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The Medicinal Chemistry of Artificial Nucleic Acids and Therapeutic Oligonucleotides

• Authors : Miklós Bege; Anikó Borbás

Subjects: oligonucleotides; phosphorothioate; phosphorodiamidate morpholino oligomers (PMOs)

• Source: Pharmaceuticals, Vol 15, Iss 909, p 909 (2022)

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Design and Catalyzed Activation of Mycophenolic Acid Prodrugs

• Authors : Michael A. Plunk (8267466); Jeremy M. Quintana (10652388); Carly M. Darden (10652391)

Subjects: Medicine; Pharmacology; Biotechnology

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Molecular Diagnosis and Novel Therapies for Neuromuscular Diseases

• Authors : Rika Maruyama; Toshifumi Yokota

Subjects: Duchenne/Becker muscular dystrophy (DMD/BMD); amyotrophic lateral sclerosis (ALS); viltolarsen

• Source: Journal of Personalized Medicine, Vol 10, Iss 129, p 129 (2020)

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Pharmacokinetic/Pharmacodynamic Modeling of a Cell-Penetrating Peptide Phosphorodiamidate Morpholino Oligomer in mdx Mice

• Authors : Samantha Foley; Shawn Harriman; Jianbo Zhang

Subjects: Duchenne muscular dystrophy; Male; Muscle tissue

• Source: Pharmaceutical Research

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Fully automated fast-flow synthesis of antisense phosphorodiamidate morpholino oligomers

• Authors : Genwei Zhang; Chengxi Li; Hong Zong ...

Subjects: 10120 Department of Chemistry; 0301 basic medicine; Time Factors

• Source: Nature Communications, Vol 12, Iss 1, Pp 1-8 (2021)Nature Communications

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Multiple Exon Skipping in the Duchenne Muscular Dystrophy Hot Spots: Prospects and Challenges

• Authors : Yusuke Echigoya; Kenji Rowel Q. Lim; Akinori Nakamura

Subjects: dystrophinopathy; Duchenne muscular dystrophy (DMD); Becker muscular dystrophy (BMD)

• Source: Journal of Personalized Medicine, Vol 8, Iss 4, p 41 (2018)

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Red‐ and Far‐Red‐Emitting Zinc Probes with Minimal Phototoxicity for Multiplexed Recording of Orchestrated Insulin Secretion

• Authors : Junwei Zhang; Jingfu Sun; Chao Tang

Subjects: Morpholino; medicine.medical_treatment; chemistry.chemical_element

• Source: Angewandte Chemie. 133:26050-26059

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Cortisol and glucocorticoid receptor 2 regulate acid secretion in medaka (Oryzias latipes) larvae

• Authors : Chia-Hao Lin; Hsin-Ju Chuang; Pung-Pung Hwang

Subjects: medicine.medical_specialty; animal structures; biology

• Source: Journal of Comparative Physiology B. 191:855-864

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Morpholino Oligomer-Induced Dystrophin Isoforms to Map the Functional Domains in the Dystrophin Protein

• Authors : Dunhui Li; Abbie M. Adams; Steve D. Wilton

Subjects: Duchenne muscular dystrophy; musculoskeletal diseases; 0301 basic medicine

• Source: Molecular Therapy. Nucleic AcidsMolecular Therapy: Nucleic Acids, Vol 22, Iss, Pp 263-272 (2020)

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Sox9a, not sox9b is required for normal cartilage development in zebrafish

• Authors : Yan He; Qiaohong Lin; Jian-Fang Gui

Subjects: animal structures; Morpholino; Mutant

• Source: Aquaculture and Fisheries, Vol 6, Iss 3, Pp 254-259 (2021)

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Casimersen: First Approval

• Authors : Matt Shirley

Subjects: musculoskeletal diseases; Oncology; congenital, hereditary, and neonatal diseases and abnormalities

• Source: Drugs. 81:875-879

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Hexose Potentiates Peptide-Conjugated Morpholino Oligomer Efficacy in Cardiac Muscles of Dystrophic Mice in an Age-Dependent Manner

• Authors : Xianjun Gao; Caorui Lin; Hong M. Moulton

Subjects: 0301 basic medicine; musculoskeletal diseases; Duchenne muscular dystrophy

• Source: Molecular Therapy. Nucleic AcidsMolecular Therapy: Nucleic Acids, Vol 18, Iss, Pp 341-350 (2019)

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The altered expression of neurofilament in mouse models and patients with spinal muscular atrophy

• Authors : Linda Greensmith; Haiyan Zhou; Andrea Malaspina

Subjects: Male; 0301 basic medicine; Genetically modified mouse

• Source: Annals of Clinical and Translational Neurology, Vol 8, Iss 4, Pp 866-876 (2021)Annals of Clinical and Translational Neurology

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Does blood flow limit acute hypoxia performance in larval zebrafish (Danio rerio)?

• Authors : M C Hughes; Steve F. Perry

Subjects: 030110 physiology; 0106 biological sciences; 0301 basic medicine

• Source: Journal of Comparative Physiology B. 191:469-478

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Insulin-Like Growth Factor-1 Enhances Motoneuron Survival and Inhibits Neuroinflammation After Spinal Cord Transection in Zebrafish

• Authors : Shu-Bing Huang; Chun Cui; Yun Shi

Subjects: 0301 basic medicine; animal structures; Morpholino

• Source: Cellular and Molecular Neurobiology. 42:1373-1384

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Mir24-2-5p suppresses the osteogenic differentiation with Gnai3 inhibition presenting a direct target via inactivating JNK-p38 MAPK signaling axis

• Authors : Jieli Ni; Yang Zhang; Shuyu Guo

Subjects: Morpholino; MAP Kinase Kinase 4; Craniofacial deformity

• Source: International Journal of Biological Sciences

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Mutated

• Authors : Muhammad Umair; Muhammad Farooq Khan; Mohammed Aldrees

Subjects: zebrafish morpholino; Microcephaly; Morpholino

• Source: Frontiers in Cell and Developmental BiologyFrontiers in Cell and Developmental Biology, Vol 9 (2021)

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Immortalized Muscle Cell Model to Test the Exon Skipping Efficacy for Duchenne Muscular Dystrophy

• Authors : Quynh Nguyen; Toshifumi Yokota

Subjects: Duchenne/Becker muscular dystrophy (DMD/BMD); antisense oligonucleotide-mediated exon skipping therapy; hDMD mice

• Source: Journal of Personalized Medicine, Vol 7, Iss 4, p 13 (2017)

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LIM Homeobox 9 knockdown by morpholino does not affect zebrafish retinal development

• Authors : Lin Gan; Kangkang Ge; Minxia Lu

Subjects: retina; lhx9; animal structures

• Source: Biology Open, Vol 10, Iss 3 (2021)Biology Openarticle-version (VoR) Version of Record

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The Atypical Kinase RIOK3 Limits RVFV Propagation and Is Regulated by Alternative Splicing

• Authors : Katherine E. Havranek; Jean-Marc Lanchy; Luke Adam White

Subjects: 0301 basic medicine; Rift Valley Fever; Morpholino

• Source: VirusesVolume 13Issue 3Viruses, Vol 13, Iss 367, p 367 (2021)

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Scavenger Receptor Class A1 Mediates Uptake of Morpholino Antisense Oligonucleotide into Dystrophic Skeletal Muscle

• Authors : Shouta Miyatake; Yuko Hara; Fazel Shabanpoor

Subjects: Duchenne muscular dystrophy; 0301 basic medicine; Morpholino

• Source: Molecular Therapy: Nucleic Acids, Vol 14, Iss, Pp 520-535 (2019)Molecular Therapy. Nucleic Acids

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Identification of Novel Antisense-Mediated Exon Skipping Targets in DYSF for Therapeutic Treatment of Dysferlinopathy

• Authors : Hidetoshi Sakurai; Rika Maruyama; Toshifumi Yokota

Subjects: 0301 basic medicine; Dysferlinopathy; Morpholino

• Source: Molecular Therapy: Nucleic Acids, Vol 13, Iss, Pp 596-604 (2018)Molecular Therapy. Nucleic Acids

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NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy

• Authors : Naoki Watanabe; Youhei Satou; Takashi Saito

Subjects: Duchenne muscular dystrophy; musculoskeletal diseases; 0301 basic medicine

• Source: Molecular Therapy: Nucleic Acids, Vol 13, Iss, Pp 442-449 (2018)Molecular Therapy. Nucleic Acids

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Shorter Phosphorodiamidate Morpholino Splice-Switching Oligonucleotides May Increase Exon-Skipping Efficacy in DMD

• Authors : Ugur Akpulat; Adriana Contreras; James S. Novak

Subjects: 0301 basic medicine; musculoskeletal diseases; congenital, hereditary, and neonatal diseases and abnormalities

• Source: Molecular Therapy. Nucleic AcidsMolecular Therapy: Nucleic Acids, Vol 13, Iss, Pp 534-542 (2018)

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A k-mer based transcriptomics approach for antisense drug discovery targeting the Ewing’s family of tumors

• Authors : James E. Summerton; Craig B. Marcus; Shawn T. O’Neil

Subjects: 0301 basic medicine; Morpholino; Drug discovery

• Source: Oncotarget

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Disabled-2: a positive regulator of the early differentiation of myoblasts

• Authors : Juliana Tsz Yan Lee; Samuel C. Mok; Na Shang

Subjects: Histology; Morpholino; Xenopus

• Source: Cell and Tissue Research

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Viltolarsen: First Approval

• Authors : Sohita Dhillon

Subjects: musculoskeletal diseases; congenital, hereditary, and neonatal diseases and abnormalities; Morpholino

• Source: Drugs. 80:1027-1031

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CAMSAP3 facilitates basal body polarity and the formation of the central pair of microtubules in motile cilia

• Authors : Satoe Takahashi; Eva J. Brotslaw; Mary Ann Cheatham

Subjects: Axoneme; Morpholino; Xenopus

• Source: Proceedings of the National Academy of Sciences of the United States of America

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Cryptic exon activation causes dystrophinopathy in two Chinese families

• Authors : Ming Jin; Jin-Jing Li; Ning Wang

Subjects: Male; Morpholino; RNA Splicing

• Source: Eur J Hum Genet

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METTL3-mediated m6A is required for murine oocyte maturation and maternal-to-zygotic transition

• Authors : Mingrui Li; Yumeng Cao; Qiqi Cao

Subjects: 0301 basic medicine; Messenger RNA; Small interfering RNA

• Source: Cell Cycle. 19:391-404

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Exons 45–55 Skipping Using Mutation-Tailored Cocktails of Antisense Morpholinos in the DMD Gene

• Authors : Nhu Trieu; Yusuke Echigoya; William Duddy ...

Subjects: musculoskeletal diseases; congenital, hereditary, and neonatal diseases and abnormalities; [SDV.BIO]Life Sciences [q-bio]/Biotechnology

• Source: Mol TherMolecular TherapyMolecular Therapy, Cell Press, 2019, 27 (11), pp.2005-2017. ⟨10.1016/j.ymthe.2019.07.012⟩

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Inhibition ofmmp13aduring zebrafish fin regeneration disrupts fin growth, osteoblasts differentiation, and Laminin organization

• Authors : Li Li; Jing Zhang; Marie-Andrée Akimenko

Subjects: 0301 basic medicine; Morpholino; Fin regeneration

• Source: Developmental Dynamics. 249:187-198

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Generation of a Triadin KnockOut Syndrome Zebrafish Model

• Authors : Marco Spreafico; Chiara Di Resta; Anna Santoni ...

Subjects: Morpholino; Muscle Fibers, Skeletal; Gene Expression

• Source: International Journal of Molecular SciencesVolume 22Issue 18International Journal of Molecular Sciences, Vol 22, Iss 9720, p 9720 (2021)

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Grhl3 promotes retention of epidermal cells under endocytic stress to maintain epidermal architecture in zebrafish

• Authors : Nazma Anjum; Mandar Phatak; Lee B. Miles

Subjects: Cancer Research; Physiology; Cell

• Source: PLoS Genetics, Vol 17, Iss 9, p e1009823 (2021)PLoS Genetics

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Vivo-Morpholino-Based Antiviral for SARS-CoV-2: Implications for Novel Therapies in the Treatment of Acute COVID-19 Disease

• Authors : James E. K. Hildreth; Jon D. Moulton; Donald J. Alcendor

Subjects: Systemic disease; Morpholino; MRCV-19

• Source: BiomedicinesVolume 9Issue 8Biomedicines, Vol 9, Iss 1018, p 1018 (2021)

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Pharmacology and toxicology of eteplirsen and SRP-5051 for DMD exon 51 skipping: an update

• Authors : Omar Sheikh; Toshifumi Yokota

Subjects: Male; Morpholino; Health, Toxicology and Mutagenesis

• Source: Archives of toxicology. 96(1)

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Targeting of SET/I2PP2A oncoprotein inhibits Gli1 transcription revealing a new modulator of Hedgehog signaling

• Authors : Simoni Besta; Zoe Karetsou; Iliana Serifi

Subjects: 0301 basic medicine; Cell biology; Embryo, Nonmammalian

• Source: Scientific ReportsScientific Reports, Vol 11, Iss 1, Pp 1-13 (2021)

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Crosslinking of CD38 Receptors Triggers Apoptosis of Malignant B Cells

• Authors : Jiawei Wang; Douglas W. Sborov; M Tommy Gambles

Subjects: Pharmaceutical Science; Organic chemistry; Apoptosis

• Source: Molecules, Vol 26, Iss 4658, p 4658 (2021)MoleculesVolume 26Issue 15

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Evaluation of Morpholino Antisense Oligos’ Role on BCR-ABL Gene Silencing in the K562 Cell Line

• Authors : Bahman Delalat; Soraya Rasi Ghaemi; Saeid Abroun

Subjects: Chronic Myeloid Leukemia; Morpholino Antisense; K562

• Source: Cell Journal, Vol 12, Iss 2, Pp 241-248 (2010)

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Zebrafish Chromosome 14 Gene Differential Expression in the fmr1hu2787 Model of Fragile X Syndrome

• Authors : Karissa Barthelson; Lachlan Baer; Yang Dong

Subjects: Genetics; Mutation; congenital, hereditary, and neonatal diseases and abnormalities

• Source: Frontiers in Genetics, Vol 12 (2021)

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A Dystrophin Exon-52 Deleted Miniature Pig Model of Duchenne Muscular Dystrophy and Evaluation of Exon Skipping

• Authors : Terence A. Partridge; Eric P. Hoffman; Frank Rohret

Subjects: antisense oligonucleotide; musculoskeletal diseases; congenital, hereditary, and neonatal diseases and abnormalities

• Source: International Journal of Molecular Sciences, Vol 22, Iss 13065, p 13065 (2021)International Journal of Molecular Sciences; Volume 22; Issue 23; Pages: 13065

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Morpholino Target Molecular Properties Affect the Swelling Process of Oligomorpholino-Functionalized Responsive Hydrogels

• Authors : Bjørn T. Stokke; Eleonóra Parelius Jonášová

Subjects: Polymers and Plastics; Morpholino; Oligonucleotide

• Source: Polymers, Vol 12, Iss 2, p 268 (2020)PolymersVolume 12Issue 2

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In Vitro Validation of Phosphorodiamidate Morpholino Oligomers

• Authors : Steve D. Wilton; Kristin A. West; Sue Fletcher

Subjects: antisense oligonucleotide; Morpholino; Cell

• Source: Molecules, Vol 24, Iss 16, p 2922 (2019)MoleculesVolume 24Issue 16

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NOX5 is expressed aberrantly but not a critical pathogenetic gene in Hirschsprung disease

• Authors : Jun Xiao; Jing Wang; Jiexiong Feng

Subjects: 0301 basic medicine; Nervous system; Hirschsprung disease

• Source: BMC PediatricsBMC Pediatrics, Vol 21, Iss 1, Pp 1-10 (2021)

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Genetic Manipulation on Zebrafish duox Recapitulate the Clinical Manifestations of Congenital Hypothyroidism

• Authors : Ping-Hui Tu; Mei Dong; Rui-Meng Yang

Subjects: 0301 basic medicine; Male; endocrine system

• Source: Endocrinology. 162(8)

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Antisense oligonucleotide development for the selective modulation of CYP3A5 in renal disease

• Authors : Lu Wang; Jonathan Himmelfarb; Patrick L. Iversen

Subjects: Morpholino; Molecular biology; Science

• Source: Scientific ReportsScientific Reports, Vol 11, Iss 1, Pp 1-20 (2021)

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son is necessary for proper vertebrate blood development

• Authors : Illiana Cajias; Jung-Hyun Kim; David L. Stachura

Subjects: 0301 basic medicine; Embryology; Embryo, Nonmammalian

• Source: PLoS ONEPLoS ONE, Vol 16, Iss 2, p e0247489 (2021)

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LIM Homeobox 4 (lhx4) regulates retinal neural differentiation and visual function in zebrafish

• Authors : Rui Guo; Donglai Sheng; Kangkang Ge

Subjects: Retinal Bipolar Cells; Rhodopsin; animal structures

• Source: Scientific ReportsScientific Reports, Vol 11, Iss 1, Pp 1-10 (2021)

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A regulatory sub-circuit downstream of Wnt signaling controls developmental transitions in neural crest formation

• Authors : Marcos Simoes-Costa; Ana Paula Azambuja

Subjects: Cancer Research; Gene regulatory network; Oligonucleotides

• Source: PLoS Genetics, Vol 17, Iss 1, p e1009296 (2021)PLoS Genetics

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Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy

• Authors : Joshua Lee; James S. Novak; Rika Maruyama ...

Subjects: Male; 0301 basic medicine; Reading Frames

• Source: Molecular TherapyMolecular Therapy, 2017, 25 (11), pp.2561-2572. ⟨10.1016/j.ymthe.2017.07.014⟩

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