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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 12132 scientific papers returned from the database with the search filters currently being used below.

Recent reconfiguration of an ancient developmental gene regulatory network in Heliocidaris sea urchins

Authors:
Davidson PL, Guo H, Swart JS, Massri AJ, Edgar A, Wang L, Berrio A, Devens HR, Koop D, Cisternas P, Zhang H, Zhang Y, Byrne M, Fan G, Wray GA
Citation:
Nat Ecol Evol. 2022;[Epub] doi:10.1038/s41559-022-01906-9
Epub:
Not Epub
Abstract:
Changes in developmental gene regulatory networks (dGRNs) underlie much of the diversity of life, but the evolutionary...
Link:
https://www.nature.com/articles/s41559-022-01906-9
Delivery Method:
microinjection
Organism or Cell Type:
Heliocidaris erythrogramma (sea urchin)
Citation Extract:
Davidson PL, Guo H, Swart JS, Massri AJ, Edgar A, Wang L, Berrio A, Devens HR, Koop D, Cisternas P, Zhang H, Zhang Y, Byrne M, Fan G, Wray GA. Recent reconfiguration of an ancient developmental gene regulatory network in Heliocidaris sea urchins. Nat Ecol Evol. 2022;[Epub] doi:10.1038/s41559-022-01906-9.

Germline-Specific Regulator of Mitochondrial Fusion is Required for Maintenance and Differentiation of Germline Stem and Progenitor Cells

Authors:
Zhang R, Tu YX, Ye D, Gu Z, Chen ZX, Sun Y
Citation:
Adv Sci (Weinh). 2022 Oct 18:e2203631. doi: 10.1002/advs.202203631. Online ahead of print
Epub:
Not Epub
Abstract:
Maintenance and differentiation of germline stem and progenitor cells (GSPCs) is important for sexual reproduction. Here, the...
Link:
https://onlinelibrary.wiley.com/doi/10.1002/advs.202203631
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Zhang R, Tu YX, Ye D, Gu Z, Chen ZX, Sun Y. Germline-Specific Regulator of Mitochondrial Fusion is Required for Maintenance and Differentiation of Germline Stem and Progenitor Cells. Adv Sci (Weinh). 2022 Oct 18:e2203631. doi: 10.1002/advs.202203631. Online ahead of print.

5-methylcytosine (m5C) RNA modification controls the innate immune response to virus infection by regulating type I interferons

Authors:
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
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...
Link:
https://www.pnas.org/doi/abs/10.1073/pnas.2123338119
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

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...
Link:
https://www.nature.com/articles/s41467-022-33836-2
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...
Link:
https://www.sciencedirect.com/science/article/pii/S0960896622005557
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...
Link:
https://www.sciencedirect.com/science/article/pii/S096089662200551X
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...
Link:
https://www.sciencedirect.com/science/article/pii/S0960896622004540
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...
Link:
https://www.sciencedirect.com/science/article/pii/S0960896622004382
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...
Link:
https://www.sciencedirect.com/science/article/pii/S0960896622004412
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...
Link:
https://www.sciencedirect.com/science/article/pii/S0960896622004497
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.

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