Translating the ALS Genetic Revolution into Therapies: A Review
Author:
Publisher
Springer Science and Business Media LLC
Subject
Neurology (clinical)
Link
https://link.springer.com/content/pdf/10.1007/s11940-024-00781-y.pdf
Reference54 articles.
1. • Akçimen F, Lopez ER, Landers JE, Nath A, Chiò A, Chia R, et al. Amyotrophic lateral sclerosis: translating genetic discoveries into therapies. Nat Rev Genet. 2023;24(9):642–58 This review highlights recent discoveries in ALS, including new mutations, gene variability and gene-environment interactions, and provides a summary of the current status of ALS therapies developed over the past 30 years.
2. • Amado DA, Davidson BL. Gene therapy for ALS: A review. Mol Ther. 2021;29(12):3345–58 This review examines the current landscape of ALS directed gene therapy focusing on various methodologies such as ASO, RNA interference, CRISPR, AAV-mediated trophic support, and antibody-based techniques, and their applications in both genetic and sporadic cases.
3. Bensimon G, Lacomblez L, Meininger V. A controlled trial of riluzole in amyotrophic lateral sclerosis. N Engl J Med. 1994;330(9):585–91.
4. • Paganoni S, Macklin EA, Hendrix S, Berry JD, Elliott MA, Maiser S, et al. Trial of sodium phenylbutyrate–taurursodiol for amyotrophic lateral sclerosis. N Engl J Med. 2020;383(10):919–30 This article provides evidence that sodium phenylbutyrate–taurursodiol results in slower functional decline than placebo over a period of 24 weeks as measured by the ALSFRS-R score.
5. Rothstein JD. Edaravone: a new drug approved for ALS. Cell. 2017;171(4):725.
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