jonmoulton @jonmoulton@qoto.org

The U1 antisense morpholino oligonucleotide (AMO) disrupts U1 snRNP structure to promote intronic PCPA modification of pre-mRNAs. https://www.jbc.org/article/S0021-9258(23)01882-3/fulltext #Morpholino

Cartilage regeneration in zebrafish depends on Nrg1/ErbB signaling pathway. https://www.frontiersin.org/articles/10.3389/fcell.2023.1123299/full #Morpholino

Genetic deletion of the inflammation-regulated microRNA miR-146a increases exon-skipping mediated dystrophin restoration in Duchenne muscular dystrophy. https://www.biorxiv.org/content/10.1101/2023.05.09.540042v1 #Morpholino

A heterozygous mutation in UBE2H in a patient with developmental delay leads to an aberrant brain development in zebrafish. https://humgenomics.biomedcentral.com/articles/10.1186/s40246-023-00491-7 #Morpholino

Embryonic Ethanol but Not Cannabinoid Exposure Affects Zebrafish Cardiac Development via Agrin and Sonic Hedgehog Interaction. https://www.mdpi.com/2073-4409/12/9/1327 #Morpholino

The challenge of dissecting gene function in model organisms: Tools to characterize genetic mutants and assess transcriptional adaptation in zebrafish. https://www.sciencedirect.com/science/article/abs/pii/S0091679X22002072 #Morpholino

Role of MicroRNAs and their Downstream Target Transcription Factors in Zebrafish Thrombopoiesis. https://www.researchsquare.com/article/rs-2807790/v1 #Morpholino

Npr3 regulates neural crest and cranial placode progenitors formation through its dual function as clearance and signaling receptor. https://elifesciences.org/articles/84036 #Morpholino

Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study. https://content.iospress.com/articles/journal-of-neuromuscular-diseases/jnd221656
#Morpholino

Molecular basis for Gβγ-mediated activation of phosphoinositide 3-kinase γ. https://www.biorxiv.org/content/10.1101/2023.05.04.539492v1
#Morpholino

The phosphatidylinositol (4,5)-bisphosphate-Rab35 axis regulates migrasome formation. https://www.nature.com/articles/s41422-023-00811-5 #Morpholino

In a zebrafish biomedical model of human Allan-Herndon-Dudley syndrome impaired MTH signaling leads to decreased neural cell diversity. https://www.frontiersin.org/articles/10.3389/fendo.2023.1157685/full #Morpholino

Epigenetic silencing of selected hypothalamic neuropeptides in narcolepsy with cataplexy. https://www.pnas.org/doi/10.1073/pnas.2220911120 #Morpholino

(Poster): Preclinical findings support the development of PYC-001
PYC-001, a peptide-conjugated phosphorodiamidate
morpholino oligomer for the treatment of autosomal
dominant optic atrophy
https://pyctx.com/wp-content/uploads/2023/05/20230424_ADOA-TIDES-Poster_FINAL-A4.pdf
preclinical peptide-#Morpholino (PPMO)

Glia maturation factor-γ is required for initiation and maintenance of hematopoietic stem and progenitor cells. https://stemcellres.biomedcentral.com/articles/10.1186/s13287-023-03328-1 #Morpholino

mRNA Location and Translation Rate Determine Protein Targeting to Dual Destinations. https://www.biorxiv.org/content/10.1101/2023.04.24.538105v1 #Morpholino

Epigenetic landscape reveals MECOM as an endothelial lineage regulator. https://www.nature.com/articles/s41467-023-38002-w #Morpholino

PIM2 kinase has a pivotal role in plasmablast generation and plasma cell survival, opening up novel treatment options in myeloma. https://www.sciencedirect.com/science/article/pii/S0006497122001471 Vivo-#Morpholino

Deciphering the pathogenesis of retinopathy associated with carnitine palmitoyltransferase I deficiency in zebrafish model. https://www.sciencedirect.com/science/article/abs/pii/S0006291X23005272 #Morpholino

Abrogation of MAP4K4 protein function causes congenital anomalies in humans and zebrafish. https://www.science.org/doi/10.1126/sciadv.ade0631 #Morpholino