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- Evaluation of cystamine-modified hyaluronic acid/chitosan polyplex as retinal gene vectorPublication . Oliveira, Ana V.; Marcelo, Adriana; Costa, Ana M. Rosa da; Silva, Gabriela A.A successful gene therapy approach can prevent or treat congenital and acquired diseases. However, there is still no ideal non-viral vector for gene delivery in a safe and timely manner. In this report the anionic polymer hyaluronic acid (HA) was investigated as a potential vector for gene therapy. Due to its intrinsic characteristics it constitutes an excellent candidate to deliver therapeutic genes, pending the modification of its surface charge.
- CYP46A1- gene therapy improves Machado-Joseph disease in mouse modelsPublication . Nóbrega, Clévio; Mendonca, L. S.; Marcelo, Adriana; Lamaziere, A.; Tome, S.; Despres, G.; Matos, C.; Mechmet, Fatich; Langui, D.; den Dunnen, W.; de Almeida, L. P.; Cartier, N.; Alves, S.Aims/Context: Machado-Joseph Disease (MJD) is a neurodegenerative disease associated with extensive neuronal death. Defects in brain cholesterol metabolism may contribute to neurodegenerative diseases.