Francesca Montanari
Adeno-associated viruses functionalized with Zwitterionic polymers for Duchenne muscular dystrophy.
Rel. Valentina Alice Cauda, Marta Guerra Rebollo, Despoina Despotopoulou. Politecnico di Torino, Corso di laurea magistrale in Ingegneria Biomedica, 2026
Abstract
Duchenne Muscular Dystrophy (DMD) is an X-linked neuromuscular disorder char acterized by progressive muscle de-generation in young male populations. Current therapeutic strategies function as symptom-mitigating agents rather than definitive cures, highlighting the urgent need for novel and more effective treatments. The recent FDA-approval of the first gene therapy, based on adeno-associated viruses (AAVs) delivering the micro-dystrophin gene, to restore protein function, brought a lot of optimism. However, important limitations remain, including the requirement for high vector doses, off-target liver accumulation, and, most importantly, immune recognition of the viral capsids. For this aim, a surface-tethering strategy was employed, using a two-step re action process.
Initially, the CliCr®Osu linker was conjugated to surface-exposed amino groups of viral capsids
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