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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 11585 scientific papers returned from the database with the search filters currently being used below.

Brain milieu induces early microglial maturation through the BAX-Notch axis

Authors:
Zhao F, He J, Tang J, Cui N, Shi Y, Li Z, Liu S, Wang Y, Ma M, Zhao C, Luo L, Li L
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

Authors:
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
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

Authors:
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
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

Authors:
Rossi R, Moore M, Torelli S, Ala P, Catapano F, Phadke R, Morgan J, Malhotra J, Muntoni F
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

Authors:
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
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

Authors:
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
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

Authors:
Desjardins C, Venkatesan R, O'Donnell E, Hall J, Russo R, Spring S, Tang K, Davis J, Weeden T, Zanotti S, Beskrovnaya O
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

Authors:
Iannaccone S, Phan H, Straub V, Muntoni F, Wolf D, Malhotra J, Chu R, Darton E, Mercuri E
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

Authors:
Yaworski A, Duong T, Low J, Gee R, Watson K, Buu M, Kaufman B, Klotz J, Day J, Guzman J, Tesi Rocha C
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.

O.14 EEV-Conjugated PMO results in nuclear foci reduction and aberrant splicing correction in myotonic dystrophy cell and animal models

Authors:
Grigenrath M, Estrella N, Hicks A, Shen X, Wysk M, Kheirabadi M, Streeter M, Lian W, Liu N, Blake S, Brennan C, Li N, Batagui V, Oye K, Gao N, Wang D, Qian X, Sethuraman N
Citation:
Neuromuscul Disord. 2022;32(Suppl 1):S96. doi:/10.1016/j.nmd.2022.07.224
Epub:
Not Epub
Abstract:
Myotonic dystrophy type 1 (DM1) is the most common adult-onset muscular dystrophy, manifesting in multisystemic effects...
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: patient-derived muytoubes, mice
Citation Extract:
Grigenrath M, Estrella N, Hicks A, Shen X, Wysk M, Kheirabadi M, Streeter M, Lian W, Liu N, Blake S, Brennan C, Li N, Batagui V, Oye K, Gao N, Wang D, Qian X, Sethuraman N. O.14 EEV-Conjugated PMO results in nuclear foci reduction and aberrant splicing correction in myotonic dystrophy cell and animal models. Neuromuscul Disord. 2022;32(Suppl 1):S96. doi:/10.1016/j.nmd.2022.07.224.

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