jonmoulton @jonmoulton@qoto.org

Deficiency of the minor spliceosome component U4atac snRNA secondarily results in ciliary defects in human and zebrafish. https://www.pnas.org/doi/10.1073/pnas.2102569120 #Morpholino

Next steps for the optimization of exon therapy for Duchenne muscular dystrophy
Galina Filonova & Annemieke Aartsma-Rus
EXPERT OPINION ON BIOLOGICAL THERAPY. 2023;23(2): 133–43. doi:10.1080/14712598.2023.216907
https://www.tandfonline.com/doi/epdf/10.1080/14712598.2023.2169070

Alternative splicing of its 5’ untranslated region controls CD20 mRNA translation and enables resistance to CD20-directed immunotherapies. https://www.biorxiv.org/content/10.1101/2023.02.19.529123v1 #Morpholino

Estrogen Signaling Influences Nephron Segmentation of the Zebrafish Embryonic Kidney. https://www.mdpi.com/2073-4409/12/4/666 #Morpholino

USP47 deubiquitylates Groucho/TLE to promote Wnt–β-catenin signaling. https://www.science.org/doi/10.1126/scisignal.abn8372 #Morpholino

FGF8 rescues motor deficits in zebrafish model of limb-girdle muscular dystrophy R18. https://www.sciencedirect.com/science/article/abs/pii/S0006291X23002206 #Morpholino

TEFM variants impair mitochondrial transcription causing childhood-onset neurological disease. https://www.nature.com/articles/s41467-023-36277-7 #Morpholino

Neurogenin 2 and Neuronal Differentiation 1 Control Proper Development of the Chick Trigeminal Ganglion and Its Nerve Branches. https://www.mdpi.com/2221-3759/11/1/8 #Morpholino

Synthesis of Chlorophosphoramidate Monomer Morpholinos and PMOs. https://currentprotocols.onlinelibrary.wiley.com/doi/10.1002/cpz1.686 #Morpholino

p16 INK4A drives non-alcoholic fatty liver disease phenotypes in high fat diet fed mice via biliary e2f1/foxo1/igf-1 signaling. https://journals.lww.com/hep/Abstract/9900/p16_INK4A_drives_non_alcoholic_fatty_liver_disease.303.aspx
#Morpholino

I'll halt #Morpholino toots for a week to go present at #PharmaR&D, 21 Feb - 23 Feb, Las Vegas, NV USA
Altering Eukaryotic Splicing with Morpholino Oligos: Drugging for Isoforms
08:40AM - 09:00AM Feb 22
https://unitedscientificgroup.com/conferences/pharma/

Genetic analysis identifies the SLC4A3 anion exchanger as a major gene for short QT syndrome. https://www.heartrhythmjournal.com/article/S1547-5271(23)00148-0/fulltext #Morpholino

Single-cell atavism reveals an ancient mechanism of cell type diversification in a sea anemone. https://www.nature.com/articles/s41467-023-36615-9 #Morpholino

CCN6 influences transcription and controls mitochondrial mass and muscle organization. https://faseb.onlinelibrary.wiley.com/doi/10.1096/fj.202201533R #Morpholino

EVA1A (Eva-1 Homolog A) Promotes Endothelial Apoptosis and Inflammatory Activation Under Disturbed Flow Via Regulation of Autophagy. https://www.ahajournals.org/doi/10.1161/ATVBAHA.122.318110 #Morpholino

Therapeutic Phosphorodiamidate Morpholino Oligonucleotides, an RNA mimic: Physical Properties, Solution Structures, and Folding Thermodynamics. https://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(23)00027-6 #Morpholino

The genetic basis and potential molecular mechanism of yellow-albino northern snakehead (Channa argus). https://royalsocietypublishing.org/doi/10.1098/rsob.220235 #Morpholino

Milk fat globule epidermal growth factor 8a regulates neurogenesis in telencephalon and affects larval behavior in zebrafish. https://www.liebertpub.com/doi/10.1089/scd.2022.0247 #Morpholino

Clcf1/Crlf1a-mediated signaling is neuroprotective and required for Müller glia proliferation in the light-damaged zebrafish retina. https://www.frontiersin.org/articles/10.3389/fcell.2023.1142586/full #Morpholino

(article) Proof-of-Concept Study Shows Promise of Complementary Gene Therapy Approach in Duchenne Muscular Dystrophy https://pediatricsnationwide.org/2023/02/10/proof-of-concept-study-shows-promise-of-complementary-gene-therapy-approach-in-duchenne-muscular-dystrophy/ #Morpholino