Suppr超能文献

相似文献

1
Lethal immunotoxicity in high-dose systemic AAV therapy.
Mol Ther. 2023 Nov 1;31(11):3123-3126. doi: 10.1016/j.ymthe.2023.10.015. Epub 2023 Oct 10.
2
[How safe is gene therapy? : Second death after Duchenne therapy].
Inn Med (Heidelb). 2024 Jun;65(6):617-623. doi: 10.1007/s00108-024-01711-5. Epub 2024 May 15.
3
Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy.
Mol Ther. 2018 Oct 3;26(10):2337-2356. doi: 10.1016/j.ymthe.2018.07.011. Epub 2018 Jul 17.
4
Perspective on Adeno-Associated Virus Capsid Modification for Duchenne Muscular Dystrophy Gene Therapy.
Hum Gene Ther. 2015 Dec;26(12):786-800. doi: 10.1089/hum.2015.107. Epub 2015 Oct 15.
6
Assessment of the Gene Therapy Immune Response in the Canine Muscular Dystrophy Model.
Methods Mol Biol. 2023;2587:353-375. doi: 10.1007/978-1-0716-2772-3_18.
8
Micro-Dystrophin Gene Therapy Goes Systemic in Duchenne Muscular Dystrophy Patients.
Hum Gene Ther. 2018 Jul;29(7):733-736. doi: 10.1089/hum.2018.012. Epub 2018 Apr 5.
9
Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy.
Nat Med. 2019 Mar;25(3):427-432. doi: 10.1038/s41591-019-0344-3. Epub 2019 Feb 18.
10
Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system.
Sci Adv. 2020 Feb 19;6(8):eaay6812. doi: 10.1126/sciadv.aay6812. eCollection 2020 Feb.

引用本文的文献

1
Fighting for every beat: cardiac therapies in Duchenne muscular dystrophy.
Skelet Muscle. 2025 Sep 9;15(1):25. doi: 10.1186/s13395-025-00394-2.
5
Trends and challenges of AAV-delivered gene editing therapeutics for CNS disorders: Implications for neurodegenerative disease.
Mol Ther Nucleic Acids. 2025 Jul 17;36(3):102635. doi: 10.1016/j.omtn.2025.102635. eCollection 2025 Sep 9.
6
Novel Human Heart-Derived Natural Adeno-Associated Virus Capsid Combines Cardiospecificity With Cardiotropism In Vivo.
Circulation. 2025 Aug 12;152(6):416-419. doi: 10.1161/CIRCULATIONAHA.124.070236. Epub 2025 Aug 11.
9
A Compact Base Editor Rescues AATD-associated Liver and Lung Disease in Mouse Models.
bioRxiv. 2025 May 9:2025.05.07.652636. doi: 10.1101/2025.05.07.652636.

本文引用的文献

1
Patient and Caregiver Outcomes After Onasemnogene Abeparvovec Treatment: Findings from the Cure SMA 2021 Membership Survey.
Adv Ther. 2023 Dec;40(12):5315-5337. doi: 10.1007/s12325-023-02685-w. Epub 2023 Sep 30.
2
An Overview of Nonclinical and Clinical Liver Toxicity Associated With AAV Gene Therapy.
Toxicol Pathol. 2023 Oct;51(7-8):400-404. doi: 10.1177/01926233231201408. Epub 2023 Sep 29.
3
Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy.
N Engl J Med. 2023 Sep 28;389(13):1203-1210. doi: 10.1056/NEJMoa2307798.
4
FDA approves first gene therapy for Duchenne muscular dystrophy, despite internal objections.
Nat Rev Drug Discov. 2023 Aug;22(8):610. doi: 10.1038/d41573-023-00103-y.
5
Dystrophin Immunity after Gene Therapy for Duchenne's Muscular Dystrophy.
N Engl J Med. 2023 Jun 15;388(24):2294-2296. doi: 10.1056/NEJMc2212912.
6
Duchenne Muscular Dystrophy Gene Therapy in 2023: Status, Perspective, and Beyond.
Hum Gene Ther. 2023 May;34(9-10):345-349. doi: 10.1089/hum.2023.29242.ddu.
7
Immune Responses to Muscle-Directed Adeno-Associated Viral Gene Transfer in Clinical Studies.
Hum Gene Ther. 2023 May;34(9-10):365-371. doi: 10.1089/hum.2023.056.
8
9
Progress in Bioengineering of Myotropic Adeno-Associated Viral Gene Therapy Vectors.
Hum Gene Ther. 2023 May;34(9-10):350-364. doi: 10.1089/hum.2023.057.

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验