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Importance: The essential steps of successful gene delivery by recombinant adeno-associated viruses (rAAVs) include vector internalization, intracellular trafficking, nuclear import, uncoating, double-stranded (ds)DNA conversion, and transgene expression. rAAV2.5T has a chimeric capsid of AAV2 VP1u and AAV5 VP2 and VP3 with the mutation A581T. Our investigation revealed that KIAA0319L, the multiple AAV serotype receptor, is not essential for vector internalization but remains critical for efficient vector transduction to human airway epithelia. Additionally, we identified that a novel gene WDR63 , whose cellular function is not well understood, plays an important role in vector transduction of human airway epithelia but not vector internalization and nuclear entry. Our study also discovered the substantial transduction potential of rAAV2.5T in basal stem cells of human airway epithelia, underscoring its utility in gene editing of human airways. Thus, the knowledge derived from this study holds promise for the advancement of gene therapy in the treatment of pulmonary genetic diseases.

Competing Interests: Z.Y. is a paid consultant for Spirovant Sciences, Inc. The remaining authors have no competing financial interests.

Titel:	Identification of host essential factors for recombinant AAV transduction of the polarized human airway epithelium.
Autor/in / Beteiligte Person:	Hao, S ; Zhang, X ; Ning, K ; Feng, Z ; Park, SY ; Aksu Kuz, C ; McFarlin, S ; Richart, D ; Cheng, F ; Zhang, EY ; Zhang-Chen, A ; Yan, Z ; Qiu, J
Zeitschrift:	Journal of virology, Jg. 97 (2023-12-21), Heft 12, S. e0133023
Veröffentlichung:	Washington Dc : American Society For Microbiology ; <i>Original Publication</i>: Baltimore, American Society for Microbiology., 2023
Medientyp:	academicJournal
ISSN:	1098-5514 (electronic)
DOI:	10.1128/jvi.01330-23
Schlagwort:	Humans Capsid Proteins genetics Capsid Proteins metabolism DNA Genetic Therapy methods Active Transport, Cell Nucleus Gene Editing trends Dependovirus genetics Dependovirus metabolism Epithelium metabolism Epithelium virology Gene Transfer Techniques trends Genetic Vectors genetics Transduction, Genetic Bronchi metabolism Bronchi virology
Sonstiges:	Nachgewiesen in: MEDLINE Sprachen: English Publication Type: Journal Article Language: English [J Virol] 2023 Dec 21; Vol. 97 (12), pp. e0133023. <i>Date of Electronic Publication: </i>2023 Nov 15. MeSH Terms: Dependovirus* / genetics ; Dependovirus* / metabolism ; Epithelium* / metabolism ; Epithelium* / virology ; Gene Transfer Techniques* / trends ; Genetic Vectors* / genetics ; Transduction, Genetic* ; Bronchi* / metabolism ; Bronchi* / virology ; Humans ; Capsid Proteins / genetics ; Capsid Proteins / metabolism ; DNA ; Genetic Therapy / methods ; Active Transport, Cell Nucleus ; Gene Editing / trends Comments: Update of: bioRxiv. 2023 Sep 27;:. (PMID: 37808760) References: Trends Microbiol. 2022 May;30(5):432-451. (PMID: 34711462) ; Nature. 2016 Feb 4;530(7588):108-12. (PMID: 26814968) ; Mol Ther Methods Clin Dev. 2020 Sep 16;19:186-200. (PMID: 33209961) ; Development. 2019 Oct 23;146(20):. (PMID: 31558434) ; J Virol. 1998 Feb;72(2):1438-45. (PMID: 9445046) ; Mol Ther Methods Clin Dev. 2020 Mar 17;17:601-611. (PMID: 32280726) ; Int J Stem Cells. 2016 May 30;9(1):31-5. (PMID: 27426083) ; Mol Ther. 2017 Jun 7;25(6):1387-1394. (PMID: 28408179) ; Virology. 2000 Mar 30;269(1):137-47. (PMID: 10725206) ; J Virol. 2018 Mar 14;92(7):. (PMID: 29343568) ; J Endod. 2015 Feb;41(2):205-11. (PMID: 25498833) ; Am J Physiol Lung Cell Mol Physiol. 2003 May;284(5):L844-54. (PMID: 12676769) ; Lancet. 2016 Aug 13;388(10045):661-72. (PMID: 27375040) ; Proc Natl Acad Sci U S A. 2009 Mar 10;106(10):3865-70. (PMID: 19237554) ; Mol Ther. 2021 Feb 3;29(2):464-488. (PMID: 33309881) ; Sci Adv. 2023 Jan 13;9(2):eade0869. (PMID: 36630517) ; iScience. 2022 Sep 12;25(10):105114. (PMID: 36185377) ; Nat Rev Drug Discov. 2019 May;18(5):358-378. (PMID: 30710128) ; Annu Rev Virol. 2014 Nov;1(1):427-51. (PMID: 26958729) ; Nat Med. 1999 May;5(5):467-8. (PMID: 10229209) ; J Virol. 2006 Sep;80(18):9093-103. (PMID: 16940521) ; J Virol. 2015 Feb;89(3):1660-72. (PMID: 25410855) ; J Gen Virol. 2019 Mar;100(3):367-368. (PMID: 30672729) ; Curr Opin Virol. 2017 Jun;24:124-131. (PMID: 28672171) ; Mol Ther. 2013 Dec;21(12):2181-94. (PMID: 23896725) ; Gene Ther. 2021 Dec;28(12):683-696. (PMID: 33658649) ; J Virol. 2017 Aug 24;91(18):. (PMID: 28679762) ; Gene Ther. 2019 Jun;26(6):240-249. (PMID: 30962536) ; Nat Commun. 2018 Dec 21;9(1):5416. (PMID: 30575746) ; Genome Biol. 2014;15(12):554. (PMID: 25476604) ; N Engl J Med. 2021 Nov 18;385(21):1961-1973. (PMID: 34788507) ; Nat Rev Drug Discov. 2021 Mar;20(3):173-174. (PMID: 33495615) ; J Virol. 2011 Sep;85(17):9023-30. (PMID: 21697483) ; Mol Ther. 2020 Feb 5;28(2):367-381. (PMID: 31784416) ; Gene Ther. 2013 Mar;20(3):328-37. (PMID: 22695783) ; Curr Opin Virol. 2016 Jun;18:89-96. (PMID: 27262111) ; Proc Natl Acad Sci U S A. 2005 Feb 22;102(8):2952-7. (PMID: 15703296) ; Virology. 1996 Mar 1;217(1):124-30. (PMID: 8599196) ; Mol Ther. 2012 Nov;20(11):2087-97. (PMID: 22850678) ; Mucosal Immunol. 2021 Sep;14(5):978-990. (PMID: 33608655) ; Methods Mol Med. 2005;107:183-206. (PMID: 15492373) ; mBio. 2020 Nov 6;11(6):. (PMID: 33158999) ; Hum Mol Genet. 2019 Oct 1;28(R1):R88-R94. (PMID: 31332440) ; J Virol. 2004 Mar;78(6):2863-74. (PMID: 14990705) ; EMBO Rep. 2020 Apr 3;21(4):e49269. (PMID: 32128961) ; mBio. 2023 Feb 28;14(1):e0352822. (PMID: 36719192) ; Virology. 1999 Nov 25;264(2):436-40. (PMID: 10562505) ; Mol Ther Methods Clin Dev. 2018 Oct 04;11:40-51. (PMID: 30397626) ; Mol Ther Methods Clin Dev. 2023 Mar 02;29:70-80. (PMID: 36950451) ; Curr Protoc Microbiol. 2020 Sep;58(1):e107. (PMID: 32639683) Grant Information: R21 AI166293 United States AI NIAID NIH HHS; P30 CA168524 United States CA NCI NIH HHS; P30 GM103326 United States GM NIGMS NIH HHS; S10 OD032207 United States OD NIH HHS; S10 OD023625 United States OD NIH HHS; P30 DK054759 United States DK NIDDK NIH HHS; R01 AI150877 United States AI NIAID NIH HHS; R21 AI156448 United States AI NIAID NIH HHS Contributed Indexing: Keywords: KIAA0319L; WDR63; human airway epithelium; rAAV2.5T; transduction Substance Nomenclature: 0 (Capsid Proteins) ; 9007-49-2 (DNA) ; 0 (DNAI3 protein, human) ; 0 (KIAA0319L protein, human) Entry Date(s): Date Created: 20231115 Date Completed: 20240102 Latest Revision: 20240329 Update Code: 20240329 PubMed Central ID: PMC10734497

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Identification of host essential factors for recombinant AAV transduction of the polarized human airway epithelium.