A humanized mouse model of Duchenne Muscular Dystrophy

This is a novel mouse model containing the full length human dystrophin gene harboring a mutation that models the cause of Duchenne muscular dystrophy. No mouse dystrophin is made, such that therapeutics focused on correction of the human gene can be tested in this model.

Duke File (IDF) Number

T-005089

Inventor(s)

  • Gersbach, Charles
  • Robinson-Hamm, Jacqueline

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College

Pratt School of Engineering