Splicing Modulation as Therapy for Human Genetic Disease
Author:
Publisher
Wiley
Link
https://onlinelibrary.wiley.com/doi/pdf/10.1002/9780470015902.a0024458
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3. Molecular mechanisms of action of antisense drugs
4. Lipid-lowering treatment for homozygous familial hypercholesterolaemia – Authors' reply
5. An Intronic Growth Hormone Receptor Mutation Causing Activation of a Pseudoexon Is Associated with a Broad Spectrum of Growth Hormone Insensitivity Phenotypes
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