Agenda Details

Learn more about sessions and presentations at the 25th Annual Meeting

Detailed Program

Inborn Metabolic Issues (Organized by the International Committee)

The objective of the international committee is to represent and to showcase the excellent science that is ongoing worldwide in gene and cell therapy. By focusing on the gene therapy of inborn errors of metabolism, this session will provide a rich perspective on preclinical efforts and remarkable recent clinical achievements in the treatment of various monogenic disorders with multisystem involvement. Results obtained with in vivo rAAV-based gene delivery and ex vivo lentiviral-modified hematopoietic stem cell, using gene addition or gene editing strategies, will be presented.

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