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Morpholino Publication Database

This database contains citations and abstracts for research using Morpholino oligos, as well as some review articles incorporating Morpholino data. You can search the content using the filter boxes below.

There are 12376 scientific papers returned from the database with the search filters currently being used below.

The nuclear pore complex function of Sec13 is required for cell survival during retinal development

Authors:
Niu X, Hong J, Zheng X, Melville DB, Knapik EW, Meng A, Peng J
Citation:
J Biol Chem. 2014 Mar 13. [Epub ahead of print]
Epub:
Yes
Abstract:
Sec13 is a dual function protein, being a core component of both the COPII coat, which mediates protein trafficking from the...
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Niu X, Hong J, Zheng X, Melville DB, Knapik EW, Meng A, Peng J. The nuclear pore complex function of Sec13 is required for cell survival during retinal development. J Biol Chem. 2014 Mar 13. [Epub ahead of print].

Modulation of the Secretory Pathway Rescues Zebrafish Polycystic Kidney Disease Pathology

Authors:
Le Corre S, Eyre D, Drummond IA
Citation:
J Am Soc Nephrol. 2014 Mar 13. [Epub ahead of print]
Epub:
Not Epub
Abstract:
Mutations in polycystin 1 and polycystin 2 are responsible for autosomal dominant polycystic kidney disease, the most common...
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Le Corre S, Eyre D, Drummond IA. Modulation of the Secretory Pathway Rescues Zebrafish Polycystic Kidney Disease Pathology. J Am Soc Nephrol. 2014 Mar 13. [Epub ahead of print].

Effect of Combined Systemic and Local Morpholino Treatment on the Spinal Muscular Atrophy Δ7 Mouse Model Phenotype

Authors:
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
Citation:
Clin Therap. 2014. 36(3):340-56. doi:10.1016/j.clinthera.2014.02.004
Epub:
Not Epub
Abstract:
Background: Spinal muscular atrophy (SMA) is a fatal motor neuron disease of childhood that is caused by mutations in the SMN1...
Delivery Method:
Vivo-Morpholino
Organism or Cell Type:
mouse SMA model
Citation Extract:
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. Effect of Combined Systemic and Local Morpholino Treatment on the Spinal Muscular Atrophy Δ7 Mouse Model Phenotype. Clin Therap. 2014. 36(3):340-56. doi:10.1016/j.clinthera.2014.02.004.

Functional role of arginine during the peri-implantation period of pregnancy. I. Consequences of loss of function of arginine transporter SLC7A1 mRNA in ovine conceptus trophectoderm

Authors:
Wang X, Frank JW, Little DR, Dunlap KA, Satterfield MC, Burghardt RC, Hansen TR, Wu G, Bazer FW
Citation:
FASEB J. 2014;[Epub ahead of print] doi:10.1096/fj.13-248757
Epub:
Yes
Abstract:
Arginine, the common substrate for production of nitric oxide (NO) and polyamines in mammals, increases in the uterine lumen...
Delivery Method:
Endo-Porter
Organism or Cell Type:
Ovis aries oocyte
Citation Extract:
Wang X, Frank JW, Little DR, Dunlap KA, Satterfield MC, Burghardt RC, Hansen TR, Wu G, Bazer FW. Functional role of arginine during the peri-implantation period of pregnancy. I. Consequences of loss of function of arginine transporter SLC7A1 mRNA in ovine conceptus trophectoderm. FASEB J. 2014;[Epub ahead of print] doi:10.1096/fj.13-248757 .

New developments in exon skipping and splice modulation therapies for neuromuscular diseases

Authors:
Touznik A, Lee JJ, Yokota T
Citation:
Expert Opin Biol Ther. 2014 Mar 12. [Epub ahead of print]
Epub:
Yes
Abstract:
Introduction: Antisense oligonucleotide (AON) therapy is a form of treatment for genetic or infectious diseases using small,...
Citation Extract:
Touznik A, Lee JJ, Yokota T. New developments in exon skipping and splice modulation therapies for neuromuscular diseases. Expert Opin Biol Ther. 2014 Mar 12. [Epub ahead of print].

BMP9 mutations cause a vascular-anomaly syndrome with phenotypic overlap with hereditary hemorrhagic telangiectasia

Authors:
Wooderchak-Donahue WL, McDonald J, O'Fallon B, Upton PD, Li W, Roman BL, Young S, Plant P, Fülöp GT, Langa C, Morrell NW, Botella LM, Bernabeu C, Stevenson DA, Runo JR, Bayrak-Toydemir P
Citation:
Am J Hum Genet. 2013 Sep 5;93(3):530-7. doi: 10.1016/j.ajhg.2013.07.004. Epub 2013 Aug 22
Epub:
Not Epub
Abstract:
Hereditary hemorrhagic telangiectasia (HHT), the most common inherited vascular disorder, is caused by mutations in genes...
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Wooderchak-Donahue WL, McDonald J, O'Fallon B, Upton PD, Li W, Roman BL, Young S, Plant P, Fülöp GT, Langa C, Morrell NW, Botella LM, Bernabeu C, Stevenson DA, Runo JR, Bayrak-Toydemir P. BMP9 mutations cause a vascular-anomaly syndrome with phenotypic overlap with hereditary hemorrhagic telangiectasia. Am J Hum Genet. 2013 Sep 5;93(3):530-7. doi: 10.1016/j.ajhg.2013.07.004. Epub 2013 Aug 22.

Snx3 regulates recycling of the transferrin receptor and iron assimilation

Authors:
Chen C, Garcia-Santos D, Ishikawa Y, Seguin A, Li L, Fegan KH, Hildick-Smith GJ, Shah DI, Cooney JD, Chen W, King MJ, Yien YY, Schultz IJ, Anderson H, Dalton AJ, Freedman ML, Kingsley PD, Palis J, Hattangadi SM, Lodish HF, Ward DM, Kaplan J, Maeda T, Ponka P, Paw BH
Citation:
Cell Metab. 2013 Mar 5;17(3):343-52. doi: 10.1016/j.cmet.2013.01.013. Epub 2013 Feb 14
Epub:
Not Epub
Abstract:
Sorting of endocytic ligands and receptors is critical for diverse cellular processes. The physiological significance of...
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Chen C, Garcia-Santos D, Ishikawa Y, Seguin A, Li L, Fegan KH, Hildick-Smith GJ, Shah DI, Cooney JD, Chen W, King MJ, Yien YY, Schultz IJ, Anderson H, Dalton AJ, Freedman ML, Kingsley PD, Palis J, Hattangadi SM, Lodish HF, Ward DM, Kaplan J, Maeda T, Ponka P, Paw BH. Snx3 regulates recycling of the transferrin receptor and iron assimilation. Cell Metab. 2013 Mar 5;17(3):343-52. doi: 10.1016/j.cmet.2013.01.013. Epub 2013 Feb 14.

What Can We Learn From Clinical Trials of Exon Skipping for DMD?

Authors:
Lu Q-l, Cirak S, Terence Partridge T
Citation:
Molec Ther Nucleic Acids. 2014;3:e152. doi:10.1038/mtna.2014.6
Epub:
Not Epub
Abstract:
No abstract - this is available as open access online.
Delivery Method:
i.v. infusion
Organism or Cell Type:
human
Citation Extract:
Lu Q-l, Cirak S, Terence Partridge T. What Can We Learn From Clinical Trials of Exon Skipping for DMD?. Molec Ther Nucleic Acids. 2014;3:e152. doi:10.1038/mtna.2014.6.

Immunophilin FKBP52 induces Tau-P301L filamentous assembly in vitro and modulates its activity in a model of tauopathy

Authors:
Giustiniani J, Chambraud B, Sardin E, Dounane O, Guillemeau K, Nakatani H, Paquet D, Kamah A, Landrieu I, Lippens G, Baulieu E-E, Tawk M
Citation:
PNAS USA. 2014;[Epub ahead of print] doi:10.1073/pnas.1402645111
Epub:
Yes
Abstract:
Growing evidence underlines the role attributed to abnormal forms of Tau in several neurodegenerative diseases known as...
Organism or Cell Type:
zebrafish
Citation Extract:
Giustiniani J, Chambraud B, Sardin E, Dounane O, Guillemeau K, Nakatani H, Paquet D, Kamah A, Landrieu I, Lippens G, Baulieu E-E, Tawk M. Immunophilin FKBP52 induces Tau-P301L filamentous assembly in vitro and modulates its activity in a model of tauopathy. PNAS USA. 2014;[Epub ahead of print] doi:10.1073/pnas.1402645111.

Multicellular Tumor Spheroids as a Model for Assessing Delivery of Oligonucleotides in Three Dimensions

Authors:
Carver K, Ming X, Juliano RL
Citation:
Mol Ther Nucleic Acids. 2014;3:e153. doi:10.1038/mtna.2014.5
Epub:
Not Epub
Abstract:
Oligonucleotides have shown promise in selectively manipulating gene expression in vitro, but that success has not translated...
Delivery Method:
peptide-linked
Organism or Cell Type:
culture: multicellular tumor spheroid
Citation Extract:
Carver K, Ming X, Juliano RL. Multicellular Tumor Spheroids as a Model for Assessing Delivery of Oligonucleotides in Three Dimensions. Mol Ther Nucleic Acids. 2014;3:e153. doi:10.1038/mtna.2014.5.

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