Title: Understanding the Genetic and Molecular Basis of a Disease and its Physiological Implications: Current Treatment Options and Development Strategies for Therapy

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Title: Understanding the Genetic and Molecular Basis of a Disease and its Physiological Implications: Current Treatment Options and Development Strategies for Therapy

you will write a short essay (no more than 1000 words total) addressing each of the following.  What is the genetic basis of the disease?  What is the molecular basis of the disease? (What is abnormal at the cellular, biochemical, or molecular level?)  What are the physiologic implications of the disease? (What is abnormal about the physiology of people with this disease?)  What are the current treatment optioluvns or the development strategies for therapy?

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