Chiara Leandro
New treatment for Duchenne muscular dystrophy based on zwitterionic polymers to encapsulate Adeno-Associated Virus vectors.
Rel. Valentina Alice Cauda. Politecnico di Torino, Corso di laurea magistrale in Ingegneria Biomedica, 2025
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Abstract
Duchenne Muscular Dystrophy (DMD) is a severe genetic disorder caused by mutations in the DMD gene, leading to progressive muscle degeneration. Current treatments, such as corticosteroids and rehabilitation, only slow disease progression without providing a cure. Gene therapy offers a promising alternative by directly addressing the genetic cause, with adeno-associated viruses (AAVs) being the most effective vectors due to their low toxicity, high stability and long-lasting transgene expression. But despite these advantages, there are also some limits. First, the size of the genome has to be lower than 4,7 kb due to the low capacity of the vector. To overcome this, researchers developed a shortened gene called micro-dystrophin which is small enough to fit inside the vector.
Moreover, there is the problem of the host immune response: neutralizing antibodies (nAbs) are present in a large part of the population and can block the transgene delivery
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