Efficacy and safety of a SOD1-targeting artificial miRNA delivered by AAV9 in mice are impacted by miRNA scaffold selection-Reference-Cited by-同舟云学术

Efficacy and safety of a SOD1-targeting artificial miRNA delivered by AAV9 in mice are impacted by miRNA scaffold selection

Published:2023-12 Issue: Volume:34 Page:102057
ISSN:2162-2531
Container-title:Molecular Therapy - Nucleic Acids
language:en
Short-container-title:Molecular Therapy - Nucleic Acids

Author:

Chen Shukkwan K.,Hawley Zachary C.E.,Zavodszky Maria I.,Hana Sam,Ferretti Daniel,Grubor Branka,Hawes Michael,Xu Shanqin,Hamann Stefan,Marsh Galina,Cullen Patrick,Challa Ravi,Carlile Thomas M.,Zhang Hang,Lee Wan-Hung,Peralta Andrea,Clarner Pete,Wei Cong,Koszka Kathryn,Gao Feng,Lo Shih-Ching

Funder

Biogen

Publisher

Elsevier BV

Subject

Drug Discovery,Molecular Medicine

Reference56 articles.

1. Amyotrophic Lateral Sclerosis: An Update for 2018;Oskarsson;Mayo Clin. Proc.,2018

2. Mutations in Cu/Zn superoxide dismutase gene are associated with familial amyotrophic lateral sclerosis;Rosen;Nature,1993

3. ALS Genetics: Gains, Losses, and Implications for Future Therapies;Kim;Neuron,2020

4. Oxidative stress causes abnormal accumulation of familial amyotrophic lateral sclerosis-related mutant SOD1 in transgenic Caenorhabditis elegans;Oeda;Hum. Mol. Genet.,2001

5. Familial amyotrophic lateral sclerosis;Siddique;J. Neural. Transm. Suppl.,1997

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