441 In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease.
https://www.nature.com/articles/s41467-021-24443-8
440 Prednisolone rescues Duchenne muscular dystrophy phenotypes in human pluripotent stem cell–derived skeletal muscle in vitro.
https://www.pnas.org/content/118/28/e2022960118
439 Mapping enhancer regulation across human cell types and tissues illuminates genome function and provides a resource to connect risk variants for common diseases to their molecular and cellular functions.
https://www.nature.com/articles/s41586-021-03446-x
438 Towards complete and error-free genome assemblies of all vertebrate species.
https://www.nature.com/articles/s41586-021-03451-0
437 Platypus and echidna genomes reveal mammalian biology and evolution.
https://www.nature.com/articles/s41586-020-03039-0
436 Heritable variation in telomere length predicts mortality in Soay sheep.
https://www.pnas.org/content/118/15/e2020563118
435 Systematic screening identifies therapeutic antisense oligonucleotides for Hutchinson–Gilford progeria syndrome.
https://www.nature.com/articles/s41591-021-01262-4
434 A modified antisense oligonucleotide that blocks pathogenic splicing of progeria transcripts reduces the production of progerin in key target organs and extends animal lifespan in mouse model of progeria syndrome.
https://www.nature.com/articles/s41591-021-01274-0
433 Genomic insights into the formation of human populations in East Asia.
https://www.nature.com/articles/s41586-021-03336-2
432 Patterns of de novo tandem repeat mutations and their role in autism.
https://www.nature.com/articles/s41586-020-03078-7
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