The 2024 Nobel Prize: Impact of the Discovery of miRNA on the Field of Gene Therapy

Abstract

Get full access to this article

View all access options for this article.

References

Lee

, Feinbaum

, Ambros

. The C. elegans heterochronic gene lin-4 encodes small RNAs with antisense complementarity to lin-14. Cell, 1993; 75(5):843–854; doi: 10.1016/0092-8674(93)90529-Y

Wightman

, Ha

, Ruvkun

. Posttranscriptional regulation of the heterochronic gene lin-14 by lin-4 mediates temporal pattern formation in C. elegans . Cell, 1993; 75(5):855–862; doi: 10.1016/0092-8674(93)90530-4

Mueller

, Berry

, McKenna-Yasek

, et al. SOD1 suppression with adeno-associated virus and MicroRNA in familial ALS. N Engl J Med, 2020; 383(2):151–158; doi: 10.1056/NEJMoa2005056

uniQure announces dosing of first patient in phase I/II Clinical Trial of AMT-162 for the treatment of SOD1-ALS. Available from: https://www.globenewswire.com/news-release/2024/10/15/2963063/0/en/uniQure-Announces-Dosing-of-First-Patient-in-Phase-I-II-Clinical-Trial-of-AMT-162-for-the-Treatment-of-SOD1-ALS.html

Xie

, Ameres

, Friedline

, et al. Long-term, efficient inhibition of microRNA function in mice using rAAV vectors. Nat Methods, 2012; 9(4):403–409; doi: 10.1038/nmeth.1903

Stelma

, van der Ree

, Sinnige

, et al. Immune phenotype and function of natural killer and T cells in chronic hepatitis C patients who received a single dose of anti-MicroRNA-122, RG-101. Hepatology, 2017; 66(1):57–68; doi: 10.1002/hep.29148