Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models

Song, YF; Morales, L; Malik, AS; Mead, AF; Greer, CD; Mitchell, MA; Petrov, MT; Su, LT; Choi, ME; Rosenblum, ST; Lu, XP; VanBelzen, DJ; Krishnankutty, RK; Balzer, FJ; Loro, E; French, R; Propert, KJ; Zhou, SZ; Kozyak, BW; Nghiem, PP; Khurana, TS; Kor

Stedman, HH (reprint author), Univ Penn, Dept Surg, Perelman Sch Med, Penn Muscle Inst,Penn Cardiovasc Inst, Philadelphia, PA 19104 USA.; Stedman, HH (reprint author), Corporal Michael J Crescenz Vet Affairs Med Ctr, Philadelphia, PA 19104 USA.

NATURE MEDICINE, 2019; 25 (10): 1505

Abstract

The essential product of the Duchenne muscular dystrophy (DMD) gene is dystrophin(1), a rod-like protein(2) that protects striated myocytes from contr......

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