_45_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V96.9.3040"},{"key":"e_1_3_7_46_2","doi-asserted-by":"publisher","DOI":"10.1172\/JCI37630"},{"key":"e_1_3_7_47_2","doi-asserted-by":"publisher","DOI":"10.1172\/JCI62189"},{"key":"e_1_3_7_48_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41591-019-0735-5"},{"key":"e_1_3_7_49_2","doi-asserted-by":"publisher","DOI":"10.1038\/sj.gt.3300895"},{"key":"e_1_3_7_50_2","doi-asserted-by":"publisher","DOI":"10.1006\/viro.1999.9850"},{"key":"e_1_3_7_51_2","doi-asserted-by":"publisher","DOI":"10.1186\/1742-4690-1-37"},{"key":"e_1_3_7_52_2","doi-asserted-by":"publisher","DOI":"10.1007\/978-1-60761-533-0_11"},{"key":"e_1_3_7_53_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jbc.2021.101397"},{"key":"e_1_3_7_54_2","doi-asserted-by":"publisher","DOI":"10.1038\/nm1079"},{"key":"e_1_3_7_55_2","doi-asserted-by":"publisher","DOI":"10.1126\/science.1137165"},{"key":"e_1_3_7_56_2","doi-asserted-by":"publisher","DOI":"10.1038\/bmt.2013.149"},{"key":"e_1_3_7_57_2","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2016.150"},{"key":"e_1_3_7_58_2","doi-asserted-by":"publisher","DOI":"10.1007\/s12325-017-0636-2"},{"key":"e_1_3_7_59_2","doi-asserted-by":"publisher","DOI":"10.1007\/s12016-020-08800-x"},{"key":"e_1_3_7_60_2","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.94.22.12133"},{"key":"e_1_3_7_61_2","doi-asserted-by":"publisher","DOI":"10.1038\/nature06607"},{"key":"e_1_3_7_62_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.cell.2007.02.048"},{"key":"e_1_3_7_63_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.cell.2007.04.040"},{"key":"e_1_3_7_64_2","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2018.137"}],"container-title":["Human Gene Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1089\/hum.2022.140","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1089\/hum.2022.140","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1089\/hum.2022.140","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,4,29]],"date-time":"2026-04-29T07:56:32Z","timestamp":1777449392000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1089\/hum.2022.140"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,1]]},"references-count":63,"journal-issue":{"issue":"1-2","published-print":{"date-parts":[[2023,1]]}},"alternative-id":["10.1089\/hum.2022.140"],"URL":"https:\/\/doi.org\/10.1089\/hum.2022.140","relation":{},"ISSN":["1043-0342","1557-7422"],"issn-type":[{"value":"1043-0342","type":"print"},{"value":"1557-7422","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,1]]}}}