Oligonucleotide and gene therapies could facilitate a significant increase in the ability to treat up to 7,000 genetic diseases. However excessive costs, technological challenges and inefficient manufacturing processes respresent only a few of the complications that can restrict the release of new therapeutics.
This compendium aims to help you understand these challenges and how to overcome them, by assisting you with:
Characterization of starting materials, side reactions and final products
Quality control
Strategies for understanding the impact of gene editing
