PMID- 22264214
OWN - NLM
STAT- MEDLINE
DCOM- 20120524
LR  - 20191112
IS  - 2212-3334 (Electronic)
IS  - 1872-2148 (Linking)
VI  - 6
IP  - 1
DP  - 2012 Jan
TI  - Present and future of adeno associated virus based gene therapy approaches.
PG  - 47-66
AB  - During the last 20 years, transgenic constructs based on adeno associated virus 
      (AAV) have been tested in disease models and proved their efficacy to revert a 
      wide range of pathologies without major side effects. Based on these results, up 
      to 20 clinical trials have been set up to prove therapeutic effect of AAV vectors 
      on neurodegenerative diseases, retinopathies and neuromuscular diseases, among 
      others. It has been shown that AAV vectors support localized long-term, gene 
      expression in the central nervous system, and that restoration of visual function 
      can be achieved in Leber's congenital amaurosis retinopathy. The clinical trials 
      also highlighted new challenges for AAV mediated gene transfer, such as the 
      circumvention of T-cell response to transduced cells. Currently, miniaturized and 
      codon-optimized transgenes, exon skipping cassettes, novel tissue-specific 
      promoters and vector chimeras with tissue-selective tropism are being tested to 
      improve the efficiency and safety of transgene delivery, as required to meet 
      pharmaceutical industry standards. The aim of this review is to revise the latest 
      patents and news on AAV vectors, in order to summarize the state of the art and 
      the potential issues that still need to be faced by pharmaceutical companies for 
      successful gene transfer and commercialization of AAV-based drugs.
FAU - Ortolano, Saida
AU  - Ortolano S
AD  - Department of Pathology and Neuropathology, Complexo Hospitalario Universitario 
      de Vigo, Hospital Meixoeiro, Spain. saida.ortolano@sergas.es
FAU - Spuch, Carlos
AU  - Spuch C
FAU - Navarro, Carmen
AU  - Navarro C
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PT  - Review
PL  - United Arab Emirates
TA  - Recent Pat Endocr Metab Immune Drug Discov
JT  - Recent patents on endocrine, metabolic & immune drug discovery
JID - 101299743
SB  - IM
MH  - Animals
MH  - Dependovirus/*genetics/growth & development
MH  - Genetic Therapy/adverse effects/legislation & jurisprudence/*trends
MH  - *Genetic Vectors/adverse effects
MH  - Humans
MH  - Patents as Topic
EDAT- 2012/01/24 06:00
MHDA- 2012/05/25 06:00
CRDT- 2012/01/24 06:00
PHST- 2011/12/07 00:00 [received]
PHST- 2011/12/15 00:00 [revised]
PHST- 2011/12/19 00:00 [accepted]
PHST- 2012/01/24 06:00 [entrez]
PHST- 2012/01/24 06:00 [pubmed]
PHST- 2012/05/25 06:00 [medline]
AID - EMI-EPUB-20120116-003 [pii]
AID - 10.2174/187221412799015245 [doi]
PST - ppublish
SO  - Recent Pat Endocr Metab Immune Drug Discov. 2012 Jan;6(1):47-66. doi: 
      10.2174/187221412799015245.