You are here

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 12376 scientific papers returned from the database with the search filters currently being used below.

Polycystin-1 regulates bone development through an interaction with the transcriptional co-activator taz

Authors:
Merrick D, Mistry K, Wu J, Gresko N, Baggs JE, Hogenesch JB, Sun Z, Caplan MJ
Citation:
Hum Mol Genet. 2018 Sep 12. doi: 10.1093/hmg/ddy322. [Epub ahead of print]
Epub:
Yes
Abstract:
Polycystin-1 (PC1), encoded by the PKD1 gene that is mutated in Autosomal Dominant Polycystic Kidney disease, regulates a...
Link:
https://academic.oup.com/hmg/advance-article-abstract/doi/10.1093/hmg/ddy322/5095320
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Merrick D, Mistry K, Wu J, Gresko N, Baggs JE, Hogenesch JB, Sun Z, Caplan MJ. Polycystin-1 regulates bone development through an interaction with the transcriptional co-activator taz. Hum Mol Genet. 2018 Sep 12. doi: 10.1093/hmg/ddy322. [Epub ahead of print].

A Japanese phase I/II study of NS-065/NCNP-01, exon 53 skipping drug, in patients with Duchenne muscular dystrophy - a dose-finding study

Authors:
Komaki H, Takeshima Y, Matsumura T, Ozasa S, Funato M, Egawa Y, Takeda S
Citation:
Neuromusc Disord. 2018;28:P129. doi:10.1016/j.nmd.2018.06.157
Epub:
Not Epub
Abstract:
no abstract available
Link:
https://www.nmd-journal.com/article/S0960-8966(18)30720-X/fulltext
Delivery Method:
i.v. infusion
Organism or Cell Type:
human
Citation Extract:
Komaki H, Takeshima Y, Matsumura T, Ozasa S, Funato M, Egawa Y, Takeda S. A Japanese phase I/II study of NS-065/NCNP-01, exon 53 skipping drug, in patients with Duchenne muscular dystrophy - a dose-finding study. Neuromusc Disord. 2018;28:P129. doi:10.1016/j.nmd.2018.06.157.

Respiratory function decline in eteplirsen-treated patients diverges from natural history comparators over time

Authors:
Khan N, Han L, Kinane B, Gordish-Dressman H, Lowes L, McDonald C
Citation:
Neuromusc Disord. 2018;28:P126. doi:10.1016/j.nmd.2018.06.154
Epub:
Not Epub
Abstract:
no abstract available
Link:
https://www.nmd-journal.com/article/S0960-8966(18)30717-X/fulltext
Delivery Method:
i.v. infusion
Organism or Cell Type:
human
Citation Extract:
Khan N, Han L, Kinane B, Gordish-Dressman H, Lowes L, McDonald C. Respiratory function decline in eteplirsen-treated patients diverges from natural history comparators over time. Neuromusc Disord. 2018;28:P126. doi:10.1016/j.nmd.2018.06.154.

A phase II, dose finding study to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of NS-065/NCNP-01 in boys with Duchenne muscular dystrophy

Authors:
Clemens P, Rao V, Connolly A, Harper A, Mah J, Smith E, McDonald C, Morgenroth L, Osaki H, Hoffman E
Citation:
Neuromusc Disord. 2018;28(Suppl 2):S67-S68. doi:10.1016/j.nmd.2018.06.156
Epub:
Not Epub
Abstract:
no abstract available
Link:
https://www.nmd-journal.com/article/S0960-8966(18)30720-X/fulltext
Delivery Method:
i.v. infusion
Organism or Cell Type:
human
Citation Extract:
Clemens P, Rao V, Connolly A, Harper A, Mah J, Smith E, McDonald C, Morgenroth L, Osaki H, Hoffman E. A phase II, dose finding study to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of NS-065/NCNP-01 in boys with Duchenne muscular dystrophy. Neuromusc Disord. 2018;28(Suppl 2):S67-S68. doi:10.1016/j.nmd.2018.06.156.

Eteplirsen treatment attenuates respiratory decline in ambulatory and non-ambulatory patients with Duchenne muscular dystrophy

Authors:
Khan N, Han L, Kinane B, Gordish-Dressman H, Lowes L, McDonald C
Citation:
Neuromusc Disord. 2018;28:P125. doi:10.1016/j.nmd.2018.06.153
Epub:
Not Epub
Abstract:
no abstract available
Link:
https://www.nmd-journal.com/article/S0960-8966(18)30716-8/fulltext
Delivery Method:
i.v. infusion
Organism or Cell Type:
human
Citation Extract:
Khan N, Han L, Kinane B, Gordish-Dressman H, Lowes L, McDonald C. Eteplirsen treatment attenuates respiratory decline in ambulatory and non-ambulatory patients with Duchenne muscular dystrophy. Neuromusc Disord. 2018;28:P125. doi:10.1016/j.nmd.2018.06.153.

Eteplirsen is well tolerated in adults with mild or moderate renal impairment

Authors:
Mix C, Naughton N, Forte S
Citation:
Neuromusc Disord. 2018;28:P124. doi:10.1016/j.nmd.2018.06.152
Epub:
Not Epub
Abstract:
no abstract available
Link:
https://www.nmd-journal.com/article/S0960-8966(18)30715-6/fulltext
Delivery Method:
i.v. infusion
Organism or Cell Type:
human
Citation Extract:
Mix C, Naughton N, Forte S. Eteplirsen is well tolerated in adults with mild or moderate renal impairment. Neuromusc Disord. 2018;28:P124. doi:10.1016/j.nmd.2018.06.152.

Humoral and cell mediated immune response to new dystrophin after morpholino-induced exon skipping therapy in dystrophin-deficient mdx mice

Authors:
Nagaraju K, Vila M, Novak J, Boehler J, Hogarth M, Zhang A, Kinder T, Mazala D, Klimek MB, Fiorillo A, van den Anker J, Hathout Y, Hoffman E, Partridge T
Citation:
Neuromusc Disord. 2018;28:S90. doi:10.1016/j.nmd.2018.06.238
Epub:
Not Epub
Abstract:
Exon skipping is a promising genetic therapeutic strategy for restoring dystrophin expression in the treatment of Duchenne...
Link:
https://www.nmd-journal.com/article/S0960-8966(18)30801-0/fulltext
Organism or Cell Type:
mice mdx
Citation Extract:
Nagaraju K, Vila M, Novak J, Boehler J, Hogarth M, Zhang A, Kinder T, Mazala D, Klimek MB, Fiorillo A, van den Anker J, Hathout Y, Hoffman E, Partridge T. Humoral and cell mediated immune response to new dystrophin after morpholino-induced exon skipping therapy in dystrophin-deficient mdx mice. Neuromusc Disord. 2018;28:S90. doi:10.1016/j.nmd.2018.06.238.

Bighead is a Wnt antagonist secreted by the Xenopus Spemann organizer that promotes Lrp6 endocytosis

Authors:
Ding Y, Colozza G, Sosa EA, Moriyama Y, Rundle S, Salwinski L, De Robertis EM
Citation:
Proc Natl Acad Sci U S A. 2018 Sep 12. pii: 201812117. doi: 10.1073/pnas.1812117115. [Epub ahead of print]
Epub:
Yes
Abstract:
The Xenopus laevis embryo has been subjected to almost saturating screens for molecules specifically expressed in dorsal...
Link:
http://www.pnas.org/content/early/2018/09/11/1812117115
Delivery Method:
microinjection
Organism or Cell Type:
Xenopus laevis
Citation Extract:
Ding Y, Colozza G, Sosa EA, Moriyama Y, Rundle S, Salwinski L, De Robertis EM. Bighead is a Wnt antagonist secreted by the Xenopus Spemann organizer that promotes Lrp6 endocytosis. Proc Natl Acad Sci U S A. 2018 Sep 12. pii: 201812117. doi: 10.1073/pnas.1812117115. [Epub ahead of print].

Zebrafish snai2 mutants fail to phenocopy morphant phenotypes

Authors:
Bickers C, Española SD, Grainger S, Pouget C, Traver D
Citation:
PLoS One. 2018 Sep 12;13(9):e0202747. doi: 10.1371/journal.pone.0202747. eCollection 2018
Epub:
Not Epub
Abstract:
Snail2 is a zinc-finger transcription factor best known to repress expression of genes encoding cell adherence proteins to...
Link:
https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0202747
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Bickers C, Española SD, Grainger S, Pouget C, Traver D. Zebrafish snai2 mutants fail to phenocopy morphant phenotypes. PLoS One. 2018 Sep 12;13(9):e0202747. doi: 10.1371/journal.pone.0202747. eCollection 2018.

The Human FSGS-Causing ANLN R431C Mutation Induces Dysregulated PI3K/AKT/mTOR/Rac1 Signaling in Podocytes

Authors:
Hall G, Lane BM, Khan K, Pediaditakis I, Xiao J, Wu G, Wang L, Kovalik ME, Chryst-Stangl M, Davis EE, Spurney RF, Gbadegesin RA
Citation:
J Am Soc Nephrol. 2018 Aug;29(8):2110-2122. doi: 10.1681/ASN.2017121338. Epub 2018 Jul 12
Epub:
Not Epub
Abstract:
BACKGROUND: We previously reported that mutations in the anillin (ANLN) gene cause familial forms of FSGS. ANLN is an F-actin...
Link:
https://jasn.asnjournals.org/content/early/2018/07/11/ASN.2017121338
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Hall G, Lane BM, Khan K, Pediaditakis I, Xiao J, Wu G, Wang L, Kovalik ME, Chryst-Stangl M, Davis EE, Spurney RF, Gbadegesin RA. The Human FSGS-Causing ANLN R431C Mutation Induces Dysregulated PI3K/AKT/mTOR/Rac1 Signaling in Podocytes. J Am Soc Nephrol. 2018 Aug;29(8):2110-2122. doi: 10.1681/ASN.2017121338. Epub 2018 Jul 12.

Pages