Size matters: Fighting repeat expansion size in fragile X syndrome using antisense oligonucleotides
Author:
Affiliation:
1. Center for Neural Science, New York University, New York, NY 10003
2. New York University Neuroscience Institute, New York University Grossman School of Medicine, New York, NY 10016
Funder
HHS | NIH | Eunice Kennedy Shriver National Institute of Child Health and Human Development
Publisher
Proceedings of the National Academy of Sciences
Subject
Multidisciplinary
Link
https://pnas.org/doi/pdf/10.1073/pnas.2309678120
Reference23 articles.
1. Fragile X syndrome
2. Fragile X syndrome: a review of clinical and molecular diagnoses
3. Molecular and cellular genetics of fragile X syndrome
4. The molecular biology of FMRP: new insights into fragile X syndrome
5. S. Shah Antisense oligonucleotide rescue of CGG expansion-dependent FMR1 mis-splicing in fragile X syndrome restores FMRP. Proc. Natl. Acad. Sci. U.S.A. 120 e2302534120 (2023). e2302534120
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