Homework

Weekly homework submissions:

• Week 1 HW: Principles and Practices

  Homework 1 Introduction Over the past few decades, we have developed increasingly sophisticated methods to sequence and understand genomes and understood the genetic basis of several diseases. There are approximately 10,000 known monogenic diseases - caused by mutations to a single gene. But out of these, very few are treatable even though we understand the mechanism. This is due to challenges with delivering genes to the right places - common vectors like AAV don’t have the capacity to store large genes, each vector has varying specificity and duration of expression and many are immunogenic.