PMID- 15960599
OWN - NLM
STAT- MEDLINE
DCOM- 20050927
LR  - 20071114
IS  - 1043-0342 (Print)
IS  - 1043-0342 (Linking)
VI  - 16
IP  - 6
DP  - 2005 Jun
TI  - Patterns of gene expression from in utero delivery of adenoviral-associated 
      vector serotype 1.
PG  - 678-84
AB  - Adenoviral-associated viral vectors (AAV) have shown significant promise for 
      efficient gene delivery to multiple tissues. Studies of different serotypes of 
      AAV revealed different expression patterns provided by gene delivery in postnatal 
      mice. Previous in utero gene delivery studies of AAV serotype 2 (AAV2) 
      demonstrated efficient gene expression in certain fetal tissues depending on 
      route of administration. We studied the pattern of gene expression from AAV 
      serotype 1 (AAV1) using intramuscular, intraperitoneal, and intravascular routes 
      of administration in embryonic day 16 C57BL/6 mice. Limb skeletal muscle 
      transduction was only achieved with AAV1 by intramuscular administration. The 
      levels of gene expression were 20-fold higher than a comparable administration of 
      AAV2. Diaphragm muscle transduction by AAV1 was achieved at the highest level by 
      intraperitoneal administration, and to a lesser degree by intravascular 
      administration. All delivery routes resulted in transgene expression in the lung. 
      Our results indicate that AAV1 can offer higher transgene expression in fetal 
      skeletal muscle than AAV2 with intramuscular administration. The transgene 
      expression pattern in different tissues, which depends on vector serotype and 
      route of administration, will need to be considered in planning therapeutic 
      studies for specific disorders.
FAU - Bilbao, Roberto
AU  - Bilbao R
AD  - Department of Neurology, University of Pittsburgh, Pittsburgh, PA 15213, USA.
FAU - Reay, Daniel P
AU  - Reay DP
FAU - Li, Juan
AU  - Li J
FAU - Xiao, Xiao
AU  - Xiao X
FAU - Clemens, Paula R
AU  - Clemens PR
LA  - eng
GR  - P01 AR45925/AR/NIAMS NIH HHS/United States
GR  - R01 AR050565/AR/NIAMS NIH HHS/United States
GR  - U01 NS46546/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  - Research Support, U.S. Gov't, P.H.S.
PL  - United States
TA  - Hum Gene Ther
JT  - Human gene therapy
JID - 9008950
RN  - EC 3.2.1.23 (beta-Galactosidase)
SB  - IM
MH  - Animals
MH  - Dependovirus/*genetics
MH  - Female
MH  - Gene Expression
MH  - *Gene Transfer Techniques
MH  - Genetic Vectors/*administration & dosage/genetics
MH  - Injections, Intramuscular
MH  - Injections, Intraperitoneal
MH  - Mice
MH  - Mice, Inbred C57BL
MH  - Muscle, Skeletal/*embryology/physiology
MH  - Pregnancy
MH  - Tissue Distribution
MH  - Transgenes
MH  - beta-Galactosidase/genetics/pharmacokinetics
EDAT- 2005/06/18 09:00
MHDA- 2005/09/28 09:00
CRDT- 2005/06/18 09:00
PHST- 2005/06/18 09:00 [pubmed]
PHST- 2005/09/28 09:00 [medline]
PHST- 2005/06/18 09:00 [entrez]
AID - 10.1089/hum.2005.16.678 [doi]
PST - ppublish
SO  - Hum Gene Ther. 2005 Jun;16(6):678-84. doi: 10.1089/hum.2005.16.678.