Gentherapien bei monogenen Erkrankungen
Author:
Publisher
Springer Science and Business Media LLC
Link
https://link.springer.com/content/pdf/10.1007/s15014-024-5359-7.pdf
Reference9 articles.
1. Ziegler A, Walter MC, Schoser BE. Molekulare Therapien: Gegenwart und Zukunft bei neuromuskulären Erkrankungen [Molecular therapies: present and future in neuromuscular diseases]. Nervenarzt. 2023 Jun;94(6):473-87
2. Au HKE, Isalan M, Mielcarek M. Gene Therapy Advances: A Meta-Analysis of AAV Usage in Clinical Settings. Front Med. (Lausanne) 2021;8:809118
3. Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, Goodspeed K, Gray SJ, Kay CN et al. Current Clinical Applications of In Vivo Gene Therapy with AAVs. Mol Ther. 2021;29(2):464-88
4. Gentherapeutika - Paul-Ehrlich-Institut (www.pei.de/DE/arzneimittel/atmp/gentherapeutika/gentherapeutika-node.html, Zugriff 11.12.2023)
5. Strauss KA, Farrar MA, Muntoni F, Saito K, Mendell JR, Servais L et al. Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial. Nat Med. 2022;28(7):1381-9
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