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Publication Details
AFRICAN RESEARCH NEXUS
SHINING A SPOTLIGHT ON AFRICAN RESEARCH
Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system
FASEB Journal, Volume 25, No. 10, Year 2011
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Description
Several diseases of the nervous system are characterized by neurodegeneration and death in childhood. Conventional medicine is ineffective, but fetal or neonatal gene therapy may provide an alternative route to treatment. We evaluated the ability of single-stranded and self-complementary adeno-associated virus pseudotype 2/9 (AAV2/9) to transduce the nervous system and target gene expression to specific neural cell types following intravenous injection into fetal and neonatal mice, using control uninjected age-matched mice. Fetal and neonatal administration produced global delivery to the central (brain, spinal cord, and all layers of the retina) and peripheral (myenteric plexus and innervating nerves) nervous system but with different expression profiles within the brain; fetal and neonatal administration resulted in expression in neurons and protoplasmic astrocytes, respectively. Neither single-stranded nor self-complementary AAV2/9 triggered a microglia-mediated immune response following either administration. In summary, intravenous AAV2/9 targets gene expression to specific neural cell types dependent on developmental stage. This represents a powerful tool for studying nervous system development and disease. Furthermore, it may provide a therapeutic strategy for treatment of early lethal genetic diseases, such as Gaucher disease, and for disabling neuropathies, such as preterm brain injury. © FASEB.
Authors & Co-Authors
Rahim, Ahad A.
United Kingdom, London
University College London
Wong, Andrew M.S.
United Kingdom, London
King's College London
Hoefer, Klemens
United Kingdom, London
King's College London
Buckley, Suzanne M.K.
United Kingdom, London
University College London
Mattar, Citra Nz
Singapore, Singapore City
National University of Singapore
Cheng, Seng H.
United States, Cambridge
Genzyme Corporation
Chan, Jerry Kok Yen
Singapore, Singapore City
National University of Singapore
Singapore, Singapore City
Kk Women's and Children's Hospital
Cooper, Jonathan D.
United Kingdom, London
King's College London
Waddington, Simon N.
United Kingdom, London
University College London
Statistics
Citations: 73
Authors: 9
Affiliations: 5
Identifiers
Doi:
10.1096/fj.11-182311
ISSN:
15306860
Research Areas
Genetics And Genomics
Health System And Policy
Maternal And Child Health
Violence And Injury