Lessons learned from vivo-morpholinos: How to avoid vivo-morpholino toxicity

BioTechniques

Volumn 56, Issue 5, 2014, Pages 251-256

  Ferguson, David P ^a,b   Dangott, Lawrence J ^b   Timothy Lightfoot, J ^b  

^a BAYLOR COLLEGE OF MEDICINE   (United States)

^b TEXAS A AND M UNIVERSITY   (United States)

Author keywords

Annexin A6; Blood clot formation; Calsequestrin 1; Vivo morpholino

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; CALPHOBINDIN II; CALSEQUESTRIN; DENDRIMER; DOPAMINE 1 RECEPTOR; GLUCOSE TRANSPORTER 4; GUANIDINE; MORPHOLINO OLIGONUCLEOTIDE; UNCLASSIFIED DRUG; VIVO MORPHOLINO; CALCIUM BINDING PROTEIN; CASQ1 PROTEIN, MOUSE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BLOOD CLOT; BLOOD CLOTTING TIME; BLOOD VISCOSITY; CONTROLLED STUDY; FATALITY; GENE SILENCING; HEART ARREST; HEART MUSCLE NECROSIS; MALE; MATRIX ASSISTED LASER DESORPTION IONIZATION TIME OF FLIGHT MASS SPECTROMETRY; MORTALITY; MOUSE; NONHUMAN; NUCLEIC ACID HYBRIDIZATION; NUCLEOTIDE SEQUENCE; ANIMAL; BLOOD CLOTTING; C57BL MOUSE; DOSE RESPONSE; DRUG EFFECTS; GENETICS; INJECTION; MASS SPECTROMETRY;

ANIMALS; ANNEXIN A6; BLOOD COAGULATION; CALCIUM-BINDING PROTEINS; DOSE-RESPONSE RELATIONSHIP, DRUG; GENE SILENCING; INJECTIONS; MALE; MICE, INBRED C57BL; MORPHOLINOS; MORTALITY; OLIGONUCLEOTIDES, ANTISENSE; SPECTROMETRY, MASS, MATRIX-ASSISTED LASER DESORPTION-IONIZATION;

EID: 84900451689     PISSN: 07366205     EISSN: 19409818     Source Type: Journal    
DOI: 10.2144/000114167     Document Type: Article

References (40)

1. Stein, D.A. 2008. Inhibition of RNA virus infections with peptide-conjugated morpholino oligomers. Curr. Pharm. Des. 14:2619-2634.
2. Morcos, P.A., Y. Li, and S. Jiang. 2008. Vivo-Morpholinos: a non-peptide transporter delivers Morpholinos into a wide array of mouse tissues. Biotechniques 45:613-618.
3. Guo, Y., L. Ma, M. Cristofanilli, R.P. Hart, A. Hao, and M. Schachner. 2011. Transcription factor Sox11b is involved in spinal cord regeneration in adult zebrafish. Neuroscience 172:329-341.
4. Kang, J.K., A. Malerba, L. Popplewell, K. Foster, and G. Dickson. 2011. Antisense-induced myostatin exon skipping leads to muscle hypertrophy in mice following octa-guanidine morpholino oligomer treatment. Mol. Ther. 19:159-164.
5. Kim, S., U.P. Radhakrishnan, S.K. Rajpurohit, V. Kulkarni, and P. Jagadeeswaran. 2010. Vivo-Morpholino knockdown of alphaIIb: A novel approach to inhibit thrombocyte function in adult zebrafish. Blood Cells Mol. Dis. 44:169-174.
6. Kizil, C. and M. Brand. 2011. Cerebroventricular microinjection (CVMI) into adult zebrafish brain is an efficient misexpression method for forebrain ventricular cells. PLoS ONE 6:e27395.
7. Kowalik, L. and A.J. Hudspeth. 2011. A search for factors specifying tonotopy implicates DNER in hair-cell development in the chick's cochlea. Dev. Biol. 354:221-231.
8. Liu, Y., H. Yu, S.K. Deaton, and B.G. Szaro. 2012. Heterogeneous nuclear ribonucleoprotein K, an RNA-binding protein, is required for optic axon regeneration in Xenopus laevis. J. Neurosci. 32:3563-3574.
9. Maki, N., R. Suetsugu-Maki, S. Sano, K. Nakamura, O. Nishimura, H. Tarui, K. Del Rio-Tsonis, K. Ohsumi, et al. 2010. Oocyte-type linker histone B4 is required for transdifferentiation of somatic cells in vivo. FASEB J. 24:3462-3467.
10. Matsuda, H. and Y.B. Shi. 2010. An essential and evolutionarily conserved role of protein arginine methyltransferase 1 for adult intestinal stem cells during postembryonic development. Stem Cells 28:2073-2083.
11. Nazmi, A., K. Dutta, and A. Basu. 2010. Antiviral and neuroprotective role of octaguanidinium dendrimer-conjugated morpholino oligomers in Japanese encephalitis. PLoS Negl. Trop. Dis. 4:e892.
12. Nazmi, A., R. Mukhopadhyay, K. Dutta, and A. Basu. 2012. STING Mediates Neuronal Innate Immune Response Following Japanese Encephalitis Virus Infection. Sci Rep. 2:347.
13. Osorio, F.G., C.L. Navarro, J. Cadinanos, I.C. Lopez-Mejia, P.M. Quiros, C. Bartoli, J. Rivera, J. Tazi, et al. 2011. Splicing-directed therapy in a new mouse model of human accelerated aging, Sci Transl Med. 106:106ra107.
14. Owen, L.A., H. Uehara, J. Cahoon, W. Huang, J. Simonis, and B.K. Ambati. 2012. Morpholino-mediated increase in soluble Flt-1 expression results in decreased ocular and tumor neovascularization. PLoS ONE 7:e33576.
15. Parra, M.K., T.L. Gallagher, S.L. Amacher, N. Mohandas, and J.G. Conboy. 2012. Deep intron elements mediate nested splicing events at consecutive AG dinucleotides to regulate alternative 3́ splice site choice in vertebrate 4.1 genes. Mol. Cell. Biol. 32:2044-2053.
16. Parra, M.K., S. Gee, N. Mohandas, and J.G. Conboy. 2011. Efficient in vivo manipulation of alternative pre-mRNA splicing events using antisense morpholinos in mice. J. Biol. Chem. 286:6033-6039.
17. Quinn, M., Y. Ueno, H.Y. Pae, L. Huang, G. Frampton, C. Galindo, H. Francis, D. Horvat, et al. 2012. Suppression of the HPA axis during extrahepatic biliary obstruction induces cholangiocyte proliferation in the rat. Am. J. Physiol. Gastrointest. Liver Physiol. 302:G182-G193.
18. Reissner, K.J., G.C. Sartor, E.M. Vazey, T.E. Dunn, G. Aston-Jones, and P.W. Kalivas. 2012. Use of vivo-morpholinos for control of protein expression in the adult rat brain. J. Neurosci. Methods 203:354-360.
19. Sartor, G.C. and G.S. Aston-Jones. 2012. A septal-hypothalamic pathway drives orexin neurons, which is necessary for conditioned cocaine preference. J. Neurosci. 32:4623-4631.
20. Shi, Y.B., T. Hasebe, L. Fu, K. Fujimoto, and A. Ishizuya-Oka. 2011. The development of the adult intestinal stem cells: Insights from studies on thyroid hormone-dependent amphibian metamorphosis. Cell Biosci. 1:30.
21. Taniguchi-Ikeda, M., K. Kobayashi, M. Kanagawa, C.C. Yu, K. Mori, T. Oda, A. Kuga, H. Kurahashi, et al. 2011. Pathogenic exon-trapping by SVA retrotransposon and rescue in Fukuyama muscular dystrophy. Nature 478:127-131.
22. Tsonis, P.A., T. Haynes, N. Maki, K. Nakamura, M.M. Casco-Robles, S. Yamada, T. Miura, C. Chiba, and K. Del Rio-Tsonis. 2011. Controlling gene loss of function in newts with emphasis on lens regeneration. Nat. Protoc. 6:593-599.
23. Vera, T. and D.E. Stec. 2010. Moderate hyperbilirubinemia improves renal hemodynamics in ANG II-dependent hypertension. Am. J. Physiol. Regul. Integr. Comp. Physiol. 299:R1044-R1049.
24. Widrick, J.J., S. Jiang, S.J. Choi, S.T. Knuth, and P.A. Morcos. 2011. An octaguanidine-morpholino oligo conjugate improves muscle function of mdx mice. Muscle Nerve 44:563-570.
25. Wu, B., E. Benrashid, P. Lu, C. Cloer, A. Zillmer, M. Shaban, and Q.L. Lu. 2011. Targeted skipping of human dystrophin exons in transgenic mouse model systemically for antisense drug development. PLoS ONE 6:e19906.
26. Wu, B., Y. Li, P.A. Morcos, T.J. Doran, P. Lu, and Q.L. Lu. 2009. Octa-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx mice. Mol. Ther. 17:864-871.
27. Zammarchi, F., E. de Stanchina, E. Bournazou, T. Supakorndej, K. Martires, E. Riedel, A.D. Corben, J.F. Bromberg, and L. Cartegni. 2011. Antitumorigenic potential of STAT3 alternative splicing modulation. Proc. Natl. Acad. Sci. USA 108:17779-17784.
28. Azoitei, N., G.V. Pusapati, A. Kleger, P. Moller, R. Kufer, F. Genze, M. Wagner, J. van Lint, et al. 2010. Protein kinase D2 is a crucial regulator of tumour cell-endothelial cell communication in gastrointestinal tumours. Gut 59:1316-1330.
29. Ferguson, D.P., E.E. Schmitt, and J.T. Lightfoot. 2013. Vivo-Morpholinos Induced Transient Knockdown of Physical Activity Related Proteins. PLoS ONE 8:e61472.
30. Wu, B., P. Lu, E. Benrashid, S. Malik, J. Ashar, T.J. Doran, and Q.L. Lu. 2010. Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino. Gene Ther. 17:132-140.
31. Notch, E.G., J.R. Shaw, B.A. Coutermarsh, M. Dzioba, and B.A. Stanton. 2011. Morpholino gene knockdown in adult Fundulus heteroclitus: role of SGK1 in seawater acclimation. PLoS ONE 6:e29462.
32. National Institute for Occupational Safety and Health., Tracor Jitco Inc., Advanced Engineering & Planning Corp. Registry of toxic effects of chemical substances, 1977-19: DHEW publication no (NIOSH), U.S. Dept. of Health, Education, and Welfare, Public Health Service, Center for Disease Control Supt. of Docs., U.S. G.P.O., Cincinnati, Ohio Washington, D.C., pp. microfiches.
33. Lightfoot, J.T., L. Leamy, D. Pomp, M.J. Turner, A.A. Fodor, A. Knab, R.S. Bowen, D. Ferguson, et al. 2010. Strain screen and haplotype association mapping of wheel running in inbred mouse strains. J. Appl. Physiol. 109:623-634.
34. Lightfoot, J.T., M.J. Turner, M. Daves, A. Vordermark, and S.R. Kleeberger. 2004. Genetic influence on daily wheel running activity level. Physiol. Genomics 19:270-276.
35. Lightfoot, J.T., M.J. Turner, D. Pomp, S.R. Kleeberger, and L.J. Leamy. 2008. Quantitative trait loci for physical activity traits in mice. Physiol. Genomics 32:401-408.
36. Fagerquist, C.K., B.R. Garbus, K.E. Williams, A.H. Bates, and L.A. Harden. 2010. Covalent attachment and dissociative loss of sinapinic acid to/from cysteine-containing proteins from bacterial cell lysates analyzed by MALDI-TOF-TOF mass spectrometry. J. Am. Soc. Mass Spectrom. 21:819-832.
37. Lim, H., J. Nam, Y. Lee, S. Xue, S. Chung, and S. Shin. Coagulation Study Using Light-Transmission Method, 14th Intrenational Conference on Miniaturized Systems for Chemistry and Life Sciences (2010) 803-806.
38. Gordon, R.J., G.K. Snyder, H. Tritel, and W.J. Taylor. 1974. Potential significance of plasma viscosity and hematocrit variations in myocardial ischemia. Am. Heart J. 87:175-182.
39. Roberts, J.C., M.K. Bhalgat, and R.T. Zera. 1996. Preliminary biological evaluation of polyamidoamine (PAMAM) Starburst dendrimers. J. Biomed. Mater. Res. 30:53-65.
40. Klajnert, B. and M. Bryszewska. 2001. Dendrimers: properties and applications. Acta Biochim. Pol. 48:199-208.