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Learn about cutting-edge base editing techniques for treating triplet repeat disorders in this 47-minute medical genetics primer lecture. Explore how base editing technology can be applied to address the genetic mechanisms underlying Huntington's Disease and Friedrich's Ataxia, two devastating neurological conditions caused by expanded triplet repeat sequences. Discover the molecular basis of these disorders, understand how triplet repeat expansions lead to disease pathology, and examine innovative therapeutic approaches using precision genome editing tools. Gain insights into the challenges and opportunities of targeting repetitive DNA sequences for potential treatments. The presentation, delivered by Dr. Mandan Arbab from Boston Children's Hospital, Harvard Medical School, and the Broad Institute, provides an in-depth introduction to this specialized area of genetic medicine as part of the Medical and Population Genetics Primer series designed for research technicians, graduate students, postdoctoral fellows, and investigators entering the field of human genetics.
