Devan L Puhl1, Anthony R D'Amato2, Ryan J Gilbert3. 1. Department of Biomedical Engineering, Rensselaer Polytechnic Institute, 110 8th Street, Troy, New York, 12180, United States; Center for Biotechnology and Interdisciplinary Studies, Rensselaer Polytechnic Institute, 1623 15th Street, Troy, New York, 12180, United States. Electronic address: puhld@rpi.edu. 2. Department of Biomedical Engineering, Rensselaer Polytechnic Institute, 110 8th Street, Troy, New York, 12180, United States; Center for Biotechnology and Interdisciplinary Studies, Rensselaer Polytechnic Institute, 1623 15th Street, Troy, New York, 12180, United States. Electronic address: ant.damato22@gmail.com. 3. Department of Biomedical Engineering, Rensselaer Polytechnic Institute, 110 8th Street, Troy, New York, 12180, United States; Center for Biotechnology and Interdisciplinary Studies, Rensselaer Polytechnic Institute, 1623 15th Street, Troy, New York, 12180, United States. Electronic address: gilber2@rpi.edu.
Abstract
Gene therapy is a promising form of treatment for those suffering from neurological disorders or central nervous system (CNS) injury, however, obstacles remain that limit its translational potential. The CNS is protected by the blood brain barrier, and this barrier blocks genes from traversing into the CNS if administered outside of the CNS. Viral and non-viral gene delivery vehicles, commonly referred to as vectors, are modified to enhance delivery efficiency to target locations in the CNS. Still, there are few gene therapy approaches approved by the FDA for CNS disease or injury treatment. The lack of viable clinical approaches is due, in part, to the unpredictable nature of many vector systems. In particular, safety concerns exist with the use of viral vectors for CNS gene delivery. To seek some alternatives to viral vectors, development of new non-viral, biomaterial vectors is occurring at a rapid rate. This review discusses the challenges of delivering various forms of genetic material to the CNS, the use and limitations of current viral vector delivery systems, and the use of non-viral, biomaterial vectors for CNS applications.
Gene therapy is a promising form of treatment for those suffering from n class="Disease">neurological disorders or n class="Disease">central nervous system (CNS) injury, however, obstacles remain that limit its translational potential. The CNS is protected by the blood brain barrier, and this barrier blocks genes from traversing into the CNS if administered outside of the CNS. Viral and non-viral gene delivery vehicles, commonly referred to as vectors, are modified to enhance delivery efficiency to target locations in the CNS. Still, there are few gene therapy approaches approved by the FDA for CNS disease or injury treatment. The lack of viable clinical approaches is due, in part, to the unpredictable nature of many vector systems. In particular, safety concerns exist with the use of viral vectors for CNS gene delivery. To seek some alternatives to viral vectors, development of new non-viral, biomaterial vectors is occurring at a rapid rate. This review discusses the challenges of delivering various forms of genetic material to the CNS, the use and limitations of current viral vector delivery systems, and the use of non-viral, biomaterial vectors for CNS applications.
Authors: Wei Ji; Yan Sun; Fang Yang; Jeroen J J P van den Beucken; Mingwen Fan; Zhi Chen; John A Jansen Journal: Pharm Res Date: 2010-11-19 Impact factor: 4.200
Authors: Dua M Anderson; Leon L Hall; Anitha R Ayyalapu; Van R Irion; Michael H Nantz; James G Hecker Journal: Hum Gene Ther Date: 2003-02-10 Impact factor: 5.695