PMID- 21453285
OWN - NLM
STAT- MEDLINE
DCOM- 20110823
LR  - 20191112
IS  - 1875-5631 (Electronic)
IS  - 1566-5232 (Linking)
VI  - 11
IP  - 3
DP  - 2011 Jun
TI  - Adeno-Associated Virus (AAV) Vectors in the CNS.
PG  - 181-8
AB  - Adeno-associated virus (AAV) vectors exhibit a number of properties that have 
      made this vector system an excellent choice for both CNS gene therapy and basic 
      neurobiological investigations. In vivo, the preponderance of AAV vector 
      transduction occurs in neurons where it is possible to obtain long-term, stable 
      gene expression with very little accompanying toxicity. Promoter selection, 
      however, significantly influences the pattern and longevity of neuronal 
      transduction distinct from the tropism inherent to AAV vectors. AAV vectors have 
      successfully manipulated CNS function using a wide variety of approaches 
      including expression of foreign genes, expression of endogenous genes, expression 
      of antisense RNA and expression of RNAi. With the discovery and characterization 
      of different AAV serotypes, as well as the creation of novel chimeric serotypes, 
      the potential patterns of in vivo vector transduction have been expanded 
      substantially, offering alternatives to the more studied AAV 2 serotype. 
      Furthermore, the development of specific AAV chimeras offers the potential to 
      further refine targeting strategies. These different AAV serotypes also provide a 
      solution to the immune silencing that proves to be a realistic likelihood given 
      broad exposure of the human population to the AAV 2 serotype. These advantageous 
      CNS properties of AAV vectors have fostered a wide range of clinically relevant 
      applications including Parkinson's disease, lysosomal storage diseases, Canavan's 
      disease, epilepsy, Huntington's disease and ALS. In many cases the proposed 
      therapies have progressed to phase I/II clinical trials. Each individual 
      application, however, presents a unique set of challenges that must be solved in 
      order to attain clinically effective gene therapies.
FAU - McCown, Thomas J
AU  - McCown TJ
AD  - UNC Gene Therapy Center, University of North Carolina School of Medicine, Chapel 
      Hill, 27599, USA. thomas_mccown@med.unc.edu
LA  - eng
GR  - NS 35633/NS/NINDS NIH HHS/United States
PT  - Journal Article
PT  - Research Support, N.I.H., Extramural
PT  - Research Support, Non-U.S. Gov't
PT  - Review
PL  - United Arab Emirates
TA  - Curr Gene Ther
JT  - Current gene therapy
JID - 101125446
SB  - IM
MH  - Animals
MH  - Central Nervous System/*virology
MH  - Central Nervous System Diseases/*therapy
MH  - Dependovirus/*genetics
MH  - Genetic Therapy/*methods
MH  - *Genetic Vectors
MH  - Humans
EDAT- 2011/04/02 06:00
MHDA- 2011/08/24 06:00
CRDT- 2011/04/02 06:00
PHST- 2011/01/13 00:00 [received]
PHST- 2011/03/08 00:00 [accepted]
PHST- 2011/04/02 06:00 [entrez]
PHST- 2011/04/02 06:00 [pubmed]
PHST- 2011/08/24 06:00 [medline]
AID - ABS- 69 [pii]
AID - 10.2174/156652311795684759 [doi]
PST - ppublish
SO  - Curr Gene Ther. 2011 Jun;11(3):181-8. doi: 10.2174/156652311795684759.