Therapies for rare diseases: therapeutic modalities, progress and challenges ahead

作者：Erik Tambuyzer, Benjamin Vandendriessche, Christopher P. Austin, Philip J. Brooks, Kristina Larsson, Katherine I. Miller Needleman, James Valentine, Kay E. Davies, Stephen C. Groft, Robert A. Preti, Tudor I. Oprea, Marco Prunotto · 发表于：Nature Reviews Drug Discovery · 年份：2019 · DOI：10.1038/s41573-019-0049-9 · 被引用次数：362 · 研究领域：CRISPR and Genetic Engineering、Genomics and Rare Diseases、Genetics and Neurodevelopmental Disorders