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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 259 scientific papers returned from the database with the search filters currently being used below.

Wild-type mouse models to screen antisense oligonucleotides for exon-skipping efficacy in duchenne muscular dystrophy

Authors:
Cao L, Han G, Gu B, Yin H
Citation:
PLoS One. 2014 Nov 3;9(11):e111079. doi: 10.1371/journal.pone.0111079. eCollection 2014
Epub:
Not Epub
Abstract:
A readily available animal model is essential for rapidly identifying effective treatments for Duchenne muscular dystrophy (DMD...
Delivery Method:
injection and peptide-coupled
Organism or Cell Type:
mice
Citation Extract:
Cao L, Han G, Gu B, Yin H. Wild-type mouse models to screen antisense oligonucleotides for exon-skipping efficacy in duchenne muscular dystrophy. PLoS One. 2014 Nov 3;9(11):e111079. doi: 10.1371/journal.pone.0111079. eCollection 2014.

Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease

Authors:
Clayton NP, Nelson CA, Weeden T, Taylor KM, Moreland RJ, Scheule RK, Phillips L, Leger AJ, Cheng SH, Wentworth BM
Citation:
Mol Ther Nucleic Acids. 2014 Oct 28;3:e206. doi: 10.1038/mtna.2014.57
Epub:
Not Epub
Abstract:
Pompe disease is an autosomal recessive disorder caused by a deficiency of acid α-glucosidase (GAA; EC 3.2.1.20) and the...
Delivery Method:
injection & electroporation or peptide-linked
Organism or Cell Type:
mice
Citation Extract:
Clayton NP, Nelson CA, Weeden T, Taylor KM, Moreland RJ, Scheule RK, Phillips L, Leger AJ, Cheng SH, Wentworth BM. Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease. Mol Ther Nucleic Acids. 2014 Oct 28;3:e206. doi: 10.1038/mtna.2014.57.

Splice-correcting oligonucleotides restore BTK function in X-linked agammaglobulinemia model

Authors:
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
Citation:
J Clin Invest. 2014 Aug 8. pii: 76175. doi: 10.1172/JCI76175. [Epub ahead of print]
Epub:
Yes
Abstract:
X-linked agammaglobulinemia (XLA) is an inherited immunodeficiency that results from mutations within the gene encoding Bruton...
Delivery Method:
Peptide-linked or bare oligo
Organism or Cell Type:
mice, cell culture: U2OS reporter cell line
Citation Extract:
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. Splice-correcting oligonucleotides restore BTK function in X-linked agammaglobulinemia model. J Clin Invest. 2014 Aug 8. pii: 76175. doi: 10.1172/JCI76175. [Epub ahead of print].

Identification and functional analysis of the primary pantothenate transporter, PfPAT, of the human malaria parasite Plasmodium falciparum

Authors:
Augagneur Y, Jaubert L, Schiavoni M, Pachikara N, Garg A, Usmani-Brown S, Wesolowski D, Zeller S, Ghosal A, Cornillot E, Said HM, Kumar P, Altman S, Ben Mamoun C
Citation:
J Biol Chem. 2013 Jul 12;288(28):20558-67. doi: 10.1074/jbc.M113.482992. Epub 2013 May 31
Epub:
Not Epub
Abstract:
The human malaria parasite Plasmodium falciparum is absolutely dependent on the acquisition of host pantothenate for its...
Delivery Method:
peptide-coupled
Organism or Cell Type:
Plasmodium falciparum
Citation Extract:
Augagneur Y, Jaubert L, Schiavoni M, Pachikara N, Garg A, Usmani-Brown S, Wesolowski D, Zeller S, Ghosal A, Cornillot E, Said HM, Kumar P, Altman S, Ben Mamoun C. Identification and functional analysis of the primary pantothenate transporter, PfPAT, of the human malaria parasite Plasmodium falciparum. J Biol Chem. 2013 Jul 12;288(28):20558-67. doi: 10.1074/jbc.M113.482992. Epub 2013 May 31.

Induced IL-10 Splice Altering Approach to Antiviral Drug Discovery

Authors:
Panchal RG, Mourich DV, Bradfute S, Hauck LL, Warfield KL, Iversen PL, Bavari S
Citation:
Nucleic Acid Ther. 2014;24(3):179-185. doi:10.1089/nat.2013.0457
Epub:
Not Epub
Abstract:
Ebola virus causes an acute hemorrhagic fever lethal in primates and rodents. The contribution of host immune factors to...
Delivery Method:
peptide-linked
Organism or Cell Type:
mice
Citation Extract:
Panchal RG, Mourich DV, Bradfute S, Hauck LL, Warfield KL, Iversen PL, Bavari S. Induced IL-10 Splice Altering Approach to Antiviral Drug Discovery. Nucleic Acid Ther. 2014;24(3):179-185. doi:10.1089/nat.2013.0457.

Unanchored K48-Linked Polyubiquitin Synthesized by the E3-Ubiquitin Ligase TRIM6 Stimulates the Interferon-IKKε Kinase-Mediated Antiviral Response

Authors:
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
Citation:
Immunity. 2014;[Epub ahead of print] doi:10.1016/j.immuni.2014.04.018
Epub:
Yes
Abstract:
Type I interferons (IFN-I) are essential antiviral cytokines produced upon microbial infection. IFN-I elicits this activity...
Delivery Method:
peptide-linked
Organism or Cell Type:
BALB/c mice
Citation Extract:
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. Unanchored K48-Linked Polyubiquitin Synthesized by the E3-Ubiquitin Ligase TRIM6 Stimulates the Interferon-IKKε Kinase-Mediated Antiviral Response. Immunity. 2014;[Epub ahead of print] doi:10.1016/j.immuni.2014.04.018.

Silencing of GRA10 protein expression inhibits Toxoplasma gondii intracellular growth and development

Authors:
Witola WH, Bauman B, McHugh M, Matthews K
Citation:
Parasitol Int. 2014 May 13. pii: S1383-5769(14)00059-2. doi: 10.1016/j.parint.2014.05.001. [Epub ahead of print]
Epub:
Yes
Abstract:
Toxoplasma gondii dense granule proteins (GRAs) are secreted abundantly in both the tachyzoite and bradyzoite stages of the...
Delivery Method:
peptide-linked
Organism or Cell Type:
Toxoplasma gondii
Citation Extract:
Witola WH, Bauman B, McHugh M, Matthews K. Silencing of GRA10 protein expression inhibits Toxoplasma gondii intracellular growth and development. Parasitol Int. 2014 May 13. pii: S1383-5769(14)00059-2. doi: 10.1016/j.parint.2014.05.001. [Epub ahead of print].

Effective dystrophin restoration by a novel muscle-homing peptide-morpholino conjugate in dystrophin-deficient mdx mice

Authors:
Gao X, Zhao J, Han G, Zhang Y, Dong X, Cao L, Wang Q, Moulton HM, Yin H
Citation:
Mol Ther. 2014 Apr 15. doi: 10.1038/mt.2014.63. [Epub ahead of print]
Epub:
Yes
Abstract:
Antisense oligonucleotide (AO) - mediated splice correction therapy for Duchenne muscular dystrophy (DMD) has shown huge...
Delivery Method:
peptide-linked
Organism or Cell Type:
mdx mice
Citation Extract:
Gao X, Zhao J, Han G, Zhang Y, Dong X, Cao L, Wang Q, Moulton HM, Yin H. Effective dystrophin restoration by a novel muscle-homing peptide-morpholino conjugate in dystrophin-deficient mdx mice. Mol Ther. 2014 Apr 15. doi: 10.1038/mt.2014.63. [Epub ahead of print].

Targeted Exon Skipping to Correct Exon Duplications in the Dystrophin Gene

Authors:
Greer KL, Lochmüller H, Flanigan K, Fletcher S, Wilton SD
Citation:
Molec Ther Nucleic Acids. 2014;3,e155. doi:10.1038/mtna.2014.8
Epub:
Not Epub
Abstract:
Duchenne muscular dystrophy is a severe muscle-wasting disease caused by mutations in the dystrophin gene that ablate...
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: primary fibroblasts induced myogenic
Citation Extract:
Greer KL, Lochmüller H, Flanigan K, Fletcher S, Wilton SD. Targeted Exon Skipping to Correct Exon Duplications in the Dystrophin Gene. Molec Ther Nucleic Acids. 2014;3,e155. doi:10.1038/mtna.2014.8.

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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