Associated Papers
Prime editing optimized RTT permits the correction of the c.8713C>T mutation in DMD gene.
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Molecular Therapy. Nucleic Acids, 2022
RNA-targeted splice-correction therapy for neuromuscular disease.
Wood MJ et al.
Brain : a Journal of Neurology, 2010
Mutational spectrum of DMD mutations in dystrophinopathy patients: application of modern diagnostic techniques to a large cohort.
Flanigan KM et al.
Human Mutation, 2009
Analysis of the EFEMP1 gene in individuals and families with early onset drusen.
Narendran N et al.
Eye (london, England), 2005
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