jonmoulton @jonmoulton@qoto.org

Improved Prediction of Antisense Oligonucleotide Efficacy for Exon Skipping Using Ensemble Learning and Feature Selection. https://www.preprints.org/manuscript/202303.0389/v1 #Morpholino

ASO targeting RBM3 temperature-controlled poison exon splicing prevents neurodegeneration in vivo. https://www.embopress.org/doi/full/10.15252/emmm.202217157
#Morpholino

An overview of recent US-approved gene therapies for Duchenne muscular dystrophy and their respective clinical development programs. https://link.springer.com/article/10.1007/s40267-023-00986-2 Review with #Morpholino $SRPT

Exosome-derived circCCAR1 promotes CD8 + T-cell dysfunction and anti-PD1 resistance in hepatocellular carcinoma. https://molecular-cancer.biomedcentral.com/articles/10.1186/s12943-023-01759-1 #Morpholino

Variations in the poly-histidine repeat motif of HOXA1 contribute to bicuspid aortic valve in mouse and zebrafish. https://www.nature.com/articles/s41467-023-37110-x #Morpholino

CLN3 deficiency leads to neurological and metabolic perturbations during early development. https://www.biorxiv.org/content/10.1101/2023.03.17.533107v1 #Morpholino

Self-transfecting GMO-PMO antisense chimera targeting Nanog enable gene silencing in vitro and suppresses tumor growth in 4T1 allografts in mouse. https://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(23)00070-7 #Morpholino

Investigating the Impact of Delivery Routes for Exon Skipping Therapies in the CNS of DMD Mouse Models. Cells. https://www.mdpi.com/2073-4409/12/6/908 #Morpholino

Genetic mapping and molecular mechanism behind color variation in the Asian vine snake. https://genomebiology.biomedcentral.com/articles/10.1186/s13059-023-02887-z #Morpholino

Diflovidazin damages the hematopoietic stem cells to zebrafish embryos via the TLR4/ NF-κB/ p53 pathway https://www.sciencedirect.com/science/article/abs/pii/S1050464823001584 #Morpholino

Nucleolar URB1 ensures 3′ ETS rRNA removal to prevent exosome surveillance. https://www.nature.com/articles/s41586-023-05767-5 #Morpholino

Mucociliary Wnt signaling promotes cilia biogenesis and beating. https://www.nature.com/articles/s41467-023-36743-2 #Morpholino

Alcam-a and Pdgfr-α are essential for the development of sclerotome-derived stromal cells that support hematopoiesis. https://www.nature.com/articles/s41467-023-36612-y #Morpholino

Migration and establishment of progenitor pool of melanocytes is governed by SEMA3E-PLXND1 signaling. https://www.biorxiv.org/content/10.1101/2023.02.28.530560v1.full #Morpholino

Temporal single-cell transcriptomic analysis of the sox1a:eGFP transgenic line identified the lateral floor plate progenitor cells as the origin of intraspinal serotonergic neurons. https://www.biorxiv.org/content/10.1101/2023.03.01.530240v1 #Morpholino

Functional Differentiation of Cyclins and Cyclin-Dependent Kinases in Giardia lamblia. https://journals.asm.org/doi/10.1128/spectrum.04919-22
#Morpholino

YULINK regulates vascular formation in zebrafish and HUVECs. https://biolres.biomedcentral.com/articles/10.1186/s40659-023-00415-8 #Morpholino

In-Cell Penetration Selection–Mass Spectrometry Produces Noncanonical Peptides for Antisense Delivery. https://pubs.acs.org/doi/10.1021/acschembio.2c00920 #Morpholino

Mob4 is required for neurodevelopment in zebrafish. https://www.micropublication.org/journals/biology/micropub-biology-000762 #Morpholino

AMO (antisense morpholino oligo) disrupts U1 snRNP structure to promote intronic premature cleavage and polyadenylation (PCPA). https://www.biorxiv.org/content/10.1101/2023.02.24.529985v1 #Morpholino