Gene Therapy in CBS Deficiency

Professor Warren Kruger of the Fox Chase Cancer Centre in Philadelphia joins the podcast to talk about homocystinuria, successful trials in gene therapy, why it costs so much to make viruses and what inspires him. Long-term functional correction of cystathionine β-synthase deficiency in mice by adeno-associated viral gene therapy Hyung-Ok Lee et al https://doi.org/10.1002/jimd.12437 Interested listeners may also wish to look at: Guidelines for the diagnosis and management of cystathionine beta-synthase deficiency Andrew A. M. Morris, et al https://doi.org/10.1007/s10545-016-9979-0