A multinational clinical trial published in the journal Nature Medicine has shown that lentiviral hematopoietic stem/progenitor cell gene therapy can be a safe and efficacious treatment option in patients with rare Wiskott-Aldrich syndrome (WAS). Here is an infographic on stem cell gene therapy resolving severe symptoms in WAS patients. Take a look! In your opinion, can stem cell gene therapy be a better treatment for rare diseases? Reference: Magnani A, Semeraro M, Adam F, and et al.
Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome. Nat Med. 2022 Jan;28(1):71-80. DOI: 10.1038/s41591-021-01641-x.
