Wiskott Aldrich syndrome – 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 Wiskott Aldrich syndrome – VJRegenMed https://mirror.vjregenmed.com 32 32 OTL-103: An autologous gene therapy for Wiskott Aldrich syndrome https://mirror.vjregenmed.com/video/kzc8zfph7hu-otl-103-an-autologous-gene-therapy-for-wiskott-aldrich-syndrome/ Thu, 03 Mar 2022 17:46:27 +0000 http://13.40.107.223/video/kzc8zfph7hu-otl-103-an-autologous-gene-therapy-for-wiskott-aldrich-syndrome/ Kent Christopherson, PhD, Orchard Therapeutics, Boston, MA, provides an overview of OTL-103, an investigational autologous gene therapy utilizing a lentiviral gene construct for patients with Wiskott Aldrich syndrome (WAS). OTL-103 aims to insert the corrected version of the WAS gene in patients, who often result in non-functioning leucocytes and platelets. Administration of the functioning cells will result in the cells differentiating into immune cells as well as the progenitor cells to platelets and a clinical trial is currently underway to assess the efficacy of OTL-103. This interview took place at Advanced Therapies Week 2022.

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