Airway basal cells from human-induced pluripotent stem cells: a new frontier in cystic fibrosis research

被引:2
作者
Demchenko, Anna [1 ]
Belova, Lyubava [1 ]
Balyasin, Maxim [2 ,3 ]
Kochergin-Nikitsky, Konstantin [1 ]
Kondrateva, Ekaterina [1 ]
Voronina, Ekaterina [4 ]
Pozhitnova, Victoria [4 ]
Tabakov, Vyacheslav [5 ]
Salikhova, Diana [6 ]
Bukharova, Tatiana [6 ]
Goldshtein, Dmitry [6 ]
Kondratyeva, Elena [7 ]
Kyian, Tatiana [7 ]
Amelina, Elena [8 ]
Zubkova, Olga [9 ]
Popova, Olga [9 ]
Ozharovskaia, Tatiana [9 ]
Lavrov, Alexander [1 ]
Smirnikhina, Svetlana [1 ]
机构
[1] Res Ctr Med Genet, Lab Genome Editing, Moscow, Russia
[2] Peoples Friendship Univ Russia, Sci & Educ Resource Ctr, Moscow, Russia
[3] Endocrinol Res Ctr, Dept Cell Technol, Moscow, Russia
[4] Res Ctr Med Genet, Lab Mutagenesis, Moscow, Russia
[5] Res Ctr Med Genet, Moscow Branch Biobank All Russian Collect Biol Sam, Moscow, Russia
[6] Res Ctr Med Genet, Stem Cell Genet Lab, Moscow, Russia
[7] Res Ctr Med Genet, Sci & Clin Dept Cyst Fibrosis, Moscow, Russia
[8] Res Inst Pulmonol, Lab Cyst Fibrosis, Moscow, Russia
[9] Minist Hlth Russian Federat, Dept Sociol & Polit Sci, Fed State Budget Inst, Natl Res Ctr Epidemiol & Microbiol Named Honorary, Moscow, Russia
来源
FRONTIERS IN CELL AND DEVELOPMENTAL BIOLOGY | 2024年 / 12卷
关键词
airway basal cells; disease modeling; induced pluripotent stem cells; differentiation; cystic fibrosis; cystic fibrosis transmembrane conductance regulator; gene therapy; viral vectors; LINES;
D O I
10.3389/fcell.2024.1336392
中图分类号
Q2 [细胞生物学];
学科分类号
071009 ; 090102 ;
摘要
Human-induced airway basal cells (hiBCs) derived from human-induced pluripotent stem cells (hiPSCs) offer a promising cell model for studying lung diseases, regenerative medicine, and developing new gene therapy methods. We analyzed existing differentiation protocols and proposed our own protocol for obtaining hiBCs, which involves step-by-step differentiation of hiPSCs into definitive endoderm, anterior foregut endoderm, NKX2.1+ lung progenitors, and cultivation on basal cell medium with subsequent cell sorting using the surface marker CD271 (NGFR). We derived hiBCs from two healthy cell lines and three cell lines with cystic fibrosis (CF). The obtained hiBCs, expressing basal cell markers (NGFR, KRT5, and TP63), could differentiate into lung organoids (LOs). We demonstrated that LOs derived from hiBCs can assess cystic fibrosis transmembrane conductance regulator (CFTR) channel function using the forskolin-induced swelling (FIS) assay. We also carried out non-viral (electroporation) and viral (recombinant adeno-associated virus (rAAV)) serotypes 6 and 9 and recombinant adenovirus (rAdV) serotype 5 transgene delivery to hiBCs and showed that rAAV serotype 6 is most effective against hiBCs, potentially applicable for gene therapy research.
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页数:11
相关论文
共 37 条
[1]   Recombinant Adeno-associated Viral Vectors Serotypes 6 and 9 are Able to Transduce Human Tracheal Epithelial Cells but Not Human Induced Pluripotent Stem Cells [J].
Belova, L. ;
Demchenko, A. ;
Kochergin-Nikitsky, K. ;
Kondrateva, E. ;
Slesarenko, Ya. ;
Salikhova, D. ;
Lavrov, A. ;
Efremova, A. ;
Bukharova, T. ;
Goldshtein, D. ;
Smirnikhina, S. .
MOLECULAR BIOTECHNOLOGY, 2023, 65 (09) :1539-1546
[2]   ilastik: interactive machine learning for (bio) image analysis [J].
Berg, Stuart ;
Kutra, Dominik ;
Kroeger, Thorben ;
Straehle, Christoph N. ;
Kausler, Bernhard X. ;
Haubold, Carsten ;
Schiegg, Martin ;
Ales, Janez ;
Beier, Thorsten ;
Rudy, Markus ;
Eren, Kemal ;
Cervantes, Jaime I. ;
Xu, Buote ;
Beuttenmueller, Fynn ;
Wolny, Adrian ;
Zhang, Chong ;
Koethe, Ullrich ;
Hamprecht, Fred A. ;
Kreshuk, Anna .
NATURE METHODS, 2019, 16 (12) :1226-1232
[3]   CellProfiler: image analysis software for identifying and quantifying cell phenotypes [J].
Carpenter, Anne E. ;
Jones, Thouis Ray ;
Lamprecht, Michael R. ;
Clarke, Colin ;
Kang, In Han ;
Friman, Ola ;
Guertin, David A. ;
Chang, Joo Han ;
Lindquist, Robert A. ;
Moffat, Jason ;
Golland, Polina ;
Sabatini, David M. .
GENOME BIOLOGY, 2006, 7 (10)
[4]   CFTR localization in native airway cells and cell lines expressing wild-type or F508del-CFTR by a panel of different antibodies [J].
Carvalho-Oliveira, I ;
Efthymiadou, A ;
Malhó, R ;
Nogueira, P ;
Tzetis, M ;
Kanavakis, E ;
Amaral, MD ;
Penque, D .
JOURNAL OF HISTOCHEMISTRY & CYTOCHEMISTRY, 2004, 52 (02) :193-203
[5]   CFTR modulator theratyping: Current status, gaps and future directions [J].
Clancy, John Paul ;
Cotton, Calvin U. ;
Donaldson, Scott H. ;
Solomon, George M. ;
VanDevanter, Donald R. ;
Boyle, Michael P. ;
Gentzsch, Martina ;
Nick, Jerry A. ;
Illek, Beate ;
Wallenburg, John C. ;
Sorscher, Eric J. ;
Amaral, Margarida D. ;
Beekman, Jeffrey M. ;
Naren, Anjaparavanda P. ;
Bridges, Robert J. ;
Thomas, Philip J. ;
Cutting, Garry ;
Rowe, Steven ;
Durmowicz, Anthony G. ;
Mense, Martin ;
Boeck, Kris D. ;
Skach, William ;
Penland, Christopher ;
Joseloff, Elizabeth ;
Bihler, Hermann ;
Mahoney, John ;
Borowitz, Drucy ;
Tuggle, Katherine L. .
JOURNAL OF CYSTIC FIBROSIS, 2019, 18 (01) :22-34
[6]   High-resolution 3D imaging of fixed and cleared organoids [J].
Dekkers, Johanna F. ;
Alieva, Maria ;
Wellens, Lianne M. ;
Ariese, Hendrikus C. R. ;
Jamieson, Paul R. ;
Vonk, Annelotte M. ;
Amatngalim, Gimano D. ;
Hu, Huili ;
Oost, Koen C. ;
Snippert, Hugo J. G. ;
Beekman, Jeffrey M. ;
Wehrens, Ellen J. ;
Visvader, Jane E. ;
Clevers, Hans ;
Rios, Anne C. .
NATURE PROTOCOLS, 2019, 14 (06) :1756-1771
[7]   A functional CFTR assay using primary cystic fibrosis intestinal organoids [J].
Dekkers, Johanna F. ;
Wiegerinck, Caroline L. ;
de Jonge, Hugo R. ;
Bronsveld, Inez ;
Janssens, Hettie M. ;
de Winter-de Groot, Karin M. ;
Brandsma, Arianne M. ;
de Jong, Nienke W. M. ;
Bijvelds, Marcel J. C. ;
Scholte, Bob J. ;
Nieuwenhuis, Edward E. S. ;
van den Brink, Stieneke ;
Clevers, Hans ;
van der Ent, Cornelis K. ;
Middendorp, Sabine ;
Beekman, Jeffrey M. .
NATURE MEDICINE, 2013, 19 (07) :939-+
[8]   Airway and Lung Organoids from Human-Induced Pluripotent Stem Cells Can Be Used to Assess CFTR Conductance [J].
Demchenko, Anna ;
Kondrateva, Ekaterina ;
Tabakov, Vyacheslav ;
Efremova, Anna ;
Salikhova, Diana ;
Bukharova, Tatiana ;
Goldshtein, Dmitry ;
Balyasin, Maxim ;
Bulatenko, Natalia ;
Amelina, Elena ;
Lavrov, Alexander ;
Smirnikhina, Svetlana .
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2023, 24 (07)
[9]   SARS-CoV-2 infects an upper airway model derived from induced pluripotent stem cells [J].
Djidrovksi, Ivo ;
Georgiou, Maria ;
Hughes, Grant L. ;
Patterson, Edward I. ;
Casas-Sanchez, Aitor ;
Penington, Shaun H. ;
Biagini, Giancarlo A. ;
Moya-Molina, Marina ;
van den Bor, Jelle ;
Smit, Martine J. ;
Chung, Git ;
Lako, Majlinda ;
Armstrong, Lyle .
STEM CELLS, 2021, 39 (10) :1310-1321
[10]   An Adeno-Associated Viral Vector Capable of Penetrating the Mucus Barrier to Inhaled Gene Therapy [J].
Duncan, Gregg A. ;
Kim, Namho ;
Colon-Cortes, Yanerys ;
Rodriguez, Jason ;
Mazur, Marina ;
Birket, Susan E. ;
Rowe, Steven M. ;
West, Natalie E. ;
Livraghi-Butrico, Alessandra ;
Boucher, Richard C. ;
Hanes, Justin ;
Aslanidi, George ;
Suk, Jung Soo .
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2018, 9 :296-304