Targeted CRISPR activation is functional in engineered human pluripotent stem cells but undergoes silencing after differentiation into cardiomyocytes and endothelium

Adli M (2018) The CRISPR tool kit for genome editing and beyond. Nat Commun 9:1911. https://doi.org/10.1038/s41467-018-04252-2

Article  ADS  CAS  PubMed  PubMed Central  Google Scholar 

Alhaji SY, Ngai SC, Abdullah S (2019) Silencing of transgene expression in mammalian cells by DNA methylation and histone modifications in gene therapy perspective. Biotechnol Genet Eng Rev 35:1–25. https://doi.org/10.1080/02648725.2018.1551594

Article  CAS  PubMed  Google Scholar 

An CI, Ichihashi Y, Peng J, Sinha NR, Hagiwara N (2016) Transcriptome Dynamics and potential roles of Sox6 in the postnatal heart. PLoS One 11:e0166574. https://doi.org/10.1371/journal.pone.0166574

Article  CAS  PubMed  PubMed Central  Google Scholar 

Atlasi Y, Stunnenberg HG (2017) The interplay of epigenetic marks during stem cell differentiation and development. Nat Rev Genet 18:643–658. https://doi.org/10.1038/nrg.2017.57

Article  CAS  PubMed  Google Scholar 

Balmas E, Sozza F, Bottini S, Ratto ML, Savore G, Becca S, Snijders KE, Bertero A (2023) Manipulating and studying gene function in human pluripotent stem cell models. FEBS Lett. https://doi.org/10.1002/1873-3468.14709

Article  PubMed  Google Scholar 

Battulin N, Korablev A, Ryzhkova A, Smirnov A, Kabirova E, Khabarova A, Lagunov T, Serova I, Serov O (2022) The human EF1a promoter does not provide expression of the transgene in mice. Transgenic Res 31:525–535. https://doi.org/10.1007/s11248-022-00319-5

Article  CAS  PubMed  PubMed Central  Google Scholar 

Bertero A, Pawlowski M, Ortmann D, Snijders K, Yiangou L, Cardoso de Brito M, Brown S, Bernard WG, Cooper JD, Giacomelli E, Gambardella L, Hannan NR, Iyer D, Sampaziotis F, Serrano F, Zonneveld MC, Sinha S, Kotter M, Vallier L (2016) Optimized inducible shRNA and CRISPR/Cas9 platforms for in vitro studies of human development using hPSCs. Development 143:4405–4418. https://doi.org/10.1242/dev.138081

Article  CAS  PubMed  PubMed Central  Google Scholar 

Bertero A, Yiangou L, Brown S, Ortmann D, Pawlowski M, Vallier L (2018) Conditional manipulation of gene function in human cells with optimized inducible shRNA. Curr Protoc Stem Cell Biol 44:5C41–5C448. https://doi.org/10.1002/cpsc.45

Article  CAS  Google Scholar 

Bertero A, Fields PA, Ramani V, Bonora G, Yardimci GG, Reinecke H, Pabon L, Noble WS, Shendure J, Murry CE (2019) Dynamics of genome reorganization during human cardiogenesis reveal an RBM20-dependent splicing factory. Nat Commun 10:1538. https://doi.org/10.1038/s41467-019-09483-5

Article  ADS  CAS  PubMed  PubMed Central  Google Scholar 

Bhagwan JR, Collins E, Mosqueira D, Bakar M, Johnson BB, Thompson A, Smith JGW, Denning C (2019) Variable expression and silencing of CRISPR-Cas9 targeted transgenes identifies the AAVS1 locus as not an entirely safe harbour. F1000Res 8:1911. https://doi.org/10.12688/f1000research.19894.2

Article  CAS  PubMed  Google Scholar 

Black JB, McCutcheon SR, Dube S, Barrera A, Klann TS, Rice GA, Adkar SS, Soderling SH, Reddy TE, Gersbach CA (2020) Master regulators and cofactors of human neuronal cell fate specification identified by CRISPR Gene Activation screens. Cell Rep 33:108460. https://doi.org/10.1016/j.celrep.2020.108460

Article  CAS  PubMed  PubMed Central  Google Scholar 

Burridge PW, Holmstrom A, Wu JC (2015) Chemically defined culture and cardiomyocyte differentiation of human pluripotent stem cells. Curr Protoc Hum Genet 87:2131–21315. https://doi.org/10.1002/0471142905.hg2103s87

Article  Google Scholar 

Cabrera A, Edelstein HI, Glykofrydis F, Love KS, Palacios S, Tycko J, Zhang M, Lensch S, Shields CE, Livingston M, Weiss R, Zhao H, Haynes KA, Morsut L, Chen YY, Khalil AS, Wong WW, Collins JJ, Rosser SJ, Polizzi K, Elowitz MB, Fussenegger M, Hilton IB, Leonard JN, Bintu L, Galloway KE, Deans TL (2022) The sound of silence: transgene silencing in mammalian cell engineering. Cell Syst 13:950–973. https://doi.org/10.1016/j.cels.2022.11.005

Article  CAS  PubMed  Google Scholar 

Casas-Mollano JA, Zinselmeier MH, Erickson SE, Smanski MJ (2020) CRISPR-Cas activators for Engineering Gene expression in higher eukaryotes. CRISPR J 3:350–364. https://doi.org/10.1089/crispr.2020.0064

Article  CAS  PubMed  PubMed Central  Google Scholar 

Cerbini T, Funahashi R, Luo Y, Liu C, Park K, Rao M, Malik N, Zou J (2015) Transcription activator-like effector nuclease (TALEN)-mediated CLYBL targeting enables enhanced transgene expression and one-step generation of dual reporter human induced pluripotent stem cell (iPSC) and neural stem cell (NSC) lines. PLoS One 10:e0116032. https://doi.org/10.1371/journal.pone.0116032

Article  CAS  PubMed  PubMed Central  Google Scholar 

Chavez A, Scheiman J, Vora S, Pruitt BW, Tuttle M, P.R.I. E S, Lin S, Kiani CD, Guzman DJ, Wiegand D, Ter-Ovanesyan JL, Braff N, Davidsohn BE, Housden N, Perrimon R, Weiss J, Aach JJ, Collins, Church GM (2015) Highly efficient Cas9-mediated transcriptional programming. Nat Methods 12:326–328. https://doi.org/10.1038/nmeth.3312

Article  CAS  PubMed  PubMed Central  Google Scholar 

Chavez A, Tuttle M, Pruitt BW, Ewen-Campen B, Chari R, Ter-Ovanesyan D, Haque SJ, Cecchi RJ, Kowal EJK, Buchthal J, Housden BE, Perrimon N, Collins JJ, Church G (2016) Comparison of Cas9 activators in multiple species. Nat Methods 13:563–567. https://doi.org/10.1038/nmeth.3871

Article  CAS  PubMed  PubMed Central  Google Scholar 

Desfarges SCA (2012) Viral integration and consequences on host gene expression. Essential Agents of Life, Viruses, pp 147–175

Google Scholar 

Ellis J (2005) Silencing and variegation of gammaretrovirus and lentivirus vectors. Hum Gene Ther 16:1241–1246. https://doi.org/10.1089/hum.2005.16.1241

Article  CAS  PubMed  Google Scholar 

Fan Y, Sanyal S, Bruzzone R (2018) Breaking bad: how viruses subvert the cell cycle. Front Cell Infect Microbiol 8:396. https://doi.org/10.3389/fcimb.2018.00396

Article  CAS  PubMed  PubMed Central  Google Scholar 

Fus-Kujawa A, Prus P, Bajdak-Rusinek K, Teper P, Gawron K, Kowalczuk A, Sieron AL (2021) An overview of methods and tools for transfection of eukaryotic cells in vitro. Front Bioeng Biotechnol 9:701031. https://doi.org/10.3389/fbioe.2021.701031

Article  PubMed  PubMed Central  Google Scholar 

Guo J, Ma D, Huang R, Ming J, Ye M, Kee K, Xie Z, Na J (2017) An inducible CRISPR-ON system for controllable gene activation in human pluripotent stem cells. Protein Cell 8:379–393. https://doi.org/10.1007/s13238-016-0360-8

Article  CAS  PubMed  PubMed Central  Google Scholar 

Gurung IS, Medina-Gomez G, Kis A, Baker M, Velagapudi V, Neogi SG, Campbell M, Rodriguez-Cuenca S, Lelliott C, McFarlane I, Oresic M, Grace AA, Vidal-Puig A, Huang CL (2011) Deletion of the metabolic transcriptional coactivator PGC1beta induces Cardiac Arrhythmia. Cardiovasc Res 92:29–38. https://doi.org/10.1093/cvr/cvr155

Article  CAS  PubMed  PubMed Central  Google Scholar 

Hazelbaker DZ, Beccard A, Angelini G, Mazzucato P, Messana A, Lam D, Eggan K, Barrett LE (2020) A multiplexed gRNA piggyBac transposon system facilitates efficient induction of CRISPRi and CRISPRa in human pluripotent stem cells. Sci Rep 10:635. https://doi.org/10.1038/s41598-020-57500-1

Article  ADS  CAS  PubMed  PubMed Central  Google Scholar 

Herbst F, Ball CR, Tuorto F, Nowrouzi A, Wang W, Zavidij O, Dieter SM, Fessler S, van der Hoeven F, Kloz U, Lyko F, Schmidt M, von Kalle C, Glimm H (2012) Extensive methylation of promoter sequences silences lentiviral transgene expression during stem cell differentiation in vivo. Mol Ther 20:1014–1021. https://doi.org/10.1038/mt.2012.46

Article  CAS  PubMed  PubMed Central  Google Scholar 

Hill AJ, McFaline-Figueroa JL, Starita LM, Gasperini MJ, Matreyek KA, Packer J, Jackson D, Shendure J, Trapnell C (2018) On the design of CRISPR-based single-cell molecular screens. Nat Methods 15:271–274. https://doi.org/10.1038/nmeth.4604

Article  CAS  PubMed  PubMed Central  Google Scholar 

Himeda CL, Chen X, Hauschka SD (2011) Design and testing of regulatory cassettes for optimal activity in skeletal and cardiac muscles. Methods Mol Biol 709:3–19. https://doi.org/10.1007/978-1-61737-982-6_1

Article  CAS  PubMed  Google Scholar 

Ho SM, Hartley BJ, Flaherty E, Rajarajan P, Abdelaal R, Obiorah I, Barretto N, Muhammad H, Phatnani HP, Akbarian S, Brennand KJ (2017) Evaluating synthetic activation and repression of neuropsychiatric-related genes in hiPSC-Derived NPCs, neurons, and astrocytes. Stem Cell Reports 9:615–628. https://doi.org/10.1016/j.stemcr.2017.06.012

Article  CAS  PubMed  PubMed Central  Google Scholar 

Hockemeyer D, Soldner F, Beard C, Gao Q, Mitalipova M, DeKelver RC, Katibah GE, Amora R, Boydston EA, Zeitler B, Meng X, Miller JC, Zhang L, Rebar EJ, Gregory PD, Urnov FD, Jaenisch R (2009) Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases. Nat Biotechnol 27:851–857. https://doi.org/10.1038/nbt.1562

Article  CAS  PubMed  PubMed Central  Google Scholar 

Irion S, Luche H, Gadue P, Fehling HJ, Kennedy M, Keller G (2007) Identification and targeting of the ROSA26 locus in human embryonic stem cells. Nat Biotechnol 25:1477–1482.

留言 (0)

沒有登入
gif