PMID- 28239341
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20200930
IS  - 1662-5129 (Print)
IS  - 1662-5129 (Electronic)
IS  - 1662-5129 (Linking)
VI  - 11
DP  - 2017
TI  - Adeno-Associated Viral Vectors Serotype 8 for Cell-Specific Delivery of 
      Therapeutic Genes in the Central Nervous System.
PG  - 2
LID - 10.3389/fnana.2017.00002 [doi]
LID - 2
AB  - Adeno-associated viruses (AAVs) have become highly promising tools for research 
      and clinical applications in the central nervous system (CNS). However, specific 
      delivery of genes to the cell type of interest is essential for the success of 
      gene therapy and therefore a correct selection of the promoter plays a very 
      important role. Here, AAV8 vectors carrying enhanced green fluorescent protein 
      (eGFP) as reporter gene under the transcriptional control of different 
      CNS-specific promoters were used and compared with a strong ubiquitous promoter. 
      Since one of the main limitations of AAV-mediated gene delivery lies in its 
      restricted cloning capacity, we focused our work on small-sized promoters. We 
      tested the transduction efficacy and specificity of each vector after 
      stereotactic injection into the mouse striatum. Three glia-specific AAV vectors 
      were generated using two truncated forms of the human promoter for glial 
      fibrillar acidic protein (GFAP) as well as a truncated form of the murine GFAP 
      promoter. All three vectors resulted in predominantly glial expression; however 
      we also observed eGFP expression in other cell-types such as oligodendrocytes, 
      but never in neurons. In addition, robust and neuron-specific eGFP expression was 
      observed using the minimal promoters for the neural protein BM88 and the neuronal 
      nicotinic receptor beta2 (CHRNB2). In summary, we developed a set of AAV vectors 
      designed for specific expression in cells of the CNS using minimal promoters to 
      drive gene expression when the size of the therapeutic gene matters.
FAU - Pignataro, Diego
AU  - Pignataro D
AD  - Department of Gene Therapy and Regulation of Gene Expression, Center for Applied 
      Medical ResearchPamplona, Spain; Department of Neurosciences, Center for Applied 
      Medical ResearchPamplona, Spain.
FAU - Sucunza, Diego
AU  - Sucunza D
AD  - Department of Gene Therapy and Regulation of Gene Expression, Center for Applied 
      Medical ResearchPamplona, Spain; Department of Neurosciences, Center for Applied 
      Medical ResearchPamplona, Spain; Centro de Investigacion Biomedica en Red sobre 
      Enfermedades NeurodegenerativasSpain.
FAU - Vanrell, Lucia
AU  - Vanrell L
AD  - Department of Gene Therapy and Regulation of Gene Expression, Center for Applied 
      Medical Research Pamplona, Spain.
FAU - Lopez-Franco, Esperanza
AU  - Lopez-Franco E
AD  - Department of Neurosciences, Center for Applied Medical Research Pamplona, Spain.
FAU - Dopeso-Reyes, Iria G
AU  - Dopeso-Reyes IG
AD  - Department of Neurosciences, Center for Applied Medical ResearchPamplona, Spain; 
      Centro de Investigacion Biomedica en Red sobre Enfermedades 
      NeurodegenerativasSpain; Instituto de Investigacion Sanitaria de NavarraPamplona, 
      Spain.
FAU - Vales, Africa
AU  - Vales A
AD  - Department of Gene Therapy and Regulation of Gene Expression, Center for Applied 
      Medical Research Pamplona, Spain.
FAU - Hommel, Mirja
AU  - Hommel M
AD  - Department of Gene Therapy and Regulation of Gene Expression, Center for Applied 
      Medical ResearchPamplona, Spain; Instituto de Investigacion Sanitaria de 
      NavarraPamplona, Spain.
FAU - Rico, Alberto J
AU  - Rico AJ
AD  - Department of Neurosciences, Center for Applied Medical ResearchPamplona, Spain; 
      Centro de Investigacion Biomedica en Red sobre Enfermedades 
      NeurodegenerativasSpain; Instituto de Investigacion Sanitaria de NavarraPamplona, 
      Spain.
FAU - Lanciego, Jose L
AU  - Lanciego JL
AD  - Department of Neurosciences, Center for Applied Medical ResearchPamplona, Spain; 
      Centro de Investigacion Biomedica en Red sobre Enfermedades 
      NeurodegenerativasSpain; Instituto de Investigacion Sanitaria de NavarraPamplona, 
      Spain.
FAU - Gonzalez-Aseguinolaza, Gloria
AU  - Gonzalez-Aseguinolaza G
AD  - Department of Gene Therapy and Regulation of Gene Expression, Center for Applied 
      Medical ResearchPamplona, Spain; Centro de Investigacion Biomedica en Red sobre 
      Enfermedades NeurodegenerativasSpain; Instituto de Investigacion Sanitaria de 
      NavarraPamplona, Spain.
LA  - eng
PT  - Journal Article
DEP - 20170210
PL  - Switzerland
TA  - Front Neuroanat
JT  - Frontiers in neuroanatomy
JID - 101477943
PMC - PMC5301009
OTO - NOTNLM
OT  - AAV
OT  - CNS
OT  - Parkinson's disease
OT  - basal ganglia
OT  - gene therapy
OT  - promoters
EDAT- 2017/02/28 06:00
MHDA- 2017/02/28 06:01
PMCR- 2017/01/01
CRDT- 2017/02/28 06:00
PHST- 2016/10/15 00:00 [received]
PHST- 2017/01/13 00:00 [accepted]
PHST- 2017/02/28 06:00 [entrez]
PHST- 2017/02/28 06:00 [pubmed]
PHST- 2017/02/28 06:01 [medline]
PHST- 2017/01/01 00:00 [pmc-release]
AID - 10.3389/fnana.2017.00002 [doi]
PST - epublish
SO  - Front Neuroanat. 2017 Feb 10;11:2. doi: 10.3389/fnana.2017.00002. eCollection 
      2017.