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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 12245 scientific papers returned from the database with the search filters currently being used below.

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.

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...
Link:
https://www.sciencedirect.com/science/article/pii/S0960896622004503
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...
Link:
https://www.sciencedirect.com/science/article/pii/S0960896622004527
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...
Link:
https://www.sciencedirect.com/science/article/pii/S0960896622004266
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.

INTS13 variants causing a recessive developmental ciliopathy disrupt assembly of the Integrator complex

Authors:
Mascibroda LG, Shboul M, Elrod ND, Colleaux L, Hamamy H, Huang K-L, Peart N, Singh MK, Lee H, Merriman B, Jodoin JN, Sitaram P, Lee LA, Fathalla R, Al-Rawashdeh B, Ababneh O, El-Khateeb M, Escande-Beillard N, Nelson SF, Wu Y, Tong L, Kenney LJ, Roy Su, Russell WK, Amiel J, Reversade B, Wagner EJ
Citation:
Nat Commun. 2022;13(1),6054. doi:10.1038/s41467-022-33547-8
Epub:
Not Epub
Abstract:
Oral-facial-digital (OFD) syndromes are a heterogeneous group of congenital disorders characterized by malformations of the...
Link:
https://www.nature.com/articles/s41467-022-33547-8
Delivery Method:
microinjection
Organism or Cell Type:
Xenopus laevis
Citation Extract:
Mascibroda LG, Shboul M, Elrod ND, Colleaux L, Hamamy H, Huang K-L, Peart N, Singh MK, Lee H, Merriman B, Jodoin JN, Sitaram P, Lee LA, Fathalla R, Al-Rawashdeh B, Ababneh O, El-Khateeb M, Escande-Beillard N, Nelson SF, Wu Y, Tong L, Kenney LJ, Roy Su, Russell WK, Amiel J, Reversade B, Wagner EJ. INTS13 variants causing a recessive developmental ciliopathy disrupt assembly of the Integrator complex. Nat Commun. 2022;13(1),6054. doi:10.1038/s41467-022-33547-8.

Trim33 conditions the lifespan of primitive macrophages and onset of definitive macrophage production

Authors:
Demy DL, Touret A-L, Lancino M, Tauzin M, Capuana L, Pierre C, Herbomel P
Citation:
Development. 2022;149(18):dev200835. doi:10.1242/dev.200835
Epub:
Not Epub
Abstract:
Trim33 (Tif1γ) is a transcriptional regulator that is notably involved in several aspects of hematopoiesis. It is essential for...
Link:
https://journals.biologists.com/dev/article-abstract/149/18/dev200835/276648/Trim33-conditions-the-lifespan-of-primitive
Organism or Cell Type:
zebrafish
Citation Extract:
Demy DL, Touret A-L, Lancino M, Tauzin M, Capuana L, Pierre C, Herbomel P. Trim33 conditions the lifespan of primitive macrophages and onset of definitive macrophage production. Development. 2022;149(18):dev200835. doi:10.1242/dev.200835.

N-myc downstream regulated gene 1 (ndrg1) functions as a molecular switch for cellular adaptation to hypoxia

Authors:
Park JS. Gabel AM, Kassir P, Kang L, Chowdhary PK, Osei-Ntansah A, Tran ND, Viswanathan S, Canales B, Ding P, Lee Y-S, Brewster R
Citation:
eLife. 2022;11:e74031. doi:10.7554/eLife.74031
Epub:
Not Epub
Abstract:
Lack of oxygen (hypoxia and anoxia) is detrimental to cell function and survival and underlies many disease conditions. Hence,...
Link:
https://elifesciences.org/articles/74031
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Park JS. Gabel AM, Kassir P, Kang L, Chowdhary PK, Osei-Ntansah A, Tran ND, Viswanathan S, Canales B, Ding P, Lee Y-S, Brewster R. N-myc downstream regulated gene 1 (ndrg1) functions as a molecular switch for cellular adaptation to hypoxia. eLife. 2022;11:e74031. doi:10.7554/eLife.74031.

Collective cell migration during optic cup formation features changing cell-matrix interactions linked to matrix topology

Authors:
Soans KG, Ramos AP, Sidhaye J, Krishna A, Solomatina A, Hoffmann KB, Schlüßler R, Guck J, Sbalzarini IF, Modes CD, Norden C
Citation:
Curr Biol. 2022;[Epub] doi:10.1016/j.cub.2022.09.034
Epub:
Yes
Abstract:
Cell migration is crucial for organismal development and shapes organisms in health and disease. Although a lot of research has...
Link:
https://www.cell.com/current-biology/pdf/S0960-9822(22)01503-2.pdf
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Soans KG, Ramos AP, Sidhaye J, Krishna A, Solomatina A, Hoffmann KB, Schlüßler R, Guck J, Sbalzarini IF, Modes CD, Norden C. Collective cell migration during optic cup formation features changing cell-matrix interactions linked to matrix topology. Curr Biol. 2022;[Epub] doi:10.1016/j.cub.2022.09.034.

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