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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 9947 scientific papers returned from the database with the search filters currently being used below.

If you know of a publication that is not in this database and you feel it should be, please submit a new publication to the database and we'll get it in there.

Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy

Authors:
Frank DE, Schnell FJ, Akana C, El-Husayni SH, Desjardins CA, Morgan J, Charleston JS, Sardone V, Domingos J, Dickson G, Straub V, Guglieri M, Mercuri E, Servais L, Muntoni F; SKIP-NMD Study Group
Citation:
Neurology. 2020 Mar 5. pii: 10.1212/WNL.0000000000009233. doi: 10.1212/WNL.0000000000009233. [Epub ahead of print]
Abstract:
Objective: To report safety, pharmacokinetics, exon 53 skipping, and dystrophin expression in golodirsen-treated patients with...
Delivery Method:
i.v. infusion
Organism or Cell Type:
human
Citation Extract:
Frank DE, Schnell FJ, Akana C, El-Husayni SH, Desjardins CA, Morgan J, Charleston JS, Sardone V, Domingos J, Dickson G, Straub V, Guglieri M, Mercuri E, Servais L, Muntoni F; SKIP-NMD Study Group. Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy. Neurology. 2020 Mar 5. pii: 10.1212/WNL.0000000000009233. doi: 10.1212/WNL.0000000000009233. [Epub ahead of print].

The RNA-binding protein DAZL functions as repressor and activator of mRNA translation during oocyte maturation

Authors:
Yang C-R, Rajkovic G, Daldello EM, Luong XG, Chen J, Conti M
Citation:
Nat Comm. 2020;11:1399. doi:10.1038/s41467-020-15209-9
Abstract:
Deleted in azoospermia-like (DAZL) is an RNA-binding protein critical for gamete development. In full-grown oocytes, the DAZL...
Delivery Method:
microinjection
Organism or Cell Type:
mouse oocyte
Citation Extract:
Yang C-R, Rajkovic G, Daldello EM, Luong XG, Chen J, Conti M. The RNA-binding protein DAZL functions as repressor and activator of mRNA translation during oocyte maturation. Nat Comm. 2020;11:1399. doi:10.1038/s41467-020-15209-9.

Pannexin-1 mediates fluid shear stress-sensitive purinergic signaling and cyst growth in polycystic kidney disease

Authors:
Verschuren EHJ, Rigalli JP, Castenmiller C, Rohrbach MU, Bindels RJM, Peters DJM, Arjona FJ, Hoenderop JGJ
Citation:
FASEB J. 2020 Mar 11. doi: 10.1096/fj.201902901R. [Epub ahead of print]
Abstract:
Tubular ATP release is regulated by mechanosensation of fluid shear stress (FSS). Polycystin-1/polycystin-2 (PC1/PC2) functions...
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Verschuren EHJ, Rigalli JP, Castenmiller C, Rohrbach MU, Bindels RJM, Peters DJM, Arjona FJ, Hoenderop JGJ. Pannexin-1 mediates fluid shear stress-sensitive purinergic signaling and cyst growth in polycystic kidney disease. FASEB J. 2020 Mar 11. doi: 10.1096/fj.201902901R. [Epub ahead of print].

U1 snRNP regulates chromatin retention of noncoding RNAs

Authors:
Yin Y, Lu JY, Zhang X, Shao W, Xu Y, Li P, Hong Y, Cui L, Shan G, Tian B, Zhang QC, Shen X
Citation:
Nature. 2020;[Epub ahead of print] doi:10.1038/s41586-020-2105-3
Abstract:
Long noncoding RNAs (lncRNAs) and promoter- or enhancer-associated unstable transcripts locate preferentially to chromatin,...
Citation Extract:
Yin Y, Lu JY, Zhang X, Shao W, Xu Y, Li P, Hong Y, Cui L, Shan G, Tian B, Zhang QC, Shen X. U1 snRNP regulates chromatin retention of noncoding RNAs. Nature. 2020;[Epub ahead of print] doi:10.1038/s41586-020-2105-3.

Glycine enhances satellite cell proliferation, cell transplantation and oligonucleotide efficacy in dystrophic muscle

Authors:
Lin C, Han G, Ning H, Song J, Ran N, Yi X, Seow Y, Yin HF
Citation:
2020 Mar 10. pii: S1525-0016(20)30137-4. doi: 10.1016/j.ymthe.2020.03.003. [Epub ahead of print]
Abstract:
The need to distribute therapy evenly systemically throughout the large muscle volume within the body makes Duchenne muscular...
Organism or Cell Type:
mice
Citation Extract:
Lin C, Han G, Ning H, Song J, Ran N, Yi X, Seow Y, Yin HF. Glycine enhances satellite cell proliferation, cell transplantation and oligonucleotide efficacy in dystrophic muscle. 2020 Mar 10. pii: S1525-0016(20)30137-4. doi: 10.1016/j.ymthe.2020.03.003. [Epub ahead of print].

Structural and mechanistic insights into secretagogin-mediated exocytosis

Authors:
Qin J, Liu Q, Liu Z, Pan Y-Z, Sifuentes-Dominguez L, Stepien KP, Wang Y, Tu Y, Tan S, Wang Y, Sun Q, Mo X, Rizo J, Burstein E, Jia D
Citation:
Proc Nat Acad Sci USA. 2020;201919698;[Epub ahead of print] doi:10.1073/pnas.1919698117
Abstract:
Secretagogin (SCGN) is a hexa–EF-hand protein that is highly expressed in the pancreas, brain, and gastrointestinal tract. SCGN...
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Qin J, Liu Q, Liu Z, Pan Y-Z, Sifuentes-Dominguez L, Stepien KP, Wang Y, Tu Y, Tan S, Wang Y, Sun Q, Mo X, Rizo J, Burstein E, Jia D. Structural and mechanistic insights into secretagogin-mediated exocytosis. Proc Nat Acad Sci USA. 2020;201919698;[Epub ahead of print] doi:10.1073/pnas.1919698117.

Genomic Repeats Categorize Genes with Distinct Functions for Orchestrated Regulation

Authors:
Lu JY, Shao W, Chang L, Ramalho-Santos M, Sun Y, Shen X
Citation:
Cell Rep. 2020;30(10):3296-3311.e5 doi:10.1016/j.celrep.2020.02.048
Abstract:
Repetitive elements are abundantly distributed in mammalian genomes. Here, we reveal a striking association between repeat...
Organism or Cell Type:
cell culture: embryonic stem cells (ESCs)
Citation Extract:
Lu JY, Shao W, Chang L, Ramalho-Santos M, Sun Y, Shen X. Genomic Repeats Categorize Genes with Distinct Functions for Orchestrated Regulation. Cell Rep. 2020;30(10):3296-3311.e5 doi:10.1016/j.celrep.2020.02.048.

Deficiency in the endocytic adaptor proteins PHETA1/2 impair renal and craniofacial development

Authors:
Ates KM, Wang T, Moreland T, Veeranan-Karmegam R, Ma M, Jeter C, Anand P, Wenzel W, Kim H-G, Wolfe LA, Joshi A. Stephen JA, Adams DR, Markello T, Tifft CJ, Settlage R, Gahl WA, Gonsalvez GB, Malicdan MC, Flanagan-Steet H, Pan YA
Citation:
Dis Models Mec. 2020;[Epub] doi:10.1242/dmm.041913
Abstract:
A critical barrier in the treatment of endosomal and lysosomal diseases is the lack of understanding of the in vivo functions...
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Ates KM, Wang T, Moreland T, Veeranan-Karmegam R, Ma M, Jeter C, Anand P, Wenzel W, Kim H-G, Wolfe LA, Joshi A. Stephen JA, Adams DR, Markello T, Tifft CJ, Settlage R, Gahl WA, Gonsalvez GB, Malicdan MC, Flanagan-Steet H, Pan YA. Deficiency in the endocytic adaptor proteins PHETA1/2 impair renal and craniofacial development. Dis Models Mec. 2020;[Epub] doi:10.1242/dmm.041913.

Histone variant H3.3 residue S31 is essential for Xenopus gastrulation regardless of the deposition pathway

Authors:
Sitbon D, Boyarchuk E, Dingli F, Loew D, Almouzni G
Citation:
Nat Comm. 2020;11:1256. doi:10.1038/s41467-020-15084-4
Abstract:
Vertebrates exhibit specific requirements for replicative H3 and non-replicative H3.3 variants during development. To...
Delivery Method:
microinjection
Organism or Cell Type:
Xenopus laevis
Citation Extract:
Sitbon D, Boyarchuk E, Dingli F, Loew D, Almouzni G. Histone variant H3.3 residue S31 is essential for Xenopus gastrulation regardless of the deposition pathway. Nat Comm. 2020;11:1256. doi:10.1038/s41467-020-15084-4.

Modeling Bainbridge-Ropers Syndrome in Xenopus laevis Embryos

Authors:
Lichtig H, Artamonov A, Polevoy H, Reid CD, Bielas SL, Frank D
Citation:
Front Physiol. 2020 Feb 18;11:75. doi: 10.3389/fphys.2020.00075. eCollection 2020
Abstract:
The Additional sex combs-like (ASXL1-3) genes are linked to human neurodevelopmental disorders. The de novo truncating variants...
Delivery Method:
microinjection
Organism or Cell Type:
Xenopus laevis
Citation Extract:
Lichtig H, Artamonov A, Polevoy H, Reid CD, Bielas SL, Frank D. Modeling Bainbridge-Ropers Syndrome in Xenopus laevis Embryos. Front Physiol. 2020 Feb 18;11:75. doi: 10.3389/fphys.2020.00075. eCollection 2020.

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