Adeno-Associated Viral Vector Gene Therapy for Cystic Fibrosis: The Problems and Possible Solutions

Esquivel, Rio Layla
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Adeno-associated virus (AAV) vectors have not shown to decrease the effects of cystic fibrosis (CF) with efficacy. The cystic fibrosis transmembrane conductance regulator (CFTR) gene is too large to be successfully transported via AAV vectors. Furthermore, the presence of a thick mucus layer, covering the lungs of humans with CF, prevents the treatment from reaching the epithelial cells. This paper describes how three different research groups have attempted to mitigate these issues associated with AAV vectors in the treatment of CF by shortening the CFTR gene and necessary regulatory regions and by mutating the AAV vector capsid to reduce heparin binding. Cumulatively, these developments may help make AAV vectors for CF gene therapy a promising concept once again.