What you'll learn

  • Nucleic Acid Immunity and Delivery Challenges

  • Removing or Modifying Endogenous Information

  • Clinical Linkage: Treating Sickle Cell Disease with Nucleic Acid Therapeutics

  • The Future of Nucleic Acid Therapeutics

Course description

Even with the thousands of approved small molecule drugs at our disposal, there remains a lack of effective prevention and treatment for many diseases. Fortunately, a new and exciting drug modality has emerged called nucleic acid therapeutics that has the potential to address current unmet needs.

This class of drugs uses nucleic acids and closely related compounds for a variety of applications, including regulating gene expression, stimulating the immune system, editing the genome, or supplying new genetic information, with the goal of treating or preventing disease. Learning about nucleic acid therapeutics is therefore essential for anyone working in health care and related sectors. This advanced course offers a unique way for professionals to learn from leading Harvard Medical School faculty and industry leaders about nucleic acid therapeutics the advances in this field that have the potential to treat, prevent, and even cure genetic diseases.

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