Why do we need Gene Therapy?
Development path for a gene therapy
In Vivo vs. Ex Vivo
Approaches
Gene Replacement Approach
Gene Supplementation or Gene Agnostic Approach
Gene Editing, Base Editing, & Prime Editing Approaches
Gene Silencing Approach
How to reach cell-type specificity
Vectors for Gene Therapy
Strategies to bypass size limitation of AAV vectors
When is gene therapy suitable?
Zuletzt geändertvor 2 Jahren
