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Autor:
Woon Chee Yee, Qian Bin Xiong, Jian Li Wang, Zacharias A.D. Pramono, Poh San Lai, Yi Jun Chen, Keng Boon Wee
Publikováno v:
Human Gene Therapy. 23:781-790
Antisense oligonucleotide (AON)-mediated exon skipping to restore dystrophin expression in Duchenne muscular dystrophy (DMD) therapy shown promise in a number of human clinical trials. Current AON design methods are semi-empirical, involving either t