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Listen "Gene Therapy in CBS Deficiency"
Episode Synopsis
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
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
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