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The fundamental understanding of the biology of hereditary rare diseases, coupled with advances in the technology to harness disarmed viruses as gene delivery vehicles, provide a ripe opportunity to investigate the next wave of potential life-changing therapies for patients.

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    Quiz

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    Barbra Streisand's first line in her first movie was...?
    A "Hello, Dolly!"
    B "Hello gorgeous!"
    C "Hi, I am here!"
    D "Papa, can you hear me?"