Tag Archives: Retinal dystrophy

The goal of gene therapy is to cure monofactorial genetic disorders, caused by mutations in a single gene. In the sequence of each gene are the instructions for the production of a protein, and every block of three bases (codon) corresponds to a specific amino acid. Alterations in the DNA sequence (mutations) affect the sequenceContinue reading “When viruses are the ally: successful gene therapy for retinal dystrophy and beta-thalassemia”