Atidarsagene autotemcel – VJRegenMed https://mirror.vjregenmed.com The Video Journal of Regenerative Medicine Tue, 16 Aug 2022 09:39:49 +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 Atidarsagene autotemcel – VJRegenMed https://mirror.vjregenmed.com 32 32 Novel gene therapies for metachromatic leukodystrophy https://mirror.vjregenmed.com/video/doz45bpgrwe-novel-gene-therapies-for-metachromatic-leukodystrophy/ Thu, 03 Mar 2022 17:46:25 +0000 http://13.40.107.223/video/doz45bpgrwe-novel-gene-therapies-for-metachromatic-leukodystrophy/ Kent Christopherson, PhD, Orchard Therapeutics, Boston, MA, describes the development and mechanism of action of atidarsagene autotemcel, a gene therapy consisting of an autologous CD34⁺ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced using a lentiviral vector. The gene therapy, which has been approved in the EU, aims to treat metachromatic leukodystrophy (MLD) and the vector encodes the human arylsulfatase-A (ARSA) gene, which is faulty in patients with MLD. HSPC cells with the functional gene can subsequently produce the correct protein, which can be trafficked and permanently integrated in the central nervous system (CNS). This interview took place at Advanced Therapies Week 2022.

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