Advances in oligonucleotide drug delivery
Author:
Publisher
Springer Science and Business Media LLC
Subject
Drug Discovery,Pharmacology,General Medicine
Link
https://www.nature.com/articles/s41573-020-0075-7.pdf
Reference302 articles.
1. Kim, J. et al. Patient-customized oligonucleotide therapy for a rare genetic disease. N. Engl. J. Med. 381, 1644–1652 (2019). This is the first study to utilize an oligonucleotide therapy tailored to a single patient.
2. Giorgio, E. et al. Allele-specific silencing as treatment for gene duplication disorders: proof-of-principle in autosomal dominant leukodystrophy. Brain 142, 1905–1920 (2019).
3. Southwell, A. L. et al. In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides. Mol. Ther. 22, 2093–2106 (2014).
4. Miller, V. M. et al. Allele-specific silencing of dominant disease genes. Proc. Natl Acad. Sci. USA 100, 7195–7200 (2003).
5. Klein, A. F. et al. Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice. J. Clin. Invest. 129, 4739–4744 (2019).
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