The 10th Oligonucleotide Therapy Approved: Golodirsen for Duchenne Muscular Dystrophy.
作者信息
Aartsma-Rus Annemieke, Corey David R
机构信息
Department of Human Genetics, Leiden University Medical Center, Leiden, the Netherlands.
Department of Pharmacology, UT Southwestern Medical Center, Dallas, Texas.
出版信息
Nucleic Acid Ther. 2020 Apr;30(2):67-70. doi: 10.1089/nat.2020.0845. Epub 2020 Feb 11.
Abstract
摘要
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Identifying and Avoiding tcDNA-ASO Sequence-Specific Toxicity for the Development of DMD Exon 51 Skipping Therapy.识别并避免用于杜氏肌营养不良症第51外显子跳跃疗法开发的tcDNA-ASO序列特异性毒性。
Mol Ther Nucleic Acids. 2020 Mar 6;19:371-383. doi: 10.1016/j.omtn.2019.11.020. Epub 2019 Nov 27.
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Drugs Today (Barc). 2019 Oct;55(10):627-639. doi: 10.1358/dot.2019.55.10.3045038.
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Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases.基于肽偶联反义寡核苷酸的剪接校正治疗杜氏肌营养不良症和其他神经肌肉疾病。
EBioMedicine. 2019 Jul;45:630-645. doi: 10.1016/j.ebiom.2019.06.036. Epub 2019 Jun 27.
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J Neuromuscul Dis. 2019;6(2):213-225. doi: 10.3233/JND-180351.
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Nat Rev Neurol. 2018 Aug;14(8):454-456. doi: 10.1038/s41582-018-0033-8.
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Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production.依特司培生治疗杜氏肌营养不良症：外显子跳跃和抗肌萎缩蛋白的产生。
Neurology. 2018 Jun 12;90(24):e2146-e2154. doi: 10.1212/WNL.0000000000005680. Epub 2018 May 11.
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Arginine-rich membrane-permeable peptides are seriously toxic.富含精氨酸的膜通透性肽具有严重的毒性。
Pharmacol Res Perspect. 2017 Oct;5(5). doi: 10.1002/prp2.334.
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Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues.《Duchenne 肌营养不良症外显子跳跃疗法的发展：批判性评价及对突出问题的展望》
Nucleic Acid Ther. 2017 Oct;27(5):251-259. doi: 10.1089/nat.2017.0682. Epub 2017 Aug 10.
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