Morpholino-Mediated Exon Skipping Targeting Human ACVR1/ALK2 for Fibrodysplasia Ossificans Progressiva
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Springer New York
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http://link.springer.com/content/pdf/10.1007/978-1-4939-8651-4_32
Reference14 articles.
1. Pignolo RJ, Shore EM, Kaplan FS (2011) Fibrodysplasia ossificans progressiva: clinical and genetic aspects. Orphanet J Rare Dis 6:80. https://doi.org/10.1186/1750-1172-6-80
2. Chakkalakal SA, Uchibe K, Convente MR et al (2016) Palovarotene inhibits heterotopic ossification and maintains limb mobility and growth in mice with the human ACVR1(R206H) fibrodysplasia ossificans progressiva (FOP) mutation. J Bone Miner Res 31(9):1666–1675. https://doi.org/10.1002/jbmr.2820
3. Hino K, Horigome K, Nishio M et al (2017) Activin-a enhances mTOR signaling to promote aberrant chondrogenesis in fibrodysplasia ossificans progressiva. J Clin Invest 127(9):3339–3352. https://doi.org/10.1172/JCI93521
4. Shore EM, Xu M, Feldman GJ et al (2006) A recurrent mutation in the BMP type I receptor ACVR1 causes inherited and sporadic fibrodysplasia ossificans progressiva. Nat Genet 38(5):525–527. https://doi.org/10.1038/ng1783
5. Kaplan FS, Xu M, Seemann P et al (2009) Classic and atypical fibrodysplasia ossificans progressiva (FOP) phenotypes are caused by mutations in the bone morphogenetic protein (BMP) type I receptor ACVR1. Hum Mutat 30(3):379–390. https://doi.org/10.1002/humu.20868
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1. Ensemble-Learning and Feature Selection Techniques for Enhanced Antisense Oligonucleotide Efficacy Prediction in Exon Skipping;Pharmaceutics;2023-06-24
2. Druggable targets, clinical trial design and proposed pharmacological management in fibrodysplasia ossificans progressiva;Expert Opinion on Orphan Drugs;2020-04-02
3. Targeting heterotopic ossification by inhibiting activin receptor‑like kinase 2 function (Review);Molecular Medicine Reports;2019-08-06
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