Examine how various genetic medicines are delivered to specific tissues—such as the liver, nervous system, eye, skin, and muscle—to treat disease.
Acquire an in-depth understanding of the current landscape of genetic medicines, including recent advancements and key challenges.
Understand how the pathophysiology of each disease shapes the choice of therapeutic approach.
While the molecular basis of many genetic diseases has been understood for decades, our ability to treat them has progressed more slowly. Genetic medicines are an emerging class of diverse therapeutics that are changing this landscape by offering new strategies for targeting the underlying mechanisms of disease. With each new approval, genetic medicines are supplying the tools that researchers, clinicians, and developers need to expand the number of druggable targets and treat more conditions, including those once considered untreatable.
This advanced HMX short course offers a unique way for professionals to learn from Harvard Medical School faculty and industry experts at the forefront of genetic medicine. Whether you are new to the field or seeking to advance your expertise, you will gain relevant, practical insights into recently FDA-approved therapies, including small interfering RNAs, antisense oligonucleotides, gene therapies, and gene editing technologies. You will examine how these therapies are designed and delivered to specific tissues to treat various genetic diseases, as well as learn about the clinical data that led to their approval. You will also explore practical concerns, such as the importance of patient choice and the issues of navigating regulatory considerations.
By the end of this course, you will be equipped with a strong understanding of relevant genetic medicines and the promise of this exciting field.
