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Autor:
Yafeng Song, Andrew F. Mead, Leonard T. Su, Hansell H. Stedman, Mihail Petrov, Marilyn A. Mitchell, Margaret E Choi, Joe N. Kornegay, Xiangping Lu, Daniel J VanBelzen, Maxwell Joffe, Alock Malik, Tejvir Tejvir Khurana
Publikováno v:
Molecular Therapy. 24:S37
The majority of mutations causing Duchenne muscular dystrophy (DMD) are multi-exon, frameshifting deletions, complicating therapy with recombinant dystrophin because of the potential for chronic immune recognition of the “non-self” protein. The p