Gene therapy for Fabry disease: Progress, challenges, and outlooks on gene-editing

JM Domm, SK Wootton, JA Medin, ML West - Molecular genetics and …, 2021 - Elsevier
Gene therapy is the delivery of a therapeutic gene for endogenous cellular expression with
the goal of rescuing a disease phenotype. It has been used to treat an increasing number of …

[PDF][PDF] Challenges and opportunities of machine-guided capsid engineering for gene therapy

ED Kelsic, GM Church - Cell Gene Ther. Insights, 2019 - dynotx.com
Advances in DNA delivery will be crucial to the enablement of new gene therapies.
Pioneering efforts in develo** recombinant Adeno-Associated Virus (AAV) technology for …