Towards Splicing Therapy for Lysosomal Storage Disorders: Methylxanthines and Luteolin Ameliorate Splicing Defects in Aspartylglucosaminuria and Classic Late Infantile Neuronal Ceroid Lipofuscinosis

Splicing defects caused by mutations in the consensus sequences at the borders of introns and exons are common in human diseases. Such defects frequently result in a complete loss of function of the protein in question. Therapy approaches based on antisense oligonucleotides for specific gene mutatio...

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Detalles Bibliográficos
Autores principales: Banning, Antje, Tikkanen, Ritva
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8616534/
https://www.ncbi.nlm.nih.gov/pubmed/34831035
http://dx.doi.org/10.3390/cells10112813

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8616534/
https://www.ncbi.nlm.nih.gov/pubmed/34831035
http://dx.doi.org/10.3390/cells10112813

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