PMID- 10998427
OWN - NLM
STAT- MEDLINE
DCOM- 20001025
LR  - 20190605
IS  - 1107-3756 (Print)
IS  - 1107-3756 (Linking)
VI  - 6
IP  - 4
DP  - 2000 Oct
TI  - Gene therapy vectors based on adeno-associated virus: characteristics and 
      applications to acquired and inherited diseases (review).
PG  - 363-75
AB  - Adeno-associated virus (AAV), a defective parvovirus, was discovered more than 30 
      years ago. Interest in this virus for human gene therapy applications focuses on 
      its non-pathogenicity, broad tropism and infectivity, site-specific integration 
      and long-term persistence. The field of rAAV research has considerably advanced: 
      titers of 1014 p/ml have been achieved, plasmid systems devised to produce 
      helper-free viruses, chimaeric vectors combining properties of rAAV ITRs and 
      large sequence capacity from Ad/HS vectors in parallel with the revolutionary 
      intron strategy based on heterodimerisation of the forming concatamers have 
      expanded the vector capacity. Muscle cells and neurons (post-mitotic cells) are 
      amongst the most efficient targets of rAAV delivery and AAV receptors and 
      co-receptors have been identified. This review will describe advances in the 
      field of rAAV technology that overcome certain limitations of the vector as a 
      gene delivery system and overview applications involving these recombinant 
      vectors for the treatment of acquired and inherited diseases.
FAU - Athanasopoulos, T
AU  - Athanasopoulos T
AD  - Department of Biochemistry, Royal Holloway, University of London, Egham, Surrey, 
      TW20 0EX, UK.
FAU - Fabb, S
AU  - Fabb S
FAU - Dickson, G
AU  - Dickson G
LA  - eng
PT  - Journal Article
PT  - Review
PL  - Greece
TA  - Int J Mol Med
JT  - International journal of molecular medicine
JID - 9810955
SB  - IM
MH  - Adenoviridae/*genetics
MH  - Animals
MH  - Disease
MH  - Gene Transfer Techniques
MH  - Genetic Diseases, Inborn/genetics/therapy
MH  - *Genetic Therapy
MH  - Genetic Vectors/*genetics
MH  - Humans
MH  - Therapeutics
RF  - 109
EDAT- 2000/09/22 11:00
MHDA- 2001/02/28 10:01
CRDT- 2000/09/22 11:00
PHST- 2000/09/22 11:00 [pubmed]
PHST- 2001/02/28 10:01 [medline]
PHST- 2000/09/22 11:00 [entrez]
AID - 10.3892/ijmm.6.4.363 [doi]
PST - ppublish
SO  - Int J Mol Med. 2000 Oct;6(4):363-75. doi: 10.3892/ijmm.6.4.363.