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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 12353 scientific papers returned from the database with the search filters currently being used below.

Mutations in multiple components of the nuclear pore complex cause nephrotic syndrome

Authors:
Braun DA, Lovric S, Schapiro D, Schneider R, Marquez J, Asif M, Hussain MS, Daga A, Widmeier E, Rao J, Ashraf S, Tan W, Lusk CP, Kolb A, Jobst-Schwan T, Schmidt JM, Hoogstraten CA, Eddy K, Kitzler TM, Shril S, Moawia A, Schrage K, Khayyat AIA, Lawson JA, Gee HY, Warejko JK, Hermle T, Majmundar AJ, Hugo H, Budde B, Motameny S, Altmüller J, Noegel AA, Fathy HM, Gale DP, Waseem SS, Khan A, Kerecuk L, Hashmi S, Mohebbi N, Ettenger R, Serdaroğlu E, Alhasan KA, Hashem M, Goncalves S, Ariceta G, Ubetagoyena M, Antonin W, Baig SM, Alkuraya FS, Shen Q, Xu H, Antignac C, Lifton RP, Mane S, Nürnberg P, Khokha MK, Hildebrandt F
Citation:
J Clin Invest. 2018 Sep 4. pii: 98688. doi: 10.1172/JCI98688. [Epub ahead of print]
Epub:
Yes
Abstract:
Steroid-resistant nephrotic syndrome (SRNS) almost invariably progresses to end-stage renal disease. Although more than 50...
Delivery Method:
microinjection
Organism or Cell Type:
Xenopus
Citation Extract:
Braun DA, Lovric S, Schapiro D, Schneider R, Marquez J, Asif M, Hussain MS, Daga A, Widmeier E, Rao J, Ashraf S, Tan W, Lusk CP, Kolb A, Jobst-Schwan T, Schmidt JM, Hoogstraten CA, Eddy K, Kitzler TM, Shril S, Moawia A, Schrage K, Khayyat AIA, Lawson JA, Gee HY, Warejko JK, Hermle T, Majmundar AJ, Hugo H, Budde B, Motameny S, Altmüller J, Noegel AA, Fathy HM, Gale DP, Waseem SS, Khan A, Kerecuk L, Hashmi S, Mohebbi N, Ettenger R, Serdaroğlu E, Alhasan KA, Hashem M, Goncalves S, Ariceta G, Ubetagoyena M, Antonin W, Baig SM, Alkuraya FS, Shen Q, Xu H, Antignac C, Lifton RP, Mane S, Nürnberg P, Khokha MK, Hildebrandt F. Mutations in multiple components of the nuclear pore complex cause nephrotic syndrome. J Clin Invest. 2018 Sep 4. pii: 98688. doi: 10.1172/JCI98688. [Epub ahead of print].

Use of Glucose-Fructose to Enhance the Exon Skipping Efficacy

Authors:
Han G, Gao X, Yin H
Citation:
Methods Mol Biol. 2018;1828:327-342. doi: 10.1007/978-1-4939-8651-4_20
Epub:
Not Epub
Abstract:
Exon-skipping antisense oligonucleotides (AOs) are promising treatments for muscle-related genetic ailments including Duchenne...
Delivery Method:
injection
Organism or Cell Type:
mice mdx
Citation Extract:
Han G, Gao X, Yin H. Use of Glucose-Fructose to Enhance the Exon Skipping Efficacy. Methods Mol Biol. 2018;1828:327-342. doi: 10.1007/978-1-4939-8651-4_20.

Systemic Intravenous Administration of Antisense Therapeutics for Combinatorial Dystrophin and Myostatin Exon Splice Modulation

Authors:
Lu-Nguyen N, Dickson G, Malerba A
Citation:
Methods Mol Biol. 2018;1828:343-354. doi: 10.1007/978-1-4939-8651-4_21
Epub:
Not Epub
Abstract:
Using antisense oligonucleotides (AOs) to reframe mutated dystrophin, a recently developed therapeutic approach for Duchenne...
Organism or Cell Type:
mice mdx
Citation Extract:
Lu-Nguyen N, Dickson G, Malerba A. Systemic Intravenous Administration of Antisense Therapeutics for Combinatorial Dystrophin and Myostatin Exon Splice Modulation. Methods Mol Biol. 2018;1828:343-354. doi: 10.1007/978-1-4939-8651-4_21.

Antisense Oligonucleotide Targeting of 3'-UTR of mRNA for Expression Knockdown

Authors:
Golshirazi G, Ciszewski L, Lu-Nguyen N, Popplewell L
Citation:
Methods Mol Biol. 2018;1828:91-124. doi: 10.1007/978-1-4939-8651-4_6
Epub:
Not Epub
Abstract:
With the recent conditional approval of an antisense oligonucleotide (AON) that restores the reading frame of DMD transcript in...
Citation Extract:
Golshirazi G, Ciszewski L, Lu-Nguyen N, Popplewell L. Antisense Oligonucleotide Targeting of 3'-UTR of mRNA for Expression Knockdown. Methods Mol Biol. 2018;1828:91-124. doi: 10.1007/978-1-4939-8651-4_6.

14-3-3εa directs the pulsatile transport of basal factors toward the apical domain for lumen growth in tubulogenesis

Authors:
Mizotani Y, Suzuki M, Hotta K, Watanabe H, Shiba K, Inaba K, Tashiro E, Oka K, Imoto M
Citation:
Proc Natl Acad Sci U S A. 2018 Aug 29. pii: 201808756. doi: 10.1073/pnas.1808756115. [Epub ahead of print]
Epub:
Yes
Abstract:
The Ciona notochord has emerged as a simple and tractable in vivo model for tubulogenesis. Here, using a chemical genetics...
Delivery Method:
microinjection
Organism or Cell Type:
Ciona intestinalis (ascidian)
Citation Extract:
Mizotani Y, Suzuki M, Hotta K, Watanabe H, Shiba K, Inaba K, Tashiro E, Oka K, Imoto M. 14-3-3εa directs the pulsatile transport of basal factors toward the apical domain for lumen growth in tubulogenesis. Proc Natl Acad Sci U S A. 2018 Aug 29. pii: 201808756. doi: 10.1073/pnas.1808756115. [Epub ahead of print].

Dnd is required for primordial germ cell specification in Oryzias celebensis

Authors:
Zhu T, Gui L, Zhu Y, Li Y, Li M
Citation:
Gene. 2018 Aug 29. pii: S0378-1119(18)30928-4. doi: 10.1016/j.gene.2018.08.068. [Epub ahead of print]
Epub:
Yes
Abstract:
Dead end (dnd) is a germ plasm component that plays an essential role for primordial germ cell (PGC) development in vertebrates...
Delivery Method:
microinjection
Organism or Cell Type:
Oryzias celebensis (Celebes ricefish)
Citation Extract:
Zhu T, Gui L, Zhu Y, Li Y, Li M. Dnd is required for primordial germ cell specification in Oryzias celebensis. Gene. 2018 Aug 29. pii: S0378-1119(18)30928-4. doi: 10.1016/j.gene.2018.08.068. [Epub ahead of print].

Exon Skipping Using Antisense Oligonucleotides for Laminin-Alpha2-Deficient Muscular Dystrophy

Authors:
Hara Y, Mizobe Y, Miyatake S, Takizawa H, Nagata T, Yokota T, Takeda S, Aoki Y
Citation:
Methods Mol Biol. 2018;1828:553-564. doi: 10.1007/978-1-4939-8651-4_36
Epub:
Not Epub
Abstract:
Phosphorodiamidate morpholino oligomer (PMO)-mediated exon skipping is among the more promising approaches available for the...
Organism or Cell Type:
mice laminin-α2-deficient dy 3K/dy 3K
Citation Extract:
Hara Y, Mizobe Y, Miyatake S, Takizawa H, Nagata T, Yokota T, Takeda S, Aoki Y. Exon Skipping Using Antisense Oligonucleotides for Laminin-Alpha2-Deficient Muscular Dystrophy. Methods Mol Biol. 2018;1828:553-564. doi: 10.1007/978-1-4939-8651-4_36.

Morpholino-Mediated Exon Skipping Targeting Human ACVR1/ALK2 for Fibrodysplasia Ossificans Progressiva

Authors:
Maruyama R, Yokota T
Citation:
Methods Mol Biol. 2018;1828:497-502. doi: 10.1007/978-1-4939-8651-4_32
Epub:
Not Epub
Abstract:
Fibrodysplasia ossificans progressiva (FOP) is a rare autosomal-dominant disorder characterized by progressive heterotopic...
Organism or Cell Type:
cell culture: Fibrodysplasia ossificans progressiva human primary cells
Citation Extract:
Maruyama R, Yokota T. Morpholino-Mediated Exon Skipping Targeting Human ACVR1/ALK2 for Fibrodysplasia Ossificans Progressiva. Methods Mol Biol. 2018;1828:497-502. doi: 10.1007/978-1-4939-8651-4_32.

Exon Skipping by Ultrasound-Enhanced Delivery of Morpholino with Bubble Liposomes for Myotonic Dystrophy Model Mice

Authors:
Negishi Y, Endo-Takahashi Y, Ishiura S
Citation:
Methods Mol Biol. 2018;1828:481-487. doi: 10.1007/978-1-4939-8651-4_30
Epub:
Not Epub
Abstract:
Abnormal splicing of the chloride channel 1 (CLCN1) gene causes myotonic dystrophy type 1 (DM1). Therefore, controlling the...
Delivery Method:
bubble liposome ultrasonic
Organism or Cell Type:
mice
Citation Extract:
Negishi Y, Endo-Takahashi Y, Ishiura S. Exon Skipping by Ultrasound-Enhanced Delivery of Morpholino with Bubble Liposomes for Myotonic Dystrophy Model Mice. Methods Mol Biol. 2018;1828:481-487. doi: 10.1007/978-1-4939-8651-4_30.

Morpholino-Mediated Exon Inclusion for SMA

Authors:
Zhou H, Muntoni F
Citation:
Methods Mol Biol. 2018;1828:467-477. doi: 10.1007/978-1-4939-8651-4_29
Epub:
Not Epub
Abstract:
The application of antisense oligonucleotides (AONs) to modify pre-messenger RNA splicing has great potential for treating...
Organism or Cell Type:
mice SMA
Citation Extract:
Zhou H, Muntoni F. Morpholino-Mediated Exon Inclusion for SMA. Methods Mol Biol. 2018;1828:467-477. doi: 10.1007/978-1-4939-8651-4_29.

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