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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 12378 scientific papers returned from the database with the search filters currently being used below.
There are 12378 scientific papers returned from the database with the search filters currently being used below.
5-methylcytosine (m5C) RNA modification controls the innate immune response to virus infection by regulating type I interferons
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Citation:
Proc Nat Acad Sci USA. 2022;119(42):e2123338119 doi:10.1073/pnas.2123338119 Epub:
Not Epub Abstract:
5-methylcytosine (m5C) is one of the most prevalent modifications of RNA, playing important roles in RNA metabolism, nuclear... Delivery Method:
Vivo-Morpholino intranasal & i.v. injection Organism or Cell Type:
mice Citation Extract: Zhang Y, Zhang L-S, Dai Q, Chen P, Lu M, Kairis EL, Murugaiah V, Xu J, Shukla RK, Liang X, Zou Z, Cormet-Boyaka E, Qiu J, Peeples ME, Sharma A, He C, Li J. 5-methylcytosine (m5C) RNA modification controls the innate immune response to virus infection by regulating type I interferons. Proc Nat Acad Sci USA. 2022;119(42):e2123338119 doi:10.1073/pnas.2123338119. |
Brain milieu induces early microglial maturation through the BAX-Notch axis
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Citation:
Nat Commun. 2022;13(1):6117. doi:10.1038/s41467-022-33836-2 Epub:
Not Epub Abstract:
Microglia are derived from primitive myeloid cells and gain their early identity in the embryonic brains. However, the... Delivery Method:
microinjection Organism or Cell Type:
zebrafish Citation Extract: Zhao F, He J, Tang J, Cui N, Shi Y, Li Z, Liu S, Wang Y, Ma M, Zhao C, Luo L, Li L. Brain milieu induces early microglial maturation through the BAX-Notch axis. Nat Commun. 2022;13(1):6117. doi:10.1038/s41467-022-33836-2. |
P.198 Durable AOC mediated exon 44 skipping in non-human primate muscle tissue and dystrophin protein restoration in DMD patient derived skeletal muscle cells
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Citation:
Neuromuscul Disord. 2022;32(Suppl 1):S127. doi:10.1016/j.nmd.2022.07.353 Epub:
Not Epub Abstract:
Duchenne muscular dystrophy (DMD) is a neuromuscular disease caused by predominantly out-of-frame mutations in the dystrophin... Delivery Method:
antibody-linked Organism or Cell Type:
cell culture: patient-derived skeletal muscle, nonhuman primates Citation Extract: Karamanlidis G, Etxaniz U, Missinato M, Diaz M, Bhardwaj R, Tyaglo O, Lemoine K, Marks I, Albin T, Leung L, Kovach P, Anderson A, Cochran M, Huan H, Younis H, Flanagan M, Levin A. P.198 Durable AOC mediated exon 44 skipping in non-human primate muscle tissue and dystrophin protein restoration in DMD patient derived skeletal muscle cells. Neuromuscul Disord. 2022;32(Suppl 1):S127. doi:10.1016/j.nmd.2022.07.353. |
P.194 Development of a novel, EEV-Conjugated PMO for Duchenne muscular dystrophy
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Citation:
Neuromuscul Disord. 2022;32(Suppl 1):S126. doi:10.1016/j.nmd.2022.07.349 Epub:
Not Epub Abstract:
Duchenne muscular dystrophy (DMD) is a rare, X-linked neuromuscular disorder caused by frameshift mutations in the DMD gene... Delivery Method:
i.v. injection Organism or Cell Type:
mice, nonhuman primates Citation Extract: Kreher N, Kumar A, Hicks A, Peddigari S, Li X, Pathak A, Kheirabadi M, K. Kamer, Estrella N, Dougherty P, Lian W, Liu N, Gao N, Wang D, Streeter M, Dhanabal M, Qian Z, Girgenrath M, Sethuraman N. P.194 Development of a novel, EEV-Conjugated PMO for Duchenne muscular dystrophy. Neuromuscul Disord. 2022;32(Suppl 1):S126. doi:10.1016/j.nmd.2022.07.349. |
VP.58 Golodirsen induced DMD transcripts localization and dystrophin production in MyoD-converted fibroblasts from 4053-101 clinical trial patients
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Citation:
Neuromuscul Disord. 2022;32(Suppl 1):S103. doi:10.1016/j.nmd.2022.07.252 Epub:
Not Epub Abstract:
Antisense oligonucleotides (AONs) are short, synthetic nucleic acid sequences that work by modulating exon incorporation at the... Delivery Method:
i.v. infusion Organism or Cell Type:
cell culture: patient-derived fibroblasts, humans Citation Extract: Rossi R, Moore M, Torelli S, Ala P, Catapano F, Phadke R, Morgan J, Malhotra J, Muntoni F. VP.58 Golodirsen induced DMD transcripts localization and dystrophin production in MyoD-converted fibroblasts from 4053-101 clinical trial patients. Neuromuscul Disord. 2022;32(Suppl 1):S103. doi:10.1016/j.nmd.2022.07.252. |
P.120 Unlocking the potential of oligonucleotide therapeutics for Duchenne muscular dystrophy through enhanced delivery
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Citation:
Neuromuscul Disord. 2022;32(Suppl 1):S99. doi:10.1016/j.nmd.2022.07.236 Epub:
Not Epub Abstract:
PGN-EDO51 is PepGen's clinical candidate to treat individuals with Duchenne muscular dystrophy (DMD) amenable to exon 51... Delivery Method:
peptide-linked Organism or Cell Type:
mice, non-human primates Citation Extract: Mellion M, McArthur J, Holland A, Gunnoo S, Ching S, Johnson R, Irwin C, Lonkar P, Bracegirdle S, Svenstrup N, Goyal J, Godfrey C, Larkindale J. P.120 Unlocking the potential of oligonucleotide therapeutics for Duchenne muscular dystrophy through enhanced delivery. Neuromuscul Disord. 2022;32(Suppl 1):S99. doi:10.1016/j.nmd.2022.07.236. |
P.123 A Phase I/II study of NS-089/NCNP-02, Exon 44 skipping drug, in patients with Duchenne muscular dystrophy
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Citation:
Neuromuscul Disord. 2022;32(Suppl 1):S99-S100. doi:10.1016/j.nmd.2022.07.239 Epub:
Not Epub Abstract:
This is to report on the current status of Phase I/II study of NS-089/NCNP-02, which is a novel phosphorodiamidate morpholino... Delivery Method:
i.v. infusion Organism or Cell Type:
human Citation Extract: Komaki H, Takeshita E, Kunitake K, Shimizu-Motohashi Y, Sasaki M, Yonee C, Maruyama S, Hida E, Matsubara D, Hatakeyama T, Muashige Y, Aoki Y. P.123 A Phase I/II study of NS-089/NCNP-02, Exon 44 skipping drug, in patients with Duchenne muscular dystrophy. Neuromuscul Disord. 2022;32(Suppl 1):S99-S100. doi:10.1016/j.nmd.2022.07.239. |
P.131 Building a FORCETM platform-based DMD franchise for the treatment of individuals with mutations amenable to exon skipping
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Citation:
Neuromuscul Disord. 2022;32(Suppl 1):S101-S102. doi:10.1016/j.nmd.2022.07.247 Epub:
Not Epub Abstract:
Current treatments for Duchenne muscular dystrophy (DMD) use phosphorodiamidate morpholino oligomer (PMO) to induce exon... Delivery Method:
Fab-linked Organism or Cell Type:
cell culture: patient-derived mytubes, mice, non-human primates Citation Extract: Desjardins C, Venkatesan R, O'Donnell E, Hall J, Russo R, Spring S, Tang K, Davis J, Weeden T, Zanotti S, Beskrovnaya O. P.131 Building a FORCETM platform-based DMD franchise for the treatment of individuals with mutations amenable to exon skipping. Neuromuscul Disord. 2022;32(Suppl 1):S101-S102. doi:10.1016/j.nmd.2022.07.247. |
P.132 Casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: Interim results from the Phase 3 ESSENCE trial
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Citation:
Neuromuscul Disord. 2022;32(Suppl 1):S102. doi:10.1016/j.nmd.2022.07.248 Epub:
Not Epub Abstract:
Casimersen is FDA approved for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed DMD gene... Delivery Method:
i.v. infusion Organism or Cell Type:
human Citation Extract: Iannaccone S, Phan H, Straub V, Muntoni F, Wolf D, Malhotra J, Chu R, Darton E, Mercuri E. P.132 Casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: Interim results from the Phase 3 ESSENCE trial. Neuromuscul Disord. 2022;32(Suppl 1):S102. doi:10.1016/j.nmd.2022.07.248. |
P.134 Real-world outcomes of exon skipping therapy use in patients with Duchenne muscular dystrophy: Experience at a single, large tertiary care center
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Citation:
Neuromuscul Disord. 2022;32(Suppl 1):S102. doi:10.1016/j.nmd.2022.07.250 Epub:
Not Epub Abstract:
To assess outcomes among patients with Duchenne muscular dystrophy (DMD) receiving exon skipping therapy in real-world practice... Delivery Method:
i.v. infusion Organism or Cell Type:
human Citation Extract: Yaworski A, Duong T, Low J, Gee R, Watson K, Buu M, Kaufman B, Klotz J, Day J, Guzman J, Tesi Rocha C. P.134 Real-world outcomes of exon skipping therapy use in patients with Duchenne muscular dystrophy: Experience at a single, large tertiary care center. Neuromuscul Disord. 2022;32(Suppl 1):S102. doi:10.1016/j.nmd.2022.07.250. |