PMID- 9630611 OWN - NLM STAT- MEDLINE DCOM- 19990329 LR - 20190614 IS - 0006-8993 (Print) IS - 1872-6240 (Electronic) IS - 0006-8993 (Linking) VI - 793 IP - 1-2 DP - 1998 May 18 TI - Recombinant adeno-associated virus vector: use for transgene expression and anterograde tract tracing in the CNS. PG - 169-75 AB - We used a recombinant adeno-associated virus vector (AAV) to deliver a foreign gene, green fluorescent protein (GFP), into mature neurons in adult rat CNS in vivo. Microinjections of AAV as small as 50 nl transduced hundreds of neurons at the injection site. There was virtually no retrograde transport as fewer than one neuron per brain was found distant from the injection site that exhibited GFP immunoreactivity. The gene product, GFP, filled the entire neuronal cytoplasmic compartment; GFP immunoreactivity was robust in cell bodies, axons, and nerve terminals. There was no tissue damage at the injection sites or pathogenicity indicated by changes in astrocytic or microglial markers. There was no inflammatory response as judged by leukocytic invasion. Gene expression in transduced cells was robust and apparently permanent: there was no evidence of phenotypic reversion up to 12 weeks following infection. AAV is an excellent vector for introducing foreign genes into mature CNS neurons. Not only might it be an ideal vehicle for gene therapy, but also the GFP-containing AAV presents a new strategy for tracing long axonal pathways in the CNS, which is difficult with current tracers (PHAL, biotinylated dextrans). CI - Copyright 1998 Elsevier Science B.V. FAU - Chamberlin, N L AU - Chamberlin NL AD - Departments of Neurology, Harvard Medical School, Beth Israel Deaconess Medical Center, Boston, MA 02215, USA. nchamber@bidmc.harvard.edu FAU - Du, B AU - Du B FAU - de Lacalle, S AU - de Lacalle S FAU - Saper, C B AU - Saper CB LA - eng GR - NS22835/NS/NINDS NIH HHS/United States GR - R01 NS033987/NS/NINDS NIH HHS/United States GR - R01 AG019597/AG/NIA NIH HHS/United States GR - R29 AG012401-06/AG/NIA NIH HHS/United States GR - AG21856/AG/NIA NIH HHS/United States GR - NS33987/NS/NINDS NIH HHS/United States GR - R01 AG019597-01/AG/NIA NIH HHS/United States PT - Journal Article PT - Research Support, U.S. Gov't, P.H.S. PL - Netherlands TA - Brain Res JT - Brain research JID - 0045503 RN - 0 (Luminescent Proteins) RN - 147336-22-9 (Green Fluorescent Proteins) SB - IM MH - Animals MH - Axonal Transport/genetics/*physiology MH - Brain/*physiology MH - Cell Line MH - Dependovirus/*genetics MH - *Gene Expression MH - Genes, Reporter MH - Genetic Vectors/*pharmacology MH - Green Fluorescent Proteins MH - Humans MH - Luminescent Proteins/analysis/genetics/metabolism MH - Male MH - Neural Pathways/*physiology MH - Rats MH - Rats, Sprague-Dawley MH - Recombination, Genetic MH - Transgenes/*genetics PMC - PMC4961038 MID - NIHMS23534 EDAT- 1998/06/19 00:00 MHDA- 1998/06/19 00:01 PMCR- 2016/07/26 CRDT- 1998/06/19 00:00 PHST- 1998/06/19 00:00 [pubmed] PHST- 1998/06/19 00:01 [medline] PHST- 1998/06/19 00:00 [entrez] PHST- 2016/07/26 00:00 [pmc-release] AID - S0006-8993(98)00169-3 [pii] AID - 10.1016/s0006-8993(98)00169-3 [doi] PST - ppublish SO - Brain Res. 1998 May 18;793(1-2):169-75. doi: 10.1016/s0006-8993(98)00169-3.