https://mirror.vjregenmed.com The Video Journal of Regenerative Medicine Thu, 26 Aug 2021 17:53:55 +0000 en-US hourly 1 https://wordpress.org/?v=6.5.2 https://d2xz56kaqxj8if.cloudfront.net/wp-content/uploads/2023/09/12102509/VJR-Favicon.png https://mirror.vjregenmed.com 32 32 https://mirror.vjregenmed.com/video/6ound_q4uoa-ctns-rd-04-hsc-gene-therapy-for-cystinosis/ Tue, 22 Jun 2021 13:25:11 +0000 http://13.40.107.223/video/6ound_q4uoa-ctns-rd-04-hsc-gene-therapy-for-cystinosis/ Currently, cystine depleting therapy using cysteamine is used to treat cystinosis, however, this is a very difficult, non-curative treatment regimen associated with numerous side effects. Stephanie Cherqui, PhD, University of California, San Diego, La Jolla, CA, discusses the updated results of an ongoing Phase I/II trial (NCT03897361) investigating the efficacy of CTNS-RD-04, a novel autologous hematopoietic stem and progenitor cell (HSPC)-based gene therapy, in patients with cystinosis. In the first three male patients treated, CTNS-RD-04 was found to decrease cystine crystal formation, decrease cystine content in white blood cells and improve photophobia. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.

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