Planet of the AAVs: The Spinal Cord Injury Episode

0553041 - ÚEM 2022 RIV CH eng J - Journal Article
Štěpánková, Kateřina - Jendelová, Pavla - Machová-Urdzíková, Lucia
Planet of the AAVs: The Spinal Cord Injury Episode.
Biomedicines. Roč. 9, č. 6 (2021), č. článku 613. E-ISSN 2227-9059
R&D Projects: GA MŠMT(CZ) EF15_003/0000419; GA ČR(CZ) GA19-10365S
Institutional support: RVO:68378041
Keywords : spinal cord injury * gene therapy * adeno-associated virus * AAV vector
OECD category: Neurosciences (including psychophysiology
Impact factor: 4.757, year: 2021
Method of publishing: Open access
https://www.mdpi.com/2227-9059/9/6/613

The spinal cord injury (SCI) is a medical and life-disrupting condition with devastating consequences for the physical, social, and professional welfare of patients, and there is no adequate treatment for it. At the same time, gene therapy has been studied as a promising approach for the treatment of neurological and neurodegenerative disorders by delivering remedial genes to the central nervous system (CNS), of which the spinal cord is a part. For gene therapy, multiple vectors have been introduced, including integrating lentiviral vectors and non-integrating adeno-associated virus (AAV) vectors. AAV vectors are a promising system for transgene delivery into the CNS due to their safety profile as well as long-term gene expression. Gene therapy mediated by AAV vectors shows potential for treating SCI by delivering certain genetic information to specific cell types. This review has focused on a potential treatment of SCI by gene therapy using AAV vectors.
Permanent Link: http://hdl.handle.net/11104/0328079