From oncogenesis to approval: the lentiviral gene therapy revolution in Wiskott-Aldrich syndrome.
This review traces the full arc of WAS gene therapy, from early preclinical proof of concept through regulatory approval, which culminated in the FDA approval of WASKYRA (etuvetidigene autotemcel) in December 2025, establishing autologous gene therapy as a licensed standard of care for WAS patients lacking a suitable H...
Z. Karimizadeh, Amirali Kalantari, M. Behfar et al.
· Regenerative medicine · 0 citations