The invention also relates to methods of identifying molecules that inhibit the nonsense-mediated mRNA decay pathway, and the use of such molecules for treatment of disorders associated with nonsense mutations.
The present disclosure relates to compositions and methods for inhibiting nonsense- mediated mRNA decay in a gene- specific manner, for example in the treatment of diseases or disorders caused by nonsense mutations.
Molecular misreading seems to be a general cause for the synthesis of nonsense transcripts.
The present invention relates to functional proteins encoded by nucleic acid sequences comprising a nonsense mutation.
The method of electroporation described herein is useful for expanding nonsense suppression methodology to mammalian cell expression systems for the incorporation of unnatural amino acids.
Variant proteins and nucleic acids including RNA splice variants, mis-sense mutants and non-sense mutants are also described.
Also provided are methods compositions and kits for detecting the BCR-ABL truncation variants.