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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 12282 scientific papers returned from the database with the search filters currently being used below.

Safety and efficacy of viltolarsen in ambulatory and nonambulatory males with Duchenne muscular dystrophy

Authors:
Harper AD, Topaloglu H, Mercuri E, Suslov V, Wu L, Ayanoglu CY, Tansey M, Previtera ML, Crozier RA, Magnus L, Clemens PR
Citation:
Sci Rep. 2024 Oct 8;14(1):23488. doi: 10.1038/s41598-024-70783-y. PMID: 39379403
Epub:
Not Epub
Abstract:
Duchenne muscular dystrophy (DMD) is an X-linked recessive disease characterized by mutations in the dystrophin gene, causing...
Link:
https://www.nature.com/articles/s41598-024-70783-y
Delivery Method:
intravenous (i.v.) infusion
Organism or Cell Type:
human
Citation Extract:
Harper AD, Topaloglu H, Mercuri E, Suslov V, Wu L, Ayanoglu CY, Tansey M, Previtera ML, Crozier RA, Magnus L, Clemens PR. Safety and efficacy of viltolarsen in ambulatory and nonambulatory males with Duchenne muscular dystrophy. Sci Rep. 2024 Oct 8;14(1):23488. doi: 10.1038/s41598-024-70783-y. PMID: 39379403.

PTBP1 mediates Sertoli cell actin cytoskeleton organization by regulating alternative splicing of actin regulators

Authors:
Wang Y, Chembazhi UV, Yee D, Chen S, Ji J, Wang Y, Nguyen KL, Lin P, Ratti A, Hess RA, Qiao H, Ko C, Yang J, Kalsotra A, Mei W
Citation:
Nucleic Acids Res. 2024 Oct 7:gkae862. doi: 10.1093/nar/gkae862. Epub ahead of print. PMID: 39373517
Epub:
Not Epub
Abstract:
Spermatogenesis is a biological process within the testis that produces haploid spermatozoa for the continuity of species....
Link:
https://academic.oup.com/nar/advance-article/doi/10.1093/nar/gkae862/7814703
Delivery Method:
Endo-Porter
Organism or Cell Type:
P22 or P35 mouse testes-derived Sertoli cells
Citation Extract:
Wang Y, Chembazhi UV, Yee D, Chen S, Ji J, Wang Y, Nguyen KL, Lin P, Ratti A, Hess RA, Qiao H, Ko C, Yang J, Kalsotra A, Mei W. PTBP1 mediates Sertoli cell actin cytoskeleton organization by regulating alternative splicing of actin regulators. Nucleic Acids Res. 2024 Oct 7:gkae862. doi: 10.1093/nar/gkae862. Epub ahead of print. PMID: 39373517.

Haploidy-linked cell proliferation defects limit larval growth in zebrafish

Authors:
Yaguchi K, Saito D, Menon T, Matsura A, Hosono M, Mizutani T, Kotani T, Nair S, Uehara R
Citation:
Open Biol. 2024 Oct;14(10):240126. doi: 10.1098/rsob.240126. Epub 2024 Oct 9. PMID: 39378986
Epub:
Not Epub
Abstract:
Haploid larvae in non-mammalian vertebrates are lethal, with characteristic organ growth retardation collectively called '...
Link:
https://royalsocietypublishing.org/doi/10.1098/rsob.240126
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Yaguchi K, Saito D, Menon T, Matsura A, Hosono M, Mizutani T, Kotani T, Nair S, Uehara R. Haploidy-linked cell proliferation defects limit larval growth in zebrafish. Open Biol. 2024 Oct;14(10):240126. doi: 10.1098/rsob.240126. Epub 2024 Oct 9. PMID: 39378986.

NPC1 links cholesterol trafficking to microglial morphology via the gastrosome

Authors:
Zareba J, Cattaneo EF, Villani A, Othman A, Streb S, Peri F
Citation:
Nat Commun. 2024 Oct 5;15(1):8638. doi: 10.1038/s41467-024-52874-6. PMID: 39366931; PMCID: PMC11452621.
Epub:
Not Epub
Abstract:
Microglia play important roles in brain development and homeostasis by removing dying neurons through efferocytosis....
Link:
https://www.nature.com/articles/s41467-024-52874-6
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Zareba J, Cattaneo EF, Villani A, Othman A, Streb S, Peri F . NPC1 links cholesterol trafficking to microglial morphology via the gastrosome. Nat Commun. 2024 Oct 5;15(1):8638. doi: 10.1038/s41467-024-52874-6. PMID: 39366931; PMCID: PMC11452621..

Elp1 function in placode-derived neurons is critical for proper trigeminal ganglion development

Authors:
Hines MA, Taneyhill LA
Citation:
Dev Dyn. 2024 Oct 9. doi: 10.1002/dvdy.749. Epub ahead of print. PMID: 39381860
Epub:
Not Epub
Abstract:
Background The trigeminal nerve is the largest cranial nerve and functions in somatosensation. Cell bodies of this nerve are...
Link:
https://anatomypubs.onlinelibrary.wiley.com/doi/10.1002/dvdy.749
Delivery Method:
electroporation
Organism or Cell Type:
chick trigeminal placode cells
Citation Extract:
Hines MA, Taneyhill LA. Elp1 function in placode-derived neurons is critical for proper trigeminal ganglion development. Dev Dyn. 2024 Oct 9. doi: 10.1002/dvdy.749. Epub ahead of print. PMID: 39381860.

An Anti-Invasive Role for Mdmx through the RhoA GTPase under the Control of the NEDD8 Pathway

Authors:
Bou Malhab LJ, Schmidt S, Fagotto-Kaufmann C, Pion E, Gadea G, Roux P, Fagotto F, Debant A, Xirodimas DP
Citation:
Cells. 2024; 13(19):1625. https://doi.org/10.3390/cells13191625
Epub:
Not Epub
Abstract:
Mdmx (Mdm4) is established as an oncogene mainly through repression of the p53 tumour suppressor. On the other hand, anti-...
Link:
https://www.mdpi.com/2073-4409/13/19/1625
Delivery Method:
microinjection
Organism or Cell Type:
Xenopus laevis
Citation Extract:
Bou Malhab LJ, Schmidt S, Fagotto-Kaufmann C, Pion E, Gadea G, Roux P, Fagotto F, Debant A, Xirodimas DP. An Anti-Invasive Role for Mdmx through the RhoA GTPase under the Control of the NEDD8 Pathway. Cells. 2024; 13(19):1625. https://doi.org/10.3390/cells13191625.

Self-Assembled Antibody-Oligonucleotide Conjugates for Targeted Delivery of Complementary Antisense Oligonucleotides

Authors:
Pan L, Zhou L, Bi J, Chang S, Bai Z, Yu J, Wang R, Li Z, Zhang X, Chou JJ
Citation:
Angew Chem Int Ed Engl. 2024 Sep 26:e202415272. doi: 10.1002/anie.202415272. Epub ahead of print. PMID: 39325927
Epub:
Not Epub
Abstract:
Antibody-oligonucleotide conjugate (AOC) affords preferential cell targeting and enhanced cellular uptake of antisense...
Link:
https://onlinelibrary.wiley.com/doi/10.1002/anie.202415272
Delivery Method:
antibody-linked; mice: intravenous (i.v.)
Organism or Cell Type:
cell culture: C2C12; mice: TfR1-humanized C57BL/6
Citation Extract:
Pan L, Zhou L, Bi J, Chang S, Bai Z, Yu J, Wang R, Li Z, Zhang X, Chou JJ. Self-Assembled Antibody-Oligonucleotide Conjugates for Targeted Delivery of Complementary Antisense Oligonucleotides. Angew Chem Int Ed Engl. 2024 Sep 26:e202415272. doi: 10.1002/anie.202415272. Epub ahead of print. PMID: 39325927.

Deciphering the impact of PROM1 alternative splicing on human photoreceptor development and maturation

Authors:
Moya-Molina M, Dorgau B, Flood E, Letteboer SJF, Lorentzen E, Coxhead J, Smith G, Roepman R, Nagaraja Grellscheid S, Armstrong L, Lako M
Citation:
Cell Death Dis. 2024 Oct 1;15(10):721. doi: 10.1038/s41419-024-07105-7. PMID: 39353897
Epub:
Not Epub
Abstract:
Alternative splicing (AS) is a crucial mechanism contributing to proteomic diversity, which is highly regulated in tissue- and...
Link:
https://www.nature.com/articles/s41419-024-07105-7
Delivery Method:
Endo-Porter PEG
Organism or Cell Type:
retinal organoids derived from hiPSCs
Citation Extract:
Moya-Molina M, Dorgau B, Flood E, Letteboer SJF, Lorentzen E, Coxhead J, Smith G, Roepman R, Nagaraja Grellscheid S, Armstrong L, Lako M. Deciphering the impact of PROM1 alternative splicing on human photoreceptor development and maturation. Cell Death Dis. 2024 Oct 1;15(10):721. doi: 10.1038/s41419-024-07105-7. PMID: 39353897.

Prox1a promotes liver growth and differentiation by repressing cdx1b expression and intestinal fate transition in zebrafish

Authors:
Hu Y, Luo Z, Wang M, Wu Z, Liu Y, Cheng Z, Sun Y, Xiong JW, Tong X, Zhu Z, Zhang B
Citation:
J Genet Genomics. 2025 Jan;52(1):66-77. doi: 10.1016/j.jgg.2024.09.010. Epub 2024 Sep 27. PMID: 39343095
Epub:
Not Epub
Abstract:
The liver is a key endoderm-derived multifunctional organ within the digestive system. Prospero homeobox 1 (Prox1) is an...
Link:
https://www.sciencedirect.com/science/article/abs/pii/S1673852724002480
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Hu Y, Luo Z, Wang M, Wu Z, Liu Y, Cheng Z, Sun Y, Xiong JW, Tong X, Zhu Z, Zhang B. Prox1a promotes liver growth and differentiation by repressing cdx1b expression and intestinal fate transition in zebrafish. J Genet Genomics. 2025 Jan;52(1):66-77. doi: 10.1016/j.jgg.2024.09.010. Epub 2024 Sep 27. PMID: 39343095.

Deoxyhypusine synthase deficiency syndrome zebrafish model: aberrant morphology, epileptiform activity, and reduced arborization of inhibitory interneurons

Authors:
Shojaeinia E, Mastracci TL, Soliman R, Devinsky O, Esguerra CV, Crawford AD
Citation:
Mol Brain. 2024 Sep 27;17(1):68. doi: 10.1186/s13041-024-01139-w. PMID: 39334388
Epub:
Not Epub
Abstract:
DHPS deficiency syndrome is an ultra-rare neurodevelopmental disorder (NDD) which results from biallelic mutations in the gene...
Link:
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11429087/
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Shojaeinia E, Mastracci TL, Soliman R, Devinsky O, Esguerra CV, Crawford AD. Deoxyhypusine synthase deficiency syndrome zebrafish model: aberrant morphology, epileptiform activity, and reduced arborization of inhibitory interneurons. Mol Brain. 2024 Sep 27;17(1):68. doi: 10.1186/s13041-024-01139-w. PMID: 39334388.

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