Summary: Synthetic analogs of NUCLEIC ACIDS composed of morpholine ring derivatives (MORPHOLINES) linked by phosphorodimidates. One standard DNA nucleic acid base (ADENINE; GUANINE; CYTOSINE; OR THYMINE) is bound to each morpholine ring.

Top Publications

  1. Fagg W, Liu N, FAIR J, Shiue L, Katzman S, Donohue J, et al. Autogenous cross-regulation of Quaking mRNA processing and translation balances Quaking functions in splicing and translation. Genes Dev. 2017;31:1894-1909 pubmed publisher
  2. Seigfried F, Cizelsky W, Pfister A, Dietmann P, Walther P, Kuhl M, et al. Frizzled 3 acts upstream of Alcam during embryonic eye development. Dev Biol. 2017;426:69-83 pubmed publisher
    ..Altogether our study reveals that alcam is activated through non-canonical Wnt signalling during embryonic eye development in Xenopus laevis and shows that this pathway plays a similar role in different tissues. ..
  3. Kroeger P, Drummond B, Miceli R, McKernan M, Gerlach G, Marra A, et al. The zebrafish kidney mutant zeppelin reveals that brca2/fancd1 is essential for pronephros development. Dev Biol. 2017;428:148-163 pubmed publisher
    ..Several independent brca2 morpholinos (MOs) phenocopied zep, causing edema, reduced podocyte number, and increased interrenal cell number...
  4. Järver P, O Donovan L, Gait M. A chemical view of oligonucleotides for exon skipping and related drug applications. Nucleic Acid Ther. 2014;24:37-47 pubmed publisher
  5. Subbotina E, Koganti S, Hodgson Zingman D, Zingman L. Morpholino-driven gene editing: A new horizon for disease treatment and prevention. Clin Pharmacol Ther. 2016;99:21-5 pubmed publisher
  6. Kesselheim A, Avorn J. Approving a Problematic Muscular Dystrophy Drug: Implications for FDA Policy. JAMA. 2016;316:2357-2358 pubmed publisher
  7. Rudigier L, Dame C, Scholz H, Kirschner K. Ex vivo cultures combined with vivo-morpholino induced gene knockdown provide a system to assess the role of WT1 and GATA4 during gonad differentiation. PLoS ONE. 2017;12:e0176296 pubmed publisher ex vivo technique for cultivating gonads in hanging droplets of media that were supplemented with vivo-morpholinos to knockdown WT1 and GATA4 either alone or in combination at the same developmental stage...
  8. Prager A, Hagenlocher C, Ott T, Schambony A, Feistel K. hmmr mediates anterior neural tube closure and morphogenesis in the frog Xenopus. Dev Biol. 2017;430:188-201 pubmed publisher
    ..Our results pinpoint a novel role of hmmr in anterior neural development and support the notion that RI is a major driving force for anterior neurulation and forebrain morphogenesis. ..
  9. Aartsma Rus A, Krieg A. FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga. Nucleic Acid Ther. 2017;27:1-3 pubmed publisher

More Information


  1. Opar A. Exon-skipping drug pulls ahead in muscular dystrophy field. Nat Med. 2012;18:1314 pubmed publisher
  2. Joris M, Schloesser M, Baurain D, Hanikenne M, Muller M, Motte P. Number of inadvertent RNA targets for morpholino knockdown in Danio rerio is largely underestimated: evidence from the study of Ser/Arg-rich splicing factors. Nucleic Acids Res. 2017;45:9547-9557 pubmed publisher
    ..Our data contribute to the debate about MO specificity, efficacy and the number of unknown targeted sequences. ..
  3. Kobayashi D, Asano Hoshino A, Nakakura T, Nishimaki T, Ansai S, Kinoshita M, et al. Loss of zinc finger MYND-type containing 10 (zmynd10) affects cilia integrity and axonemal localization of dynein arms, resulting in ciliary dysmotility, polycystic kidney and scoliosis in medaka (Oryzias latipes). Dev Biol. 2017;430:69-79 pubmed publisher
    ..Adult mutants exhibited sperm dysmotility, scoliosis and progressive polycystic kidney. ..
  4. Williams R, Winkfein R, Ginger R, Green M, Schnetkamp P, Wheeler G. A functional approach to understanding the role of NCKX5 in Xenopus pigmentation. PLoS ONE. 2017;12:e0180465 pubmed publisher
    ..Further rescue experiments including the use of ion exchange inactive SLC24A5 constructs raise the possibility that full ion exchanger function of NCKX5 may not be required for rescue of pigmentation. ..
  5. Lai J, Collins M, Uribe V, Jiménez Amilburu V, Günther S, Maischein H, et al. The Hippo pathway effector Wwtr1 regulates cardiac wall maturation in zebrafish. Development. 2018;145: pubmed publisher
    ..Altogether, these data suggest that Wwtr1 establishes the compact wall architecture necessary for trabeculation, and that Nrg/Erbb2 signaling negatively regulates its nuclear localization and therefore its activity. ..
  6. Seguin A, Takahashi Makise N, Yien Y, Huston N, Whitman J, Musso G, et al. Reductions in the mitochondrial ABC transporter Abcb10 affect the transcriptional profile of heme biosynthesis genes. J Biol Chem. 2017;292:16284-16299 pubmed publisher
  7. Wong H, Lin J, Ströhl F, Roque C, Cioni J, Cagnetta R, et al. RNA Docking and Local Translation Regulate Site-Specific Axon Remodeling In Vivo. Neuron. 2017;95:852-868.e8 pubmed publisher
    ..The results demonstrate a requirement for LPS in building arbor complexity and suggest a key role for pre-synaptic LPS in assembling neural circuits. ..
  8. Ke W, Ye D, Mersch K, Xu H, Chen S, Lin F. G?1 is required for neutrophil migration in zebrafish. Dev Biol. 2017;428:135-147 pubmed publisher
    ..Collectively, our data indicate that G?1 signaling controls proper neutrophil migration by activating PI3K and modulating actin dynamics. Moreover, they illustrate a role for a specific G? isoform in chemotaxis in vivo. ..
  9. Miyake A, Mekata Y, Fujibayashi H, Nakanishi K, Konishi M, Itoh N. Brorin is required for neurogenesis, gliogenesis, and commissural axon guidance in the zebrafish forebrain. PLoS ONE. 2017;12:e0176036 pubmed publisher
    ..The present results suggest that Brorin is a secreted Bmp antagonist predominantly expressed in developing neural tissues and that it plays multiple roles in the development of the zebrafish forebrain. ..
  10. Janesick A, Tang W, Nguyen T, Blumberg B. RAR?2 is required for vertebrate somitogenesis. Development. 2017;144:1997-2008 pubmed publisher
    ..These results demonstrate for the first time an early and essential role for RAR?2 in vertebrate somitogenesis. ..
  11. Karunakaran D, Kanadia R. In Vivo and Explant Electroporation of Morpholinos in the Developing Mouse Retina. Methods Mol Biol. 2017;1565:215-227 pubmed publisher
    ..Here, we describe a rapid and efficient delivery of morpholinos to add another tool to perturb gene expression during mouse retinal development.
  12. Dong X, Li J, He L, Gu C, Jia W, Yue Y, et al. Zebrafish Znfl1 proteins control the expression of hoxb1b gene in the posterior neuroectoderm by acting upstream of pou5f3 and sall4 genes. J Biol Chem. 2017;292:13045-13055 pubmed publisher
  13. Bian S, Zheng X, Sun H, Chen J, Lu Y, Liu Y, et al. Clock1a affects mesoderm development and primitive hematopoiesis by regulating Nodal-Smad3 signaling in the zebrafish embryo. J Biol Chem. 2017;292:14165-14175 pubmed publisher
  14. Vejlegaard K, Paul S, Kosbar T, Wengel J, Caruthers M. Oligodeoxynucleotides containing 2'-amino-LNA nucleotides as constrained morpholino phosphoramidate and phosphorodiamidate monomers. Bioorg Med Chem Lett. 2017;27:3173-3176 pubmed publisher
    ..Thermal denaturation studies showed that the novel 2'-amino-LNA-based morpholino monomers exert a destabilizing effects on duplexes formed with complementary DNA and RNA. ..
  15. D Agati G, Beltre R, Sessa A, Burger A, Zhou Y, Mosimann C, et al. A defect in the mitochondrial protein Mpv17 underlies the transparent casper zebrafish. Dev Biol. 2017;430:11-17 pubmed publisher
    ..Identification of mpv17 mutants will allow for further work probing the relationship between mitochondrial function and pigmentation, which has to date received little attention. ..
  16. Piatek M, HENDERSON V, Fearn A, Chaudhry B, Werner A. Ectopically expressed Slc34a2a sense-antisense transcripts cause a cerebellar phenotype in zebrafish embryos depending on RNA complementarity and Dicer. PLoS ONE. 2017;12:e0178219 pubmed publisher
    ..Our findings suggest that RNAi is involved in gene regulation by certain natural antisense RNAs. ..
  17. Kelu J, Webb S, Parrington J, Galione A, Miller A. Ca2+ release via two-pore channel type 2 (TPC2) is required for slow muscle cell myofibrillogenesis and myotomal patterning in intact zebrafish embryos. Dev Biol. 2017;425:109-129 pubmed publisher
  18. Kyritsi K, Meng F, Zhou T, Wu N, Venter J, Francis H, et al. Knockdown of Hepatic Gonadotropin-Releasing Hormone by Vivo-Morpholino Decreases Liver Fibrosis in Multidrug Resistance Gene 2 Knockout Mice by Down-Regulation of miR-200b. Am J Pathol. 2017;187:1551-1565 pubmed publisher
    ..Targeting the GnRH/GnRHR1/miR-200b axis may be key for the management of hepatic fibrosis during the progression of PSC. ..
  19. Stein C, Castanotto D. FDA-Approved Oligonucleotide Therapies in 2017. Mol Ther. 2017;25:1069-1075 pubmed publisher
    ..Of all the molecules evaluated as of January 2017, the regulatory authorities assessed that six provided clear clinical benefit in rigorously controlled trials. The story of these six is given in this review. ..