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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:

A nonreplicating adeno-associated virus (AAV) vector A stable, fully-functioning transgene Self-complementary DNA technology A promoter that initiates gene expression
See How GRT Works
Get to Know GRT >

Learn about the history of gene therapy and how it may treat monogenic diseases.

How GRT Works >

Trace the delivery path of a GRT from outside the body to inside the cell nucleus.

Building a GRT >

Explore the composition of a GRT and how viral vectors act as a delivery system.

News >

Check out news and publications
about GRT.