Gene Therapeutic Delivery to the Salivary Glands.
Gene therapy
Gene transfer
Radiation damage
Salivary gland regeneration
Sjogren’s syndrome
Journal
Advances in experimental medicine and biology
ISSN: 0065-2598
Titre abrégé: Adv Exp Med Biol
Pays: United States
ID NLM: 0121103
Informations de publication
Date de publication:
2023
2023
Historique:
medline:
25
8
2023
pubmed:
25
2
2023
entrez:
24
2
2023
Statut:
ppublish
Résumé
The salivary glands, exocrine glands in our body producing saliva, can be easily damaged by various factors. Radiation therapy and Sjogren's syndrome (a systemic autoimmune disease) are the two main causes of salivary gland damage, leading to a severe reduction in patients' quality of life. Gene transfer to the salivary glands has been considered a promising approach to treating the dysfunction. Gene therapy has long been applied to cure multiple diseases, including cancers, and hereditary and infectious diseases, which are proven to be safe and effective for the well-being of patients. The application of this treatment on salivary gland injuries has been studied for decades, yet its clinical progress is delayed. This chapter provides a coup d'oeil into gene transfer methods and various gene/vector types for salivary glands to help the new scientists and update established scientists on the progress that has been made during the past decades for the treatment of salivary gland disorders.
Identifiants
pubmed: 36826746
doi: 10.1007/5584_2023_766
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
55-68Informations de copyright
© 2023. The Author(s), under exclusive license to Springer Nature Switzerland AG.
Références
Nakagami H, Morishita R (2022) COVID-19 vaccine utilizing the technology of gene therapy. Nihon Ronen Igakkai Zasshi 59(1):34–38
Abu Abed OS (2021) Gene therapy avenues and COVID-19 vaccines. Genes Immun 22(2):120–124
pubmed: 34079091
pmcid: 8170448
doi: 10.1038/s41435-021-00136-6
Acosta A, Hurtado MD, Gorbatyuk O, La Sala M, Duncan D, Aslanidi G et al (2011) Salivary PYY: a putative bypass to satiety. PLoS One 6(10):e26137
pubmed: 22028819
pmcid: 3189958
doi: 10.1371/journal.pone.0026137
Alevizos I, Zheng C, Cotrim AP, Liu S, McCullagh L, Billings ME et al (2017) Late responses to adenoviral-mediated transfer of the aquaporin-1 gene for radiation-induced salivary hypofunction. Gene Ther 24(3):176–186
pubmed: 27996967
doi: 10.1038/gt.2016.87
Arany S, Xu Q, Hernady E, Benoit DSW, Dewhurst S, Ovitt CE (2012) Pro-apoptotic gene knockdown mediated by nanocomplexed siRNA reduces radiation damage in primary salivary gland cultures. J Cell Biochem 113(6):1955–1965
pubmed: 22253051
pmcid: 3360791
doi: 10.1002/jcb.24064
Arany S, Benoit DSW, Dewhurst S, Ovitt CE (2013) Nanoparticle-mediated gene silencing confers radioprotection to salivary glands in vivo. Mol Ther 21(6):1182–1194
pubmed: 23511246
pmcid: 3677305
doi: 10.1038/mt.2013.42
Azuma M, Tamatani T, Kasai Y, Sato M (1993) Immortalization of normal human salivary gland cells with duct-, myoepithelial-, acinar-, or squamous phenotype by transfection with SV40 ori- mutant deoxyribonucleic acid. Lab Investig 69(1):24–42
pubmed: 7687310
Baba Y, Yazawa T, Kanegae Y, Sakamoto S, Saito I, Morimura N et al (2007) Keratinocyte growth factor gene transduction ameliorates acute lung injury and mortality in mice. Hum Gene Ther 18(2):130–141
pubmed: 17328680
doi: 10.1089/hum.2006.137
Barka T, van der Noen HM (1996) Retrovirus-mediated gene transfer into salivary glands in vivo. Hum Gene Ther 7(5):613–618
pubmed: 8845386
doi: 10.1089/hum.1996.7.5-613
Baum BJ, O’Connell BC (1999) In vivo gene transfer to salivary glands. Crit Rev Oral Biol Med 10(3):276–283
pubmed: 10759409
doi: 10.1177/10454411990100030201
Baum BJ, Alevizos I, Chiorini JA, Cotrim AP, Zheng C (2015) Advances in salivary gland gene therapy – oral and systemic implications. Expert Opin Biol Ther 15(10):1443–1454
pubmed: 26149284
pmcid: 4823768
doi: 10.1517/14712598.2015.1064894
Baum BJ, Afione S, Chiorini JA, Cotrim AP, Goldsmith CM, Zheng C (2017) Gene therapy of salivary diseases. In: Seymour GJ, Cullinan MP, Heng NC (eds) Oral biology: molecular techniques and applications. Springer, New York, pp 107–123
doi: 10.1007/978-1-4939-6685-1_6
Baylis F, Ikemoto L (2017) The Council of Europe and the prohibition on human germline genome editing. EMBO Rep 18(12):2084–2085
pubmed: 29141984
pmcid: 5709742
doi: 10.15252/embr.201745343
Blum S, Shapir N, Miari R, Lerner B, Koren B, Doenyas-Barak K et al (2017) TARGT gene therapy platform for correction of anemia in end-stage renal disease. N Engl J Med 376(2):189–191
pubmed: 28076704
doi: 10.1056/NEJMc1606202
Bovolenta C, Porcellini S, Alberici L (2013) Therapeutic genes for anti-HIV/AIDS gene therapy. Curr Pharm Biotechnol 14(5):488–500
pubmed: 22429132
doi: 10.2174/138920101405131111104009
Brendel C, Negre O, Rothe M, Guda S, Parsons G, Harris C et al (2020) Preclinical evaluation of a novel Lentiviral vector driving lineage-specific BCL11A knockdown for sickle cell gene therapy. Mol Ther Methods Clin Dev 17:589–600
pubmed: 32300607
pmcid: 7150438
doi: 10.1016/j.omtm.2020.03.015
Chen Y, Zheng Y, Kang Y, Yang W, Niu Y, Guo X et al (2015) Functional disruption of the dystrophin gene in rhesus monkey using CRISPR/Cas9. Hum Mol Genet 24(13):3764–3774
pubmed: 25859012
pmcid: 5007610
doi: 10.1093/hmg/ddv120
Chen XZ, Guo R, Zhao C, Xu J, Song H, Yu H et al (2022) A novel anti-cancer therapy: CRISPR/Cas9 gene editing. Front Pharmacol 13:939090
pubmed: 35935840
pmcid: 9353945
doi: 10.3389/fphar.2022.939090
Convertine AJ, Diab C, Prieve M, Paschal A, Hoffman AS, Johnson PH et al (2010) pH-responsive polymeric micelle carriers for siRNA drugs. Biomacromolecules 11(11):2904–2911
pubmed: 20886830
pmcid: 3026907
doi: 10.1021/bm100652w
Corden A, Handelman B, Yin H, Cotrim A, Alevizos I, Chiorini JA (2017) Neutralizing antibodies against adeno-associated viruses in Sjögren’s patients: implications for gene therapy. Gene Ther 24(4):241–244
pubmed: 28150697
pmcid: 5810933
doi: 10.1038/gt.2017.1
Cornel MC, Howard HC, Lim D, Bonham VL, Wartiovaara K (2019) Moving towards a cure in genetics: what is needed to bring somatic gene therapy to the clinic? Eur J Hum Genet 27(3):484–487
pubmed: 30568241
doi: 10.1038/s41431-018-0309-x
Di Pasquale G, Perez Riveros P, Tora M, Sheikh T, Son A, Teos L et al (2020) Transduction of salivary gland acinar cells with a novel AAV vector 44.9. Mol Ther – Methods Clin Dev 19:459–466
pubmed: 33294494
pmcid: 7689275
doi: 10.1016/j.omtm.2020.10.006
Dong C, Xiong J, Ni J, Fang X, Zhang J, Zhu D et al (2022) Intracellular miRNA-triggered surface-enhanced Raman scattering imaging and dual gene-silencing therapy of cancer cell. Anal Chem 94(26):9336–9344
pubmed: 35728270
doi: 10.1021/acs.analchem.2c00842
Doxzen K, Halpern J (2020) Focusing on Human Rights: a framework for CRISPR germline genome editing ethics and regulation. Perspect Biol Med 63(1):44–53
pubmed: 32063585
doi: 10.1353/pbm.2020.0003
El Helou G, Goodman JF, Blevins M, Caudell DL, Ponzio TA, Sanders JW (2021) Retrograde parotid gland infusion through Stensen’s duct in a non-human primate for vectored gene delivery. JoVE 174:e62645
Ferreira JNA, Zheng C, Lombaert IMA, Goldsmith CM, Cotrim AP, Symonds JM et al (2018) Neurturin gene therapy protects parasympathetic function to prevent irradiation-induced murine salivary gland hypofunction. Mol Ther Methods Clin Dev 9:172–180
pubmed: 29560384
pmcid: 5857485
doi: 10.1016/j.omtm.2018.02.008
Flotte TR, Gao G (2021) Immune responses to recombinant adenoviruses as gene therapy vectors and COVID-19 vaccines: a two-edged sword. Hum Gene Ther 32(13–14):645–646
pubmed: 34283643
doi: 10.1089/hum.2021.29169.trf
Fox JL (2002) Eugenics concerns rekindle with application of gene therapy and genetic counseling. Nat Biotechnol 20(6):531–532
pubmed: 12042840
doi: 10.1038/nbt0602-531b
Fukumura M, Ishibashi K, Nakaguro M, Nagao T, Saida K, Urano M et al (2022) Salivary gland polymorphous adenocarcinoma: Clinicopathological features and gene alterations in 36 Japanese patients. J Oral Pathol Med 51(8):710–720
pubmed: 35880805
doi: 10.1111/jop.13336
Geguchadze RN, Machen L, Zourelias L, Gallo PH, Passineau MJ (2012) An AAV2/5 vector enhances safety of gene transfer to the mouse salivary gland. J Dent Res 91(4):382–386
pubmed: 22307036
pmcid: 3310756
doi: 10.1177/0022034512437373
Geguchadze R, Wang Z, Zourelias L, Perez-Riveros P, Edwards PC, Machen L et al (2014) Proteomic profiling of salivary gland after nonviral gene transfer mediated by conventional plasmids and minicircles. Mol Ther Methods Clin Dev 1:14007
pubmed: 25414909
pmcid: 4236002
doi: 10.1038/mtm.2014.7
Guo L, Gao R, Xu J, Jin L, Cotrim AP, Yan X et al (2014) AdLTR2EF1α-FGF2-mediated prevention of fractionated irradiation-induced salivary hypofunction in swine. Gene Ther 21(10):866–873
pubmed: 25030610
doi: 10.1038/gt.2014.63
Hai B, Qin L, Yang Z, Zhao Q, Shangguan L, Ti X et al (2014) Transient activation of hedgehog pathway rescued irradiation-induced hyposalivation by preserving salivary stem/progenitor cells and parasympathetic innervation. Clin Cancer Res 20(1):140–150
pubmed: 24150232
doi: 10.1158/1078-0432.CCR-13-1434
Hai B, Zhao Q, Qin L, Rangaraj D, Gutti VR, Liu F (2016) Rescue effects and underlying mechanisms of Intragland Shh gene delivery on irradiation-induced hyposalivation. Hum Gene Ther 27(5):390–399
pubmed: 27021743
pmcid: 4841000
doi: 10.1089/hum.2016.005
Hai B, Zhao Q, Deveau MA, Liu F (2018) Delivery of sonic hedgehog gene repressed irradiation-induced cellular senescence in salivary glands by promoting DNA repair and reducing oxidative stress. Theranostics 8(4):1159–1167
pubmed: 29464006
pmcid: 5817117
doi: 10.7150/thno.23373
Harford JB, Kim SS, Pirollo KF, Chang EH (2022) TP53 gene therapy as a potential treatment for patients with COVID-19. Viruses 14(4):739
pubmed: 35458469
pmcid: 9027273
doi: 10.3390/v14040739
Herold BC, Marcellino D, Marcelin G, Wilson P, Burrow C, Satlin LM (2002) Herpes simplex virus as a model vector system for gene therapy in renal disease. Kidney Int 61(Suppl 1):S3–S8
pubmed: 11841605
doi: 10.1046/j.1523-1755.2002.0610s1003.x
Hsieh MM, Bonner M, Pierciey FJ, Uchida N, Rottman J, Demopoulos L et al (2020) Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease. Blood Adv 4(9):2058–2063
pubmed: 32396618
pmcid: 7218414
doi: 10.1182/bloodadvances.2019001330
Hu L, Zhu Z, Hai B, Chang S, Ma L, Xu Y et al (2018) Intragland Shh gene delivery mitigated irradiation-induced hyposalivation in a miniature pig model. Theranostics 8(16):4321–4331
pubmed: 30214623
pmcid: 6134926
doi: 10.7150/thno.26509
Huai C, Jia C, Sun R, Xu P, Min T, Wang Q et al (2017) CRISPR/Cas9-mediated somatic and germline gene correction to restore hemostasis in hemophilia B mice. Hum Genet 136(7):875–883
pubmed: 28508290
doi: 10.1007/s00439-017-1801-z
Hutt JA, Assaf BT, Bolon B, Cavagnaro J, Galbreath E, Grubor B et al (2022) Scientific and regulatory policy committee points to consider: nonclinical research and development of in vivo gene therapy products, emphasizing adeno-associated virus vectors. Toxicol Pathol 50(1):118–146
pubmed: 34657529
doi: 10.1177/01926233211041962
Iacobas S, Iacobas DA (2022) Personalized 3-gene panel for prostate cancer target therapy. Curr Issues Mol Biol 44(1):360–382
pubmed: 35723406
pmcid: 8929157
doi: 10.3390/cimb44010027
Kim YJ, Nomakuchi T, Papaleonidopoulou F, Yang L, Zhang Q, Krainer AR (2022) Gene-specific nonsense-mediated mRNA decay targeting for cystic fibrosis therapy. Nat Commun 13(1):2978
pubmed: 35624092
pmcid: 9142507
doi: 10.1038/s41467-022-30668-y
Lai Z, Yin H, Cabrera-Pérez J, Guimaro MC, Afione S, Michael DG et al (2016) Aquaporin gene therapy corrects Sjögren’s syndrome phenotype in mice. Proc Natl Acad Sci U S A 113(20):5694–5699
pubmed: 27140635
pmcid: 4878512
doi: 10.1073/pnas.1601992113
Liu XY, Zhang X, Yang JB, Wu CY, Wang Q, Lu ZL et al (2022) Multifunctional amphiphilic peptide dendrimer as nonviral gene vectors for effective cancer therapy via combined gene/photodynamic therapies. Colloids Surf B Biointerfaces 217:112651
pubmed: 35759892
doi: 10.1016/j.colsurfb.2022.112651
Lombaert IMA, Patel VN, Jones CE, Villier DC, Canada AE, Moore MR et al (2020) CERE-120 prevents irradiation-induced Hypofunction and restores immune homeostasis in porcine salivary glands. Mol Ther Methods Clin Dev 18:839–855
pubmed: 32953934
pmcid: 7479444
doi: 10.1016/j.omtm.2020.07.016
Malcolm DW, Varghese JJ, Sorrells JE, Ovitt CE, Benoit DSW (2018) The effects of biological fluids on colloidal stability and siRNA delivery of a pH-responsive micellar nanoparticle delivery system. ACS Nano 12(1):187–197
pubmed: 29232104
doi: 10.1021/acsnano.7b05528
Marquez Loza LI, Cooney AL, Dong Q, Randak CO, Rivella S, Sinn PL et al (2021) Increased CFTR expression and function from an optimized lentiviral vector for cystic fibrosis gene therapy. Mol Ther Methods Clin Dev 21:94–106
pubmed: 33768133
pmcid: 7973238
doi: 10.1016/j.omtm.2021.02.020
Maruyama S (2006) Gene therapy for interstitial renal disease. Nihon Rinsho 64(Suppl 2):662–666
pubmed: 16523973
Mastrangeli A, O’Connell B, Aladib W, Fox PC, Baum BJ, Crystal RG (1994) Direct in vivo adenovirus-mediated gene transfer to salivary glands. Am J Phys 266(6 Pt 1):G1146–G1155
Nakagami H (2021) Development of COVID-19 vaccines utilizing gene therapy technology. Int Immunol 33(10):521–527
pubmed: 33772572
doi: 10.1093/intimm/dxab013
Nguyen CQ, Yin H, Lee BH, Chiorini JA, Peck AB (2011) IL17: potential therapeutic target in Sjögren’s syndrome using adenovirus-mediated gene transfer. Lab Investig 91(1):54–62
pubmed: 20856230
doi: 10.1038/labinvest.2010.164
Oishi T, Ito M, Koizumi S, Horikawa M, Yamamoto T, Yamagishi S et al (2022) Efficacy of HSV-TK/GCV system suicide gene therapy using SHED expressing modified HSV-TK against lung cancer brain metastases. Mol Ther Methods Clin Dev 26:253–265
pubmed: 35892087
pmcid: 9307584
doi: 10.1016/j.omtm.2022.07.001
Passineau M (2017) Salivary gland gene therapy in experimental and clinical trials. In: Cha S (ed) Salivary gland development and regeneration: advances in research and clinical approaches to functional restoration. Springer International Publishing, Cham, pp 217–228
doi: 10.1007/978-3-319-43513-8_12
Passineau MJ, Zourelias L, Machen L, Edwards PC, Benza RL (2010) Ultrasound-assisted non-viral gene transfer to the salivary glands. Gene Ther 17(11):1318–1324
pubmed: 20508599
doi: 10.1038/gt.2010.86
Qadir MI, Zafar M (2017) TSG101: tumor susceptibility gene 101 (tsg101) product-role in therapy against HIV/AIDS. Crit Rev Eukaryot Gene Expr 27(3):237–246
pubmed: 29199609
doi: 10.1615/CritRevEukaryotGeneExpr.2017019418
Racz G, Zheng C, Goldsmith C, Baum B, Cawley N (2015) Toward gene therapy for growth hormone deficiency via salivary gland expression of growth hormone. Oral Dis 21(2):149–155
pubmed: 24320050
doi: 10.1111/odi.12217
Reyne N, Cmielewski P, McCarron A, Delhove J, Parsons D, Donnelley M (2021) Single-dose Lentiviral mediated gene therapy recovers CFTR function in cystic fibrosis knockout rats. Front Pharmacol 12:682299
pubmed: 34084147
pmcid: 8167067
doi: 10.3389/fphar.2021.682299
Rocha EM, Cotrim AP, Zheng C, Riveros PP, Baum BJ, Chiorini JA (2013) Recovery of radiation-induced dry eye and corneal damage by Pretreatment with adenoviral vector-mediated transfer of erythropoietin to the salivary glands in mice. Hum Gene Ther 24(4):417–423
pubmed: 23402345
pmcid: 3631015
doi: 10.1089/hum.2012.111
Rowzee AM, Perez-Riveros PJ, Zheng C, Krygowski S, Baum BJ, Cawley NX (2013) Expression and secretion of human proinsulin-B10 from mouse salivary glands: implications for the treatment of type I diabetes mellitus. PLoS One 8(3):e59222
pubmed: 23554999
pmcid: 3598661
doi: 10.1371/journal.pone.0059222
Samuni Y, Baum BJ (2011) Gene delivery in salivary glands: from the bench to the clinic. Biochim Biophys Acta (BBA) – Mol Basis Dis 1812(11):1515–1521
doi: 10.1016/j.bbadis.2011.06.014
Samuni Y, Zheng C, Cawley NX, Cotrim AP, Loh YP, Baum BJ (2008) Sorting of growth hormone-erythropoietin fusion proteins in rat salivary glands. Biochem Biophys Res Commun 373(1):136–139
pubmed: 18544341
pmcid: 2525869
doi: 10.1016/j.bbrc.2008.05.177
Shai E, Falk H, Honigman A, Panet A, Palmon A (2002) Gene transfer mediated by different viral vectors following direct cannulation of mouse submandibular salivary glands. Eur J Oral Sci 110(3):254–260
pubmed: 12120712
doi: 10.1034/j.1600-0722.2002.21200.x
Stenman G, Fehr A, Skalova A, Vander Poorten V, Hellquist H, Mikkelsen LH et al (2022) Chromosome translocations, gene fusions, and their molecular consequences in pleomorphic salivary gland adenomas. Biomedicine 10(8):1970
Stone EF, Avecilla ST, Wuest DL, Lomas-Francis C, Westhoff CM, Diuguid DL et al (2021) Severe delayed hemolytic transfusion reaction due to anti-Fy3 in a patient with sickle cell disease undergoing red cell exchange prior to hematopoietic progenitor cell collection for gene therapy. Haematologica 106(1):310–312
pubmed: 32817291
doi: 10.3324/haematol.2020.253229
Teos LY, Zheng CY, Liu X, Swaim WD, Goldsmith CM, Cotrim AP et al (2016) Adenovirus-mediated hAQP1 expression in irradiated mouse salivary glands causes recovery of saliva secretion by enhancing acinar cell volume decrease. Gene Ther 23(7):572–579
pubmed: 26966862
pmcid: 5798877
doi: 10.1038/gt.2016.29
Timiri Shanmugam PS, Dayton RD, Palaniyandi S, Abreo F, Caldito G, Klein RL et al (2013) Recombinant AAV9-TLK1B administration ameliorates fractionated radiation-induced xerostomia. Hum Gene Ther 24(6):604–612
pubmed: 23614651
doi: 10.1089/hum.2012.235
Vitolo J, Baum B (2002) The use of gene transfer for the protection and repair of salivary glands. Oral Dis 8(4):183–191
pubmed: 12206399
doi: 10.1034/j.1601-0825.2002.02865.x
Voutetakis A, Kok MR, Zheng C, Bossis I, Wang J, Cotrim AP et al (2004) Reengineered salivary glands are stable endogenous bioreactors for systemic gene therapeutics. Proc Natl Acad Sci USA 101(9):3053–3058
pubmed: 14978265
pmcid: 365743
doi: 10.1073/pnas.0400136101
Wang J, Cawley NX, Voutetakis A, Rodriguez YM, Goldsmith CM, Nieman LK et al (2005) Partial redirection of transgenic human growth hormone secretion from rat salivary glands. Hum Gene Ther 16(5):571–583
pubmed: 15916482
doi: 10.1089/hum.2005.16.571
Wang J, Wang F, Xu J, Ding S, Guo Y (2014) Double-strand adeno-associated virus-mediated exendin-4 expression in salivary glands is efficient in a diabetic rat model. Diabetes Res Clin Pract 103(3):466–473
pubmed: 24438876
doi: 10.1016/j.diabres.2013.12.006
Wang Z, Zourelias L, Wu C, Edwards PC, Trombetta M, Passineau MJ (2015a) Ultrasound-assisted nonviral gene transfer of AQP1 to the irradiated minipig parotid gland restores fluid secretion. Gene Ther 22(9):739–749
pubmed: 25871828
pmcid: 4560616
doi: 10.1038/gt.2015.36
Wang J, Wen J, Bai D, Guo Y (2015b) Injection of submandibular gland with recombinant Exendin-4 and adeno-associated virus for the treatment of diabetic rats. Zhong Nan Da Xue Xue Bao Yi Xue Ban 40(11):1179–1185
pubmed: 26643419
Wang Z, Benza RL, Zourelias L, Sanguino A, Geguchadze R, Shields KJ et al (2017a) In vivo endocrine secretion of prostacyclin following expression of a Cyclooxygenase-1/prostacyclin fusion protein in the salivary glands of rats via nonviral gene therapy. Hum Gene Ther 28(8):681–689
pubmed: 28530128
pmcid: 5567413
doi: 10.1089/hum.2017.040
Wang Z, Pradhan-Bhatt S, Farach-Carson MC, Passineau MJ (2017b) Artificial induction of native Aquaporin-1 expression in human salivary cells. J Dent Res 96(4):444–449
pubmed: 28072927
pmcid: 5384490
doi: 10.1177/0022034516685045
Wu C, Wang Z, Zourelias L, Thakker H, Passineau MJ (2015) IL-17 sequestration via salivary gland gene therapy in a mouse model of Sjogren’s syndrome suppresses disease-associated expression of the putative autoantigen Klk1b22. Arthritis Res Ther 17(1):198
pubmed: 26245278
pmcid: 4527205
doi: 10.1186/s13075-015-0714-2
Xia E, Zhang Y, Cao H, Li J, Duan R, Hu J (2019) TALEN-mediated gene targeting for cystic fibrosis-gene therapy. Genes (Basel) 10(1):39
pubmed: 30641980
doi: 10.3390/genes10010039
Xiao Q, Guo D, Chen S (2019) Application of CRISPR/Cas9-based gene editing in HIV-1/AIDS therapy. Front Cell Infect Microbiol 9:69
pubmed: 30968001
pmcid: 6439341
doi: 10.3389/fcimb.2019.00069
Yang Q, Soltis AR, Sukumar G, Zhang X, Caohuy H, Freedy J et al (2019) Gene therapy-emulating small molecule treatments in cystic fibrosis airway epithelial cells and patients. Respir Res 20(1):290
pubmed: 31864360
pmcid: 6925517
doi: 10.1186/s12931-019-1214-8
Yuan L, Sui T, Chen M, Deng J, Huang Y, Zeng J et al (2016) CRISPR/Cas9-mediated GJA8 knockout in rabbits recapitulates human congenital cataracts. Sci Rep 6:22024
pubmed: 26912477
pmcid: 4766569
doi: 10.1038/srep22024
Zaidane I, Ouladlahsen A, Bensghir R, Chihab H, Jadid FZ, Fihry RE et al (2020) An assessment of toll-like receptor 7 and 8 gene polymorphisms with susceptibility to HIV-1 infection, AIDS development and response to antiretroviral therapy. Immunol Lett 227:88–95
pubmed: 32888973
doi: 10.1016/j.imlet.2020.08.008
Zeng M, Szymczak M, Ahuja M, Zheng C, Yin H, Swaim W et al (2017) Correction of ductal CFTR activity rescues acinar cell and pancreatic and salivary gland functions in mouse models of autoimmune disease. Gastroenterology 153(4):1148–1159
pubmed: 28634110
doi: 10.1053/j.gastro.2017.06.011
Zhang Z, Hou W, Chen S (2022) Updates on CRISPR-based gene editing in HIV-1/AIDS therapy. Virol Sin 37(1):1–10
pubmed: 35234622
pmcid: 8922418
doi: 10.1016/j.virs.2022.01.017
Zheng C, Vitolo JM, Zhang W, Mineshiba F, Chiorini JA, Baum BJ (2008a) Extended transgene expression from a nonintegrating adenoviral vector containing retroviral elements. Mol Ther 16(6):1089–1097
pubmed: 18388914
doi: 10.1038/mt.2008.56
Zheng C, Vitolo JM, Zhang W, Mineshiba F, Chiorini JA, Baum BJ (2008b) Extended transgene expression from a nonintegrating adenoviral vector containing retroviral elements. Mol Thery 16(6):1089–1097
doi: 10.1038/mt.2008.56
Zheng C, Cotrim AP, Sunshine AN, Sugito T, Liu L, Sowers A et al (2009) Prevention of radiation-induced oral mucositis after adenoviral vector-mediated transfer of the keratinocyte growth factor cDNA to mouse submandibular glands. Clin Cancer Res 15(14):4641–4648
pubmed: 19584147
pmcid: 2769927
doi: 10.1158/1078-0432.CCR-09-0819
Zheng C, Cotrim AP, Nikolov N, Mineshiba F, Swaim W, Baum BJ (2012) A novel hybrid Adenoretroviral vector with more extensive E3 deletion extends transgene expression in submandibular glands. Hum Gene Ther Methods 23(3):169–181
pubmed: 22817829
pmcid: 4015066
doi: 10.1089/hgtb.2011.175
Zheng C, Baum BJ, Liu X, Goldsmith CM, Perez P, Jang SI et al (2015) Persistence of hAQP1 expression in human salivary gland cells following AdhAQP1 transduction is associated with a lack of methylation of hCMV promoter. Gene Ther 22(9):758–766
pubmed: 26177970
pmcid: 5798873
doi: 10.1038/gt.2015.55
Zubler RH (2007) Ex vivo expansion of haematopoietic stem cells and gene therapy development. Swiss Med Wkly 137(Suppl 155):31S–35S
pubmed: 17874498
Zufferey R, Aebischer P (2004) Salivary glands and gene therapy: the mouth waters. Gene Ther 11(19):1425–1426
pubmed: 15269715
doi: 10.1038/sj.gt.3302321