Associated Papers
To splice or not to splice: pseudoexons in neurological disease and opportunities for intervention.
Fletcher S et al.
Current Opinion in Genetics & Development, 2025
Regulating PCCA gene expression by modulation of pseudoexon splicing patterns to rescue enzyme activity in propionic acidemia.
Spangsberg Petersen US et al.
Molecular Therapy. Nucleic Acids, 2024
Splice-Modulating Antisense Oligonucleotides as Therapeutics for Inherited Metabolic Diseases.
Chen S et al.
Biodrugs : Clinical Immunotherapeutics, Biopharmaceuticals and Gene Therapy, 2024
Therapeutic potential of splice-switching oligonucleotides.
Bauman J et al.
Oligonucleotides, 2009
Propionic and methylmalonic acidemia: antisense therapeutics for intronic variations causing aberrantly spliced messenger RNA.
Rincón A et al.
American Journal of Human Genetics, 2007
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