Generation of Atrial-Specific Construct Using Sarcolipin Promoter-Associated CRM4 Enhancer.
AAV9
Atrium
CRM4
Cis-acting regulatory module
Gene therapy
Sarcolipin
Journal
Methods in molecular biology (Clifton, N.J.)
ISSN: 1940-6029
Titre abrégé: Methods Mol Biol
Pays: United States
ID NLM: 9214969
Informations de publication
Date de publication:
2022
2022
Historique:
entrez:
30
8
2022
pubmed:
31
8
2022
medline:
3
9
2022
Statut:
ppublish
Résumé
Cardiac gene therapy has been hampered by off-target expression of gene of interest irrespective of variety of delivery methods. To overcome this issue, cardiac-specific promoters provide target tissue specificity, although expression is often debilitated compared to that of ubiquitous promoters. We have previously shown that sarcolipin promoter with an enhancer calsequestrin cis-regulatory module 4 (CRM4) combination has an improved atrial specificity. Moreover, it showed a minimal extra-atrial expression, which is a significant advantage for AAV9-mediated cardiac gene therapy. Therefore, it can be a useful tool to study and treat atrial-specific diseases such as atrial fibrillation. In this chapter, we introduce practical and simple methodology for atrial-specific gene therapy using sarcolipin promoter with an enhancer CRM4.
Identifiants
pubmed: 36040590
doi: 10.1007/978-1-0716-2707-5_9
doi:
Substances chimiques
Calsequestrin
0
Muscle Proteins
0
Proteolipids
0
sarcolipin
145018-73-1
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
115-132Informations de copyright
© 2022. The Author(s), under exclusive license to Springer Science+Business Media, LLC, part of Springer Nature.
Références
Carlsson L, Duker G, Jacobson I (2010) New pharmacological targets and treatments for atrial fibrillation. Trends Pharmacol Sci 31(8):364–371
doi: 10.1016/j.tips.2010.05.001
Bongianino R, Priori SG (2015) Gene therapy to treat cardiac arrhythmias. Nat Rev Cardiol 12(9):531–546
doi: 10.1038/nrcardio.2015.61
Bikou O, Thomas D, Trappe K, Lugenbiel P, Kelemen K, Koch M, Soucek R, Voss F, Becker R, Katus HA (2011) Connexin 43 gene therapy prevents persistent atrial fibrillation in a porcine model. Cardiovasc Res 92(2):218–225
doi: 10.1093/cvr/cvr209
Inagaki K, Fuess S, Storm TA, Gibson GA, Mctiernan CF, Kay MA, Nakai H (2006) Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8. Mol Ther 14(1):45–53
doi: 10.1016/j.ymthe.2006.03.014
Zincarelli C, Soltys S, Rengo G, Rabinowitz JE (2008) Analysis of AAV serotypes 1–9 mediated gene expression and tropism in mice after systemic injection. Mol Ther 16(6):1073–1080
doi: 10.1038/mt.2008.76
Del Monte F, Williams E, Lebeche D, Schmidt U, Rosenzweig A, Gwathmey JK, Lewandowski ED, Hajjar RJ (2001) Improvement in survival and cardiac metabolism after gene transfer of sarcoplasmic reticulum Ca2+-ATPase in a rat model of heart failure. Circulation 104(12):1424–1429
doi: 10.1161/hc3601.095574
Jeong D, Lee M-A, Li Y, Yang DK, Kho C, Oh JG, Hong G, Lee A, Song MH, LaRocca TJ (2016) Matricellular protein CCN5 reverses established cardiac fibrosis. J Am Coll Cardiol 67(13):1556–1568
doi: 10.1016/j.jacc.2016.01.030
Miyamoto MI, Del Monte F, Schmidt U, DiSalvo TS, Kang ZB, Matsui T, Guerrero JL, Gwathmey JK, Rosenzweig A, Hajjar RJ (2000) Adenoviral gene transfer of SERCA2a improves left-ventricular function in aortic-banded rats in transition to heart failure. Proc Natl Acad Sci 97(2):793–798
doi: 10.1073/pnas.97.2.793
Wahlquist C, Jeong D, Rojas-Muñoz A, Kho C, Lee A, Mitsuyama S, van Mil A, Park WJ, Sluijter JP, Doevendans PA (2014) Inhibition of miR-25 improves cardiac contractility in the failing heart. Nature 508(7497):531–535
doi: 10.1038/nature13073
Müller O, Schinkel S, Kleinschmidt J, Katus H, Bekeredjian R (2008) Augmentation of AAV-mediated cardiac gene transfer after systemic administration in adult rats. Gene Ther 15(23):1558–1565
doi: 10.1038/gt.2008.111
Yoo J, Kohlbrenner E, Kim O, Hajjar RJ, Jeong D (2018) Enhancing atrial-specific gene expression using a calsequestrin cis-regulatory module 4 with a sarcolipin promoter. J Gene Med 20(12):e3060
doi: 10.1002/jgm.3060
Nettelbeck DM, Jérôme V, Müller R (1998) A strategy for enhancing the transcriptional activity of weak cell type-specific promoters. Gene Ther 5(12):1656–1664
doi: 10.1038/sj.gt.3300778
Prasad KR, Xu Y, Yang Z, Acton ST, French BA (2011) Robust cardiomyocyte-specific gene expression following systemic injection of AAV: in vivo gene delivery follows a Poisson distribution. Gene Ther 18(1):43–52
doi: 10.1038/gt.2010.105
Aikawa R, Huggins GS, Snyder RO (2002) Cardiomyocyte-specific gene expression following recombinant adeno-associated viral vector transduction. J Biol Chem 277(21):18979–18985
doi: 10.1074/jbc.M201257200
Werfel S, Jungmann A, Lehmann L, Ksienzyk J, Bekeredjian R, Kaya Z, Leuchs B, Nordheim A, Backs J, Engelhardt S (2014) Rapid and highly efficient inducible cardiac gene knockout in adult mice using AAV-mediated expression of Cre recombinase. Cardiovasc Res 104(1):15–23
doi: 10.1093/cvr/cvu174
Franz W-M, Rothmann T, Frey N, Katus HA (1997) Analysis of tissue-specific gene delivery by recombinant adenoviruses containing cardiac-specific promoters. Cardiovasc Res 35(3):560–566
doi: 10.1016/S0008-6363(97)00154-5
Ni L, Scott L Jr, Campbell HM, Pan X, Alsina KM, Reynolds J, Philippen LE, Hulsurkar M, Lagor WR, Li N (2019) Atrial-specific gene delivery using an adeno-associated viral vector. Circ Res 124(2):256–262
doi: 10.1161/CIRCRESAHA.118.313811
Small EM, Krieg PA (2004) Molecular regulation of cardiac chamber-specific gene expression. Trends Cardiovasc Med 14(1):13–18
doi: 10.1016/j.tcm.2003.09.005
Chen Z, Xian W, Bellin M, Dorn T, Tian Q, Goedel A, Dreizehnter L, Schneider CM, Ward-van Oostwaard D, Ng JKM (2017) Subtype-specific promoter-driven action potential imaging for precise disease modelling and drug testing in hiPSC-derived cardiomyocytes. Eur Heart J 38(4):292–301
Biendarra-Tiegs SM, Secreto FJ, Nelson TJ (2020) Addressing variability and heterogeneity of induced pluripotent stem cell-derived cardiomyocytes. Adv Exp Med Biol 1212(6):1–29
Babu GJ, Bhupathy P, Petrashevskaya NN, Wang H, Raman S, Wheeler D, Jagatheesan G, Wieczorek D, Schwartz A, Janssen PM (2006) Targeted overexpression of sarcolipin in the mouse heart decreases sarcoplasmic reticulum calcium transport and cardiac contractility. J Biol Chem 281(7):3972–3979
doi: 10.1074/jbc.M508998200
Zheng J, Yancey DM, Ahmed MI, Wei C-C, Powell PC, Shanmugam M, Gupta H, Lloyd SG, McGiffin DC, Schiros CG (2014) Increased sarcolipin expression and adrenergic drive in humans with preserved left ventricular ejection fraction and chronic isolated mitral regurgitation. Circ Heart Fail 7(1):194–202
doi: 10.1161/CIRCHEARTFAILURE.113.000519
Pashmforoush M, Lu JT, Chen H, St Amand T, Kondo R, Pradervand S, Evans SM, Clark B, Feramisco JR, Giles W (2004) Nkx2-5 pathways and congenital heart disease: loss of ventricular myocyte lineage specification leads to progressive cardiomyopathy and complete heart block. Cell 117(3):373–386
doi: 10.1016/S0092-8674(04)00405-2
Uemura N, Ohkusa T, Hamano K, Nakagome M, Hori H, Shimizu M, Matsuzaki M, Mochizuki S, Minamisawa S, Ishikawa Y (2004) Down-regulation of sarcolipin mRNA expression in chronic atrial fibrillation. Eur J Clin Investig 34(11):723–730
doi: 10.1111/j.1365-2362.2004.01422.x
Bhupathy P, Babu GJ, Periasamy M (2007) Sarcolipin and phospholamban as regulators of cardiac sarcoplasmic reticulum Ca2+ ATPase. J Mol Cell Cardiol 42(5):903–911
doi: 10.1016/j.yjmcc.2007.03.738
Voit A, Patel V, Pachon R, Shah V, Bakhutma M, Kohlbrenner E, McArdle JJ, Dell’Italia LJ, Mendell JR, Xie L-H (2017) Reducing sarcolipin expression mitigates Duchenne muscular dystrophy and associated cardiomyopathy in mice. Nat Commun 8(1):1–14
doi: 10.1038/s41467-017-01146-7
Rincon MY, Sarcar S, Danso-Abeam D, Keyaerts M, Matrai J, Samara-Kuko E, Acosta-Sanchez A, Athanasopoulos T, Dickson G, Lahoutte T (2015) Genome-wide computational analysis reveals cardiomyocyte-specific transcriptional cis-regulatory motifs that enable efficient cardiac gene therapy. Mol Ther 23(1):43–52
doi: 10.1038/mt.2014.178
Wilmott P, Lisowski L, Alexander IE, Logan GJ (2019) A user’s guide to the inverted terminal repeats of adeno-associated virus. Hum Gene Ther Methods 30(6):206–213
doi: 10.1089/hgtb.2019.276
Xie J, Mao Q, Tai PW, He R, Ai J, Su Q, Zhu Y, Ma H, Li J, Gong S (2017) Short DNA hairpins compromise recombinant adeno-associated virus genome homogeneity. Mol Ther 25(6):1363–1374
doi: 10.1016/j.ymthe.2017.03.028
White SM, Constantin PE, Claycomb WC (2004) Cardiac physiology at the cellular level: use of cultured HL-1 cardiomyocytes for studies of cardiac muscle cell structure and function. Am J Phys Heart Circ Phys 286(3):H823–H829
Claycomb WC, Lanson NA, Stallworth BS, Egeland DB, Delcarpio JB, Bahinski A, Izzo NJ (1998) HL-1 cells: a cardiac muscle cell line that contracts and retains phenotypic characteristics of the adult cardiomyocyte. Proc Natl Acad Sci 95(6):2979–2984
doi: 10.1073/pnas.95.6.2979
Bajaj G, Sharma RK (2006) TNF-α-mediated cardiomyocyte apoptosis involves caspase-12 and calpain. Biochem Biophys Res Commun 345(4):1558–1564
doi: 10.1016/j.bbrc.2006.05.059
Burt R, Graves BM, Gao M, Li C, Williams DL, Fregoso SP, Hoover DB, Li Y, Wright GL, Wondergem R (2013) 9-Phenanthrol and flufenamic acid inhibit calcium oscillations in HL-1 mouse cardiomyocytes. Cell Calcium 54(3):193–201
doi: 10.1016/j.ceca.2013.06.003