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Trends in Gene Therapies for Rare Diseases

Trends in Gene Therapies for Rare Diseases Written by: Sam Bott Edited by: Genese Hendrickson "Doctors are men who prescribe medicines of which they know little, to cure diseases of which they know less, in human beings of whom they know nothing." -Voltaire The term “gene therapy” is used frequently in healthcare discussion, but what does it really mean and what implications does this hold for patients with rare disease? Gene therapy is an exciting new class of therapies that