参考资料（可上下滑动查看）
[1] Roberts et al, (2023). Therapeutic approaches for Duchenne muscular dystrophy. Nature Reviews Drug Discovery. https://doi.org/10.1038/s41573-023-00775-6
[2] MDA Kicks Off Muscular Dystrophy Awareness Month in September with ‘30 Days of Strength’ Campaign with Hundreds of Events to Advance Research and Care for the Neuromuscular Disease Community. Retrieved September 19, 2024, from https://www.mda.org/press-releases/2024/mda-kicks-off-muscular-dystrophy-awareness-month-in-september-with-30-days-of-strength-campaign-with-hundreds-of-events-to-advance-research-and-care-for-the-neuromuscular-disease-community
[3] RNA Platform Leveraging RNA technology to treat rare disease. Retrieved September 19, 2024, from https://www.sarepta.com/science/rna-platform
[4] Sarepta Therapeutics Corporate Presentation 2024. Retrieved September 19, 2024, from https://investorrelations.sarepta.com/static-files/54793bd6-09b6-4c68-8281-39e586071e88
[5] Roberts TC, Langer R, Wood MJA. Advances in oligonucleotide drug delivery. Nat Rev Drug Discov. 2020 Oct;19(10):673-694. doi: 10.1038/s41573-020-0075-7. Epub 2020 Aug 11. PMID: 32782413; PMCID: PMC7419031.
[6] Avidity Biosciences Announces Positive AOC 1044 Data Demonstrated Significant Increase of 25% in Dystrophin Production and Reduction of Creatine Kinase Levels to Near Normal in People Living with Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping in the Phase 1/2 EXPLORE44™ Trial. Retrieved September 22, 2024 from https://www.prnewswire.com/news-releases/avidity-biosciences-announces-positive-aoc-1044-data-demonstrated-significant-increase-of-25-in-dystrophin-production-and-reduction-of-creatine-kinase-levels-to-near-normal-in-people-living-with-duchenne-muscular-dystrophy-amenab-302218647.html
[7] Sarepta Therapeutics Announces Positive Data from Part B of MOMENTUM, a Phase 2 Study of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51. Retrieved September 22, 2024 from https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-positive-data-part-b-momentum
[8] Advancing the Use of Peptide-Conjugated Oligonucleotides to Target Neuromuscular Disorders: Enhanced Delivery Oligonucleotides for DMD and DM1. Retrieved September 22, 2024 from https://investors.pepgen.com/static-files/ff601ea2-0a29-435f-82b1-c885924cfb94
[9] Watanabe N, Tone Y, Nagata T, Masuda S, Saito T, Motohashi N, Takagaki K, Aoki Y, Takeda S. Exon 44 skipping in Duchenne muscular dystrophy: NS-089/NCNP-02, a dual-targeting antisense oligonucleotide. Mol Ther Nucleic Acids. 2023 Sep 20;34:102034. doi: 10.1016/j.omtn.2023.102034. PMID: 37854955; PMCID: PMC10579524.
[10] Myotonic Dystrophy Type 1 Program. Retrieved September 22, 2024 from https://www.entradatx.com/dm1
[11] Tsoumpra MK, Fukumoto S, Matsumoto T, Takeda S, Wood MJA, Aoki Y. Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases. EBioMedicine. 2019 Jul;45:630-645. doi: 10.1016/j.ebiom.2019.06.036. Epub 2019 Jun 27. PMID: 31257147; PMCID: PMC6642283.
