vector system for expressing the coding sequence of a gene of interest in a cell, host cell, pharmaceutical composition, method for treating and / or preventing a condition or disease, using a nucleotide sequence of a degradation signal in a system vector and method for decreasing expression of a protein in truncated form

摘要

The present invention relates to constructs, vectors, relative host cells and pharmaceutical compositions which allow effective gene therapy, in particular of genes larger than 5 kb.