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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.

Human genetic defects in SRP19 and SRPRA cause severe congenital neutropenia with distinctive proteome changes

Authors:
Linder MI, Mizoguchi Y, Hesse S, Csaba G, Tatematsu M, Łyszkiewicz M, Ziȩtara N, Jeske T, Hastreiter M, Rohlfs M, Liu Y, Grabowski P, Ahomaa K, Maier-Begandt D, Schwestka M, Pazhakh V, Isiaku AI, Miranda BB, Blombery P, Saito MK, Rusha E, Alizadeh Z, Pourpak Z, Kobayashi M, Rezaei N, Unal E, Hauck F, Drukker M, Walzog B, Rappsilber J, Zimmer R, Lieschke GJ, Klein C
Citation:
Blood. 2023;141(6):645-58. doi:10.1182/blood.2022016783
Epub:
Not Epub
Abstract:
The mechanisms of coordinated changes in proteome composition and their relevance for the differentiation of neutrophil...
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Linder MI, Mizoguchi Y, Hesse S, Csaba G, Tatematsu M, Łyszkiewicz M, Ziȩtara N, Jeske T, Hastreiter M, Rohlfs M, Liu Y, Grabowski P, Ahomaa K, Maier-Begandt D, Schwestka M, Pazhakh V, Isiaku AI, Miranda BB, Blombery P, Saito MK, Rusha E, Alizadeh Z, Pourpak Z, Kobayashi M, Rezaei N, Unal E, Hauck F, Drukker M, Walzog B, Rappsilber J, Zimmer R, Lieschke GJ, Klein C. Human genetic defects in SRP19 and SRPRA cause severe congenital neutropenia with distinctive proteome changes. Blood. 2023;141(6):645-58. doi:10.1182/blood.2022016783.

Converging Role for REEP1/SPG31 in Oxidative Stress

Authors:
Naef V, Meschini MC, Tessa A, Morani F, Corsinovi D, Ogi A, Marchese M, Ori M, Santorelli FM, Doccini S
Citation:
Int J Mol Sci. 2023;24(4):3527. doi:10.3390/ijms24043527
Epub:
Not Epub
Abstract:
Mutations in the receptor expression-enhancing protein 1 gene (REEP1) are associated with hereditary spastic paraplegia type 31...
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Naef V, Meschini MC, Tessa A, Morani F, Corsinovi D, Ogi A, Marchese M, Ori M, Santorelli FM, Doccini S. Converging Role for REEP1/SPG31 in Oxidative Stress. Int J Mol Sci. 2023;24(4):3527. doi:10.3390/ijms24043527.

Systemic administration of the antisense oligonucleotide NS-089/NCNP-02 for skipping of exon 44 in patients with Duchenne muscular dystrophy: study protocol for a phase I/II clinical trial

Authors:
Ishizuka T, Komaki H, Asahina Y, Nakamura H, Motohashi N, Takeshita E, Motohashi Y, Ishiyama A, Yonee C, Maruyama S, Hida E, Aoki Y
Citation:
medRxiv. 2023;[preprint] doi:10.1101/2023.02.06.23285500
Epub:
Not Epub
Abstract:
Aim: The purpose of this study is to evaluate the safety and pharmacokinetics of the novel morpholino oligomer NS-089/NCNP-02...
Delivery Method:
i.v. infusion
Organism or Cell Type:
human
Citation Extract:
Ishizuka T, Komaki H, Asahina Y, Nakamura H, Motohashi N, Takeshita E, Motohashi Y, Ishiyama A, Yonee C, Maruyama S, Hida E, Aoki Y. Systemic administration of the antisense oligonucleotide NS-089/NCNP-02 for skipping of exon 44 in patients with Duchenne muscular dystrophy: study protocol for a phase I/II clinical trial. medRxiv. 2023;[preprint] doi:10.1101/2023.02.06.23285500 .

Specific Activation of Yamanaka Factors via HSF1 Signaling in the Early Stage of Zebrafish Optic Nerve Regeneration

Authors:
Sugitani K, Mokuya T, Homma S, Maeda M, Konno A, Ogai K
Citation:
Int J Mol Sci. 2023;24(4):3253. doi:10.3390/ijms24043253
Epub:
Not Epub
Abstract:
In contrast to the case in mammals, the fish optic nerve can spontaneously regenerate and visual function can be fully restored...
Delivery Method:
Vivo-Morpholino intraocular injection
Organism or Cell Type:
zebrafish
Citation Extract:
Sugitani K, Mokuya T, Homma S, Maeda M, Konno A, Ogai K. Specific Activation of Yamanaka Factors via HSF1 Signaling in the Early Stage of Zebrafish Optic Nerve Regeneration. Int J Mol Sci. 2023;24(4):3253. doi:10.3390/ijms24043253.

Cpeb1b-mediated cytoplasmic polyadenylation of shha mRNA modulates zebrafish definitive hematopoiesis

Authors:
Heng J, Shi B, Zhou JY, Zhang Y, Ma D, Yang YG, Liu F
Citation:
Proc Natl Acad Sci U S A. 2023 Feb 14;120(7):e2212212120. doi: 10.1073/pnas.2212212120. Epub 2023 Feb 6
Epub:
Not Epub
Abstract:
During vertebrate embryogenesis, hematopoietic stem and progenitor cell (HSPC) production through endothelial-to-hematopoietic...
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Heng J, Shi B, Zhou JY, Zhang Y, Ma D, Yang YG, Liu F. Cpeb1b-mediated cytoplasmic polyadenylation of shha mRNA modulates zebrafish definitive hematopoiesis. Proc Natl Acad Sci U S A. 2023 Feb 14;120(7):e2212212120. doi: 10.1073/pnas.2212212120. Epub 2023 Feb 6.

Gaucher disease protects against tuberculosis

Authors:
Fan J, Hale VL, Lelieveld LT, Ramakrishna L
Citation:
Proc Natl Acad Sci USA. 2023;120(7):e2217673120 doi:10.1073/pnas.2217673120
Epub:
Not Epub
Abstract:
Biallelic mutations in the glucocerebrosidase (GBA1) gene cause Gaucher disease, characterized by lysosomal accumulation of...
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Fan J, Hale VL, Lelieveld LT, Ramakrishna L. Gaucher disease protects against tuberculosis. Proc Natl Acad Sci USA. 2023;120(7):e2217673120 doi:10.1073/pnas.2217673120.

Ubiquitylation of BBSome is required for ciliary assembly and signaling

Authors:
Chiuso F, Delle Donne R, Giamundo G, Rinaldi L, Borzacchiello D, Moraca F, Intartaglia D, Iannucci R, Senatore E, Lignitto L, Garbi C, Conflitti P, Catalanotti B, Conte I, Feliciello A
Citation:
Ubiquitylation of BBSome is required for ciliary assembly and signaling. EMBO Rep. 2023 Feb 6:e55571. doi:10.15252/embr.202255571. Online ahead of print
Epub:
Yes
Abstract:
Bardet-Biedl syndrome (BBS) is a ciliopathy characterized by retinal degeneration, obesity, renal abnormalities, postaxial...
Delivery Method:
microinjection
Organism or Cell Type:
Oryzias latipes (medaka fish)
Citation Extract:
Chiuso F, Delle Donne R, Giamundo G, Rinaldi L, Borzacchiello D, Moraca F, Intartaglia D, Iannucci R, Senatore E, Lignitto L, Garbi C, Conflitti P, Catalanotti B, Conte I, Feliciello A. Ubiquitylation of BBSome is required for ciliary assembly and signaling. Ubiquitylation of BBSome is required for ciliary assembly and signaling. EMBO Rep. 2023 Feb 6:e55571. doi:10.15252/embr.202255571. Online ahead of print.

Genetic Variants in ARHGEF6 Cause Congenital Anomalies of the Kidneys and Urinary Tract in Humans, Mice, and Frogs

Authors:
Klämbt V, Buerger F, Wang C, Naert T, Richter K, Nauth T, Weiss AC, Sieckmann T, Lai E, Connaughton D, Seltzsam S, Mann N, Majmundar A, Wu CH, Onuchic-Whitford A, Shril S, Schneider S, Schierbaum L, Dai R, Bekheirnia MR, Joosten M, Shlomovitz O, Vivante A, Banne E, Mane S, Lifton RP, Kirschner K, Kispert A, Rosenberger G, Fischer KD, Lienkamp S, Zegers M, Hildebrandt F
Citation:
J Am Soc Nephrol. 2022 Nov 22:ASN.2022010050. doi: 10.1681/ASN.2022010050. Online ahead of print
Epub:
Yes
Abstract:
Background About 40 disease genes have been described to date for isolated congenital anomalies of the kidneys and urinary...
Organism or Cell Type:
Xenopus
Citation Extract:
Klämbt V, Buerger F, Wang C, Naert T, Richter K, Nauth T, Weiss AC, Sieckmann T, Lai E, Connaughton D, Seltzsam S, Mann N, Majmundar A, Wu CH, Onuchic-Whitford A, Shril S, Schneider S, Schierbaum L, Dai R, Bekheirnia MR, Joosten M, Shlomovitz O, Vivante A, Banne E, Mane S, Lifton RP, Kirschner K, Kispert A, Rosenberger G, Fischer KD, Lienkamp S, Zegers M, Hildebrandt F . Genetic Variants in ARHGEF6 Cause Congenital Anomalies of the Kidneys and Urinary Tract in Humans, Mice, and Frogs. J Am Soc Nephrol. 2022 Nov 22:ASN.2022010050. doi: 10.1681/ASN.2022010050. Online ahead of print.

Confined environments induce polarized paraspeckle condensates

Authors:
Todorovski V, McCluggage F, Li Y, Meid A, Spatz JP, Holle AW, Fox AH, Choi Y
Citation:
Commun Biol 6, 145 (2023). doi:10.1038/s42003-023-04528-4
Epub:
Not Epub
Abstract:
Cancer cells experience confinement as they navigate the tumour microenvironment during metastasis. Recent studies have...
Delivery Method:
electroporation
Organism or Cell Type:
cell culture: MDA-MB-231 human breast cancer cells
Citation Extract:
Todorovski V, McCluggage F, Li Y, Meid A, Spatz JP, Holle AW, Fox AH, Choi Y. Confined environments induce polarized paraspeckle condensates. Commun Biol 6, 145 (2023). doi:10.1038/s42003-023-04528-4.

Tmem88 confines ectodermal Wnt2bb signaling in pharyngeal arch artery progenitors for balancing cell cycle progression and cell fate decision

Authors:
Zhang M, Liu J, Mao A, Ning G, Cao Y, Zhang W, Wang Q
Citation:
Nat Cardiovasc Res. 2023;[epub] doi:10.1038/s44161-023-00215-z
Epub:
Not Epub
Abstract:
Pharyngeal arch artery (PAA) progenitors undergo proliferative expansion and angioblast differentiation to build vessels...
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Zhang M, Liu J, Mao A, Ning G, Cao Y, Zhang W, Wang Q. Tmem88 confines ectodermal Wnt2bb signaling in pharyngeal arch artery progenitors for balancing cell cycle progression and cell fate decision. Nat Cardiovasc Res. 2023;[epub] doi:10.1038/s44161-023-00215-z.

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