We've Come A Long Way Since Mendel
Explore the site to discover the history and the process of gene replacement therapy (GRT). AAV-mediated GRT requires 4 critical elements1-5:
requires 4 critical elements1-5:
|A nonreplicating adeno-associated virus (AAV) vector||A stable, fully-functioning transgene||Self-complementary DNA technology||A promoter that initiates gene expression|
Learn about the history of gene therapy and how it may treat monogenic diseases.