GENE SILENCING

摘要

The invention provides a mirtron capable of reducing the expression of the LRRK2 gene in a mammalian cell, the sequence of the mirtron comprising: (i) a 5' splice site; (ii) a 3' splice site; (iii) a branch-point recognition sequence; (iv) a 3' polypyrimidine tract greater than 15 nucleotides in length; and (v) an antisense sequence that is at least partially complementary to a sequence in the LRRK2 mRNA, or a gene capable of expressing said mirtron.