… These properties have made chitosan an attractive non-viral vector for gene therapy. In …
gene therapy, where enhanced particle uptake was evidenced by human blood-brain barrier …

… Abstract—Gene therapy is the straightforward approach for the application of recent … One
of the major obstacles in the development of gene therapy is the delivery of the effector to and …

… development of successful gene therapies, focusing on direct in vivo administration of viral
vectors… crosses the blood-brain barrier was engineered to carry the SMN1 gene, and given as …

… viral vector systems for gene therapy are promising treatment … to risk assessment and
developing procedures to minimize … the viral vector systems utilized for gene therapy and some of …

… viral vector-based gene therapy landscape, we focus on these three highly regarded vector
… cellular toxicity continue to be major obstacles for Ad gene therapy, despite the fact that Ad …

… Design of gene therapy vectors and their clinical development are advancing rapidly. This
… some of the major successes in clinical gene therapy of recent years. Gene therapy seeks to …

… viral vectors, non-viral gene therapy approaches will not be discussed further. Instead, an
overview on different viral vectors … However, the large number of viral vectors evaluated for a …

… people with LeberLs Congenital Amaurosis [6,7]. Murine γ-retroviral vectors have also been
employed in gene therapy … to be the biggest barrier as three gene therapies gain regulatory …

… face three major barriers to the use of nonviral vectors in gene … developing gene delivery
systems is also a major challenge. Although there are still many problems associated with gene …

… the main obstacles to a wider clinical use of these vectors. Efficient and safe non-viral vectors
are emerging as a promising alternative and facilitate clinical gene therapy studies. Here, …