杜氏进行性肌营养不良症课件.pptVIP

DuchenneMuscular Dystrophy Sherri Garcia Muscular Dystrophy: Walk a Mile in Their Shoes CDC’s 2005 Science Ambassador Program Overview Sympathy vs. empathy Basic information about Duchenne muscular dystrophy Inheritance Prevalence Symptoms Treatments Understanding(4) Sympathy A feeling or an expression of pity or sorrow for the distress of another; compassion or commiseration Understanding (4) Empathy Identification with and understanding of anothers situation, feelings, and motives What Is Duchenne Muscular Dystrophy?(1) Genetic Progressive muscle weakness Defects in muscle proteins Death of muscle tissue Where is This Gene? Duchenne Muscular Dystrophy Inheritance(1) Mother carries the recessive gene and passes it to her child Trait is usually expressed in males only Prevalence of DMD(1) Affects one in 3500 to 5000 newborn males 1/3 of these with previous family history 2/3 sporadic Symptoms of DMD(1) Delayed developmental milestones Loss of motor skills Characteristic gait Calf hypertrophy Clumsiness/frequent falls More Symptoms of DMD(1) Muscle weakness Difficulty climbing stairs or hills Difficulty rising (Gower’s sign) Difficulty walking/running Treatments for DMD (1) To improve breathing: O2 therapy Ventilator Scoliosis surgery Tracheotomy Treatments (cont.) (1) To improve mobility: Physical therapy Surgery on tight joints Prednisone Non-steroidal medications Wheelchair Advances in Gene Therapy(3) Researches have developed minigenes, which carry instructions for a slightly smaller version of dystrophin, that can fit inside a virus Researchers have also created the so-called gutted virus, a virus that has had its own genes removed so that it is carrying only the dystrophin gene Problems with Gene Therapy(3) Muscle tissue is large and relatively impenetrable Viruses might provoke the immune system and cause the destruction of muscle fibers with the new genes Summary Empathy vs. sympathy Definition of muscular dystrophy In