Concepedia

Safe and effective superoxide dismutase 1 silencing using artificial microRNA in macaques

Florie Borel, Gwladys Gernoux, Huaming Sun, Rachel Stock, Meghan Blackwood, Robert H. Brown, Christian Mueller

Science Translational Medicine · 2018 · 77 citations · 36 references

DOI

Concepts

Abstract

Intrathecal delivery of artificial microRNA reduced SOD1 expression in motor neurons in macaques.

References

36

Mutations in Cu/Zn superoxide dismutase gene are associated with familial amyotrophic lateral sclerosis

D.R. Rosen, Teepu Siddique, David T. Patterson et al. · Nature · 1993 · 7K citations

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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

Jerry R. Mendell, Samiah Al-Zaidy, Richard Shell et al. · New England Journal of Medicine · 2017 · 2.3K citations · Full text

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Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Catherine S. Manno, Glenn F. Pierce, Valder R. Arruda et al. · Nature Medicine · 2006 · 2.1K citations

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Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

Amit C. Nathwani, Edward G. D. Tuddenham, Savita Rangarajan et al. · New England Journal of Medicine · 2011 · 1.8K citations · Full text

, , +13

Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways

Dirk Grimm, Konrad L. Streetz, Catherine L. Jopling et al. · Nature · 2006 · 1.7K citations

, , +8