Educational content on VJRegenMed is intended for healthcare professionals only. By visiting this website and accessing this information you confirm that you are a healthcare professional.

Share this video  

ESGCT 2021 | Highlights from ESGCT 2021: gene therapy clinical data, advances in gene editing & AAV toxicity

Paul Wuh-Liang Hwu, MD, PhD, National Taiwan University Hospital, Taipei, Taiwan, offers some of his highlights from the 2021 European Society of Gene & Cell Therapy (ESGCT) Congress. He discusses his excitement around promising clinical data presented at the meeting evaluating various gene therapy approaches in rare disorders such as mucolipidosis III, as well as advances in gene editing and the promise of base editing. He also considers the importance of talks on the safety issues associated with gene therapy, such as the integration of adeno-associated virus (AAV) vectors into the liver, resulting in toxicity. This interview took place at the ESGCT Virtual Congress 2021.


Wuh-Liang Hwu participated as an advisory board member, received consulting fees, and was a speaker for PTC Therapeutics, Inc., BioMarin, and Sanofi. He was a grant recipient for PTC Therapeutics, Inc. and BioMarin and a research investigator for PTC Therapeutics.