Adeno Associated Virus (AAV) for Cell and Gene Therapy
Type
Blog Post
...caused by a mutation in a single gene, it can potentially be treated by correcting the mutation or replacing...bubble boy syndrome. This disease is caused by mutations in the common cytokine receptor gamma chain (c... so, their T-cells were grown in vitro, their mutations corrected, and the T-cells were transferred back...T-cells outside the body and thereby correct the mutations using retroviral vectors. However, many diseases...improved specificity.
AAV vectors are able to facilitate and enhance DNA repair via homology directed ...especially amenable to correcting disease-associated mutations.
AAV vectors maintain persistent transgene expression...congenital amurosis (LCA). This disease is caused by mutations in the gene RPE65. It is characterized by childhood...