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Of Mice and Morpholinos, 2014

These are some papers using Morpholinos in mouse, published in 2014.

Vivo-Morpholino

WT1 controls antagonistic FGF and BMP-pSMAD pathways in early renal progenitors . Motamedi FJ, Badro DA, Clarkson M, Rita Lecca M, Bradford ST, Buske FA, Saar K, Hübner N, Brändli AW, Schedl A. Nat Commun. 2014 Jul 17;5:4444. doi: 10.1038/ncomms5444.
Vivo-Morpholino in kidney organ culture.

Effect of Combined Systemic and Local Morpholino Treatment on the Spinal Muscular Atrophy Δ7 Mouse Model Phenotype. Nizzardo N, Simone C, Salani S, Ruepp M-D, Rizzo F, Ruggieri M, Zanetta C, Brajkovic S, Moulton HM, Müehlemann O, Bresolin N, Comi GP, Corti S. Clin Therap. 2014. 36(3):340-56. doi:10.1016/j.clinthera.2014.02.004.
Vivo-Morpholino in SMA model.

miR-142-3p balances proliferation and differentiation of mesenchymal cells during lung development. Carraro G, Shrestha A, Rostkovius J, Contreras A, Chao CM, El Agha E, Mackenzie B, Dilai S, Guidolin D, Taketo MM, Günther A, Kumar ME, Seeger W, De Langhe S, Barreto G, Bellusci S. Development. 2014 Feb 19. [Epub ahead of print].
Vivo-Morpholino in lung explant culture.

Secretin Stimulates Biliary Cell Proliferation by Regulating Expression of MicroRNA 125b and MicroRNA let7a in Mice. Glaser S, Meng F, Han Y, Onori P, Chow BK, Francis H, Venter J, McDaniel K, Marzioni M, Invernizzi P, Ueno Y, Lai JM, Huang L, Standeford H, Alvaro D, Gaudio E, Franchitto A, Alpini G. Gastroenterology. 2014 Feb 25. pii: S0016-5085(14)00241-8. doi: 10.1053/j.gastro.2014.02.030. [Epub ahead of print].
Vivo-Morpholino.

Differential Skeletal Muscle Proteome of High and Low Active Mice. Ferguson DP, Dangott LJ, Schmitt EE, Vellers HL, Lightfoot JT. Differential J Appl Physiol. 2014 Feb 6. [Epub ahead of print].
Vivo-Morpholino.

A sensitive assay system to test antisense oligonucleotides for splice suppression therapy in the mouse liver. Gallego-Villar L, Viecelli HM, Pérez B, Harding CO, Ugarte M, Thöny B, Desviat LR. Mol Ther Nucleic Acids. 2014 Sep 16;3:e193. doi: 10.1038/mtna.2014.44.
Vivo-Morpholino.

Regulation of the extrinsic apoptotic pathway by microRNA-21 in alcoholic liver injury. Francis H, McDaniel K, Han Y, Liu X, Kennedy L, Yang F, McCarra J, Zhou T, Glaser S, Venter J, Huang L, Levine P, Lai J-M, Liu C-G, Alpini G, Meng F. J Biol Chem. 2014;[Epub ahead of print] doi:10.1074/jbc.M114.602383.
Vivo-Morpholino.

Antisense-mediated Exon Skipping Decreases Tau Protein Expression: A Potential Therapy For Tauopathies. Sud R, Geller ET, Schellenberg GD. Mol Ther Nucleic Acids. 2014 Jul 29;3:e180. doi: 10.1038/mtna.2014.30.
Vivo-Morpholino.

Gli1 Activation and Protection Against Hepatic Encephalopathy is Suppressed by Circulating Transforming Growth Factor β1 in Mice. McMillin M, Galindo C, Pae HY, Frampton G, Di Patre PL, Quinn M, Whittington E, DeMorrow S. J Hepatol. 2014;[Epub ahead of print] doi:10.1016/j.jhep.2014.07.015.
Vivo-Morpholino.

Genetic Targets of Hydrogen Sulfide in Ventilator-Induced Lung Injury - A Microarray Study. Spassov S, Pfeifer D, Strosing K, Ryter S, Hummel M, Faller S, Hoetzel A. PLoS One. 2014 Jul 15;9(7):e102401. doi: 10.1371/journal.pone.0102401. eCollection 2014.
Vivo-Morpholino.

TLR7 is a key regulator of innate immunity against Japanese Encephalitis Virus infection. Nazmi A, Mukherjee S, Kundu K, Dutta K, Mahadevan A, Shankar SK, Basu A. Neurobiol Dis. 2014 Jun 5. pii: S0969-9961(14)00154-5. doi: 10.1016/j.nbd.2014.05.036. [Epub ahead of print].
Vivo-Morpholino.

Lessons learned from vivo-morpholinos: How to avoid vivo-morpholino toxicity. Ferguson DP, Dangott JJ, Timothy Lightfoot JT. BioTechniques. 2014;56(5):251-6.
Vivo-Morpholino.

Dynamic Expression of Chymotrypsin-Like Elastase 1 Over the Course of Murine Lung Development. Liu S, Young SM, Varisco BM. Am J Physiol Lung Cell Mol Physiol. 2014 May 2. [Epub ahead of print].
Vivo-Morpholino.

De novo mutation in ATP6V1B2 impairs lysosome acidification and causes dominant deafness-onychodystrophy syndrome. Yuan Y, Zhang J, Chang Q, Zeng J, Xin F, Wang J, Zhu Q, Wu J, Lu J, Guo W, Yan X, Jiang H, Zhou B, Li Q, Gao X, Yuan H, Yang S, Han D, Mao Z, Chen P, Lin X, Dai P. Cell Res. 2014 Jun 10. doi: 10.1038/cr.2014.77. [Epub ahead of print].
Intracochlear injection of Vivo-Morpholino.

Peptide-linked Morpholino

Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease. Clayton NP, Nelson CA, Weeden T, Taylor KM, Moreland RJ, Scheule RK, Phillips L, Leger AJ, Cheng SH, Wentworth BM. Mol Ther Nucleic Acids. 2014 Oct 28;3:e206. doi: 10.1038/mtna.2014.57.
Injection & Electroporation or Peptide-linked Morpholino.

Splice-correcting oligonucleotides restore BTK function in X-linked agammaglobulinemia model. Bestas B, Moreno PM, Blomberg KE, Mohammad DK, Saleh AF, Sutlu T, Nordin JZ, Guterstam P, Gustafsson MO, Kharazi S, Piątosa B, Roberts TC, Behlke MA, Wood MJ, Gait MJ, Lundin KE, El Andaloussi S, Månsson R, Berglöf A, Wengel J, Smith CI. J Clin Invest. 2014 Aug 8. pii: 76175. doi: 10.1172/JCI76175. [Epub ahead of print].
Peptide-linked Morpholino.

Assessment of RT-qPCR Normalization Strategies for Accurate Quantification of Extracellular microRNAs in Murine Serum. Roberts TC, Coenen-Stass AML, Wood MJA. PLoS ONE. 2014;9(2):e89237. doi:10.1371/journal.pone.0089237.
Peptide-linked Morpholino.

Exon skipping restores dystrophin expression, but fails to prevent disease progression in later stage dystrophic dko mice. Wu C, Cloer C, Lu P, Milazi S, Shaban M, Shah SN, Marston-Poe L, Moulton HM, Lu QL. Gene Ther. 2014;[Epub ahead of print] doi:10.1038/gt.2014.53.
Peptide-linked Morpholino.

Unanchored K48-Linked Polyubiquitin Synthesized by the E3-Ubiquitin Ligase TRIM6 Stimulates the Interferon-IKKε Kinase-Mediated Antiviral Response. Rajsbaum R, Versteeg GA, Schmid S, Maestre AM, Belicha-Villanueva A, Martínez-Romero C, Patel JR, Morrison J, Pisanelli G, Miorin L, Laurent-Rolle M, Moulton HM, Stein DA, Fernandez-Sesma A, tenOever BR, García-Sastre A. Immunity. 2014;[Epub ahead of print] doi:10.1016/j.immuni.2014.04.018.
Peptide-linked Morpholino.

Induced IL-10 Splice Altering Approach to Antiviral Drug Discovery. Panchal RG, Mourich DV, Bradfute S, Hauck LL, Warfield KL, Iversen PL, Bavari S. Nucleic Acid Ther. 2014;24(3):179-185. doi:10.1089/nat.2013.0457.
Peptide-linked Morpholino.

Effective dystrophin restoration by a novel muscle-homing peptide-morpholino conjugate in dystrophin-deficient mdx mice. Gao X, Zhao J, Han G, Zhang Y, Dong X, Cao L, Wang Q, Moulton HM, Yin H. Mol Ther. 2014 Apr 15. doi: 10.1038/mt.2014.63. [Epub ahead of print].
Peptide-linked Morpholino.

Oocyte/Embryo Injection

Hira-Mediated H3.3 Incorporation Is Required for DNA Replication and Ribosomal RNA Transcription in the Mouse Zygote. Lin C-J, Koh FM, Wong P, Conti M, Ramalho-Santos M. Dev Cell. 2014;[Epub ahead of print] doi:10.1016/j.devcel.2014.06.022.
Oocyte microinjection.

WASH complex regulates Arp2/3 complex for actin-based polar body extrusion in mouse oocytes. Wang F, Zhang L, Zhang GL, Wang ZB, Cui XS, Kim NH, Sun SC. Sci Rep. 2014 Jul 7;4:5596. doi: 10.1038/srep05596.
Oocyte microinjection.

Possible Role of p38 MAPK-MNK1-EMI2 Cascade in Metaphase-II Arrest of Mouse Oocytes. Miyagaki Y, Kanemori Y, Tanaka F, Baba T. Biol Reprod. 2014;[Epub ahead of print] doi:10.1095/biolreprod.113.116962.
Oocyte microinjection.

Knockdown of RBBP7 unveils a requirement of histone deacetylation for CPC function in mouse oocytes. Balboula AZ, Stein P, Schultz RM, Schindler K. Cell Cycle. 2014;[epub ahead of print]13:0 - -1; PMID: 24317350.
Oocyte microinjection.

Comparison of Epigenetic Mediator Expression and Function in Mouse and Human Embryonic Blastomeres. Chavez SL, McElroy SL, Bossert NL, De Jonge CJ, Rodriguez MV, Leong DE, Behr B, Westphal LM, Pera RAR. Hum Mol Genet. 2014;[Epub ahead of print] doi:10.1093/hmg/ddu212.
Zygote microinjection.

Intracerebroventricular Injection of Bare Morpholino

Morpholino Antisense Oligonucleotides Targeting Intronic Repressor Element1 Improve Phenotype in SMA Mouse Models. Osman EY, Miller MR, Robbins KL, Lombardi AM, Atkinson AK, Brehm AJ, Lorson CL. Hum Mol Genet. 2014;[Epub ahead of print] doi:10.1093/hmg/ddu198.
SMNΔ7 ICV injection.

Electrophysiological Biomarkers in Spinal Muscular Atrophy: Proof of Concept. Arnold WD, Porensky PN, McGovern VL, Iyer CC, Duque S, Li X, Meyer K, Schmelzer L, Kaspar BK, Kolb SJ, Kissel JT, Burghes AH. Ann Clin Transl Neurol. 2014 Jan 1;1(1):34-44.
ICV injection.

Pharmacology of a central nervous system delivered 2'-o-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates. Rigo F, Chun SJ, Norris DA, Hung G, Lee S, Matson J, Fey RA, Gaus H, Hua Y, Grundy JS, Krainer AR, Henry SP, Bennett CF. J Pharmacol Exp Ther. 2014 Jul;350(1):46-55. doi: 10.1124/jpet.113.212407. Epub 2014 Apr 30.
ICV injection.

RIG-I knockdown impedes neurogenesis in a murine model of Japanese encephalitis. Mukherjee S, Ghosh S, Nazmi A, Basu A. Cell Biol Int. 2014 Jul 31. doi: 10.1002/cbin.10354. [Epub ahead of print].
Intracerebral injection; murine neurospheres.

Other Delivery Strategies

HSPA5 is an essential host factor for Ebola virus infection. Patrick Reid S, Shurtleff AC, Costantino JA, Tritsch SR, Retterer C, Spurgers KB, Bavari S. Antiviral Res. 2014 Sep;109:171-4. doi: 10.1016/j.antiviral.2014.07.004. Epub 2014 Jul 11.
IP injection.

Serum-Free Culture of Mid-gestation Mouse Embryos: A Tool for the Study of Endoderm-Derived Organs. Gordon J, Moore BA, Blackburn CC, Manley NR. Methods Mol Biol 2014;[Epub ahead of print] doi: 10.1007/978-1-60327-292-6_12.
Electroporation into mouse embryo.

MiR-153 targets the nuclear factor-1 family and protects against teratogenic effects of ethanol exposure in fetal neural stem cells. Tsai P-C, Bake S, Balaraman S, Rawlings J, Holgate RR, Dubois D, Miranda RC. Biology Open. 2014;[Epub]doi:10.1242/bio.20147765.
Electroporation into single-cell suspensions from murine neurospheres.

Near-Infrared-Light-Based Nano-Platform Boosts Endosomal Escape and Controls Gene Knockdown in Vivo. Jayakumar MK, Bansal A, Huang K, Yao R, Li BN, Zhang Y. ACS Nano. 2014 Apr 14. [Epub ahead of print].
Nanoparticle.

Polymalic Acid-based Nano Biopolymers for Targeting of Multiple Tumor Markers: An Opportunity for Personalized Medicine? Ljubimova JY, Ding H, Portilla-Arias J, Patil R, Gangalum PR, Chesnokova A, Inoue S, Rekechenetskiy A, Nassoura T, Black KL, Holler E. J Vis Exp. 2014;(88):e50668. doi:10.3791/50668.
Nanoparticle.

Blockade of CD47 mediated CTSS-PAR2 signaling provides a therapeutic target for hepatocellular carcinoma. Lee TKW, Cheung VCH, Lu P, Lau EYT, Ma S, Tang KH, Tong M, Lo J, Ng IOL. Hepatology. 2014;[Epub ahead of print] doi:10.1002/hep.27070.
Intratumoral injection into human xenograft in mice.

Bubble Liposomes and Ultrasound Exposure Improve Localized Morpholino Oligomer Delivery into the Skeletal Muscles of Dystrophic mdx Mice. Negishi Y, Ishii Y, Shiono H, Akiyama S, Sekine S, Kojima T, Mayama S, Kikuchi T, Hamano N, Endo-Takahashi Y, Suzuki R, Maruyama K, Aramaki Y. Molec Pharmaceutics. 2014;[epub ahead of print] doi:10.1021/mp4004755.
Bubble liposomes & ultrasound delivery.

Evaluation of Tris[2-(acryloyloxy)ethyl]isocyanurate Cross-linked Polyethylenimine as Antisense Morpholino Oligomer Delivery Vehicle in Cell Culture and Dystrophic mdx Mice. Wang M. Hum Gene Ther. 2014 Jan 9. [Epub ahead of print].
Covalent PEI.

Splicing-Directed Therapy in a New Mouse Model of Human Accelerated Aging. Osorio FG, Navarro CL, Cadiñanos J, López-Mejía IC, Quirós PM, Bartoli C, Rivera J, Tazi J, Guzmán G, Varela I, Depetris D, de Carlos F, Cobo J, Andrés V, Sandre-Giovannoli AD, Freije JM, Lévy N, López-Otín C. Sci Transl Med. 2011 Oct 26;3(106):106ra107. doi: 10.1126/scitranslmed.3002847.

Phosphorodiamidate Morpholino Oligomers (PMOs) suppress mutant huntingtin expression and attenuate neurotoxicity. Sun X, Marque LO, Cordner Z, Pruitt JL, Bhat M, Li P, Kannan G, Ladenheim EE, Moran TH, Margolis RL, Rudnicki DD. Hum Mol Genet. 2014;[Epub ahead of print] doi:10.1093/hmg/ddu349.

Impaired functional communication between the L-type calcium channel and mitochondria contributes to metabolic inhibition in the mdx heart. Viola HM, Adams AM, Davies SM, Fletcher S, Filipovska A, Hool LC. Proc Natl Acad Sci U S A. 2014 Jun 26. pii: 201402544. [Epub ahead of print].

Cardiac CD47 Drives Left Ventricular Heart Failure Through Ca2+‐CaMKII‐Regulated Induction of HDAC3arifi‐Sanjani M, Shoushtari AH, Quiroz M, Baust J, Sestito SF, Mosher M, Ross M, McTiernan CF, St. Croix CM, Bilonick RA, Champion HC, Isenberg JS. J Am Heart Assoc. 2014;3:e000670, originally published June 10, 2014, doi:10.1161/JAHA.113.000670.

Lomofungin and dilomofungin: inhibitors of MBNL1-CUG RNA binding with distinct cellular effects. Hoskins JW, Ofori LO, Chen CZ, Kumar A, Sobczak K, Nakamori M, Southall N, Patnaik S, Marugan JJ, Zheng W, Austin CP, Disney MD, Miller BL, Thornton CA. Nucleic Acids Res. 2014 May 5. [Epub ahead of print].
Electroporation into cultured mouse C2C12 myogenic cells.

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