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

admin_vjr_user — Fri, 24 Dec 2021 10:13:24 +0000

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.

Eladocagene exuparvovec: AAV2-mediated gene therapy for AADC deficiency

admin_vjr_user — Fri, 24 Dec 2021 10:07:54 +0000

Paul Wuh-Liang Hwu, MD, PhD, National Taiwan University Hospital, Taipei, Taiwan, describes the rationale and ongoing research into gene therapy for aromatic l-amino acid decarboxylase (AADC) deficiency. AADC deficiency is a very rare genetic disorder caused by mutations in the dopa decarboxylase gene leading to reduced AADC enzyme activity, resulting in delayed cognitive and speech development. Dr Hwu describes the study of eladocagene exuparvovec, a recombinant adeno-associated viral vector (AAV) serotype 2 carrying cDNA encoding the human AADC gene, which is administered via bilateral infusion to the putamen. Results demonstrate the efficacy of this treatment in improving cognition and motor development. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.

Paul Wuh-Liang Hwu – VJRegenMed

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

Eladocagene exuparvovec: AAV2-mediated gene therapy for AADC deficiency