ps7 - MIT Biology Department 7.012: Introductory Biology -...

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NAME_____________________________________________________________TA__________________ SEC____ 7.012 Problem Set 7 FRIDAY December 3, 2004 Not due unless you have had a prior agreement with Claudette Gardel Solutions will be posted on the web. Question 1 Leukemia is type of cancer characterized by the uncontrolled proliferation of white blood cells (leukocytes). Chronic Myelogenous Leukemia (CML) is a type of leukemia primarily caused by a translocation that relocates an oncogene, called abl , from the long arm of chromosome 9 to the long arm of chromosome 22 in the bcr region (breakpoint cluster region). The resulting bcr-abl fusion protein encodes a constitutively active tyrosine kinase, which when expressed, leads to the CML phenotype. a) Gleevec is an effective drug treatment of CML. What design principles were applied to the development of Gleevec? How is this approach different from that used to develop conventional cancer therapies? b) How does Gleevec work at the molecular level? c) How many protein targets does Gleevec have? Name each target. d) Besides target specificity, what other characteristics of a drug should you consider when designing a therapy? MIT Biology Department 7.012: Introductory Biology - Fall 2004 Instructors: Professor Eric Lander, Professor Robert A. Weinberg, Dr. Claudette Gardel
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2 In contrast to drug treatments, gene therapy attempts to correct the defect by introducing a functional copy of the malfunctioning gene that is responsible for the disease phenotype. The functional gene copy can be introduced directly into the diseased organ or can be used to genetically modify isolated tissue that is later re-introduced into the patient. e) What do you need to know about the target disease in order to apply gene therapy? f) How could you deliver the functional gene into the diseased cells? g) What do you think are some of the challenges facing gene therapy? h) Based on your understanding of oncogenes, why would bcr-abl be a particularly challenging target for gene therapy?
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3 Question 2 Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder caused by mutations in the gene encoding dystrophin, a protein involved in maintaining membrane integrity in muscle cells. The dystrophin
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This note was uploaded on 11/11/2011 for the course BIO 7.012 taught by Professor Lander during the Fall '10 term at MIT.

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ps7 - MIT Biology Department 7.012: Introductory Biology -...

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