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Title: Perinatal gene transfer to the liver
Authors: Mckay, T.R.
Rahim, A.A.
Buckley, S.M.K.
Ward, N.J.
Chan, J.K.Y. 
Howe, S.J.
Waddington, S.N.
Keywords: Fetal gene therapy
Glycogen storage disease
In utero gene therapy
Lysosomal storage disease
Neonatal gene therapy
Viral vector
Issue Date: Aug-2011
Abstract: The liver acts as a host to many functions hence raising the possibility that any one may be compromised by a single gene defect. Inherited or de novo mutations in these genes may result in relatively mild diseases or be so devastating that death within the first weeks or months of life is inevitable. Some diseases can be managed using conventional medicines whereas others are, as yet, untreatable. In this review we consider the application of early intervention gene therapy in neonatal and fetal preclinical studies. We appraise the tools of this technology, including lentivirus, adenovirus and adeno-associated virus (AAV)-based vectors. We highlight the application of these for a range of diseases including hemophilia, urea cycle disorders such as ornithine transcarbamylase deficiency, organic acidemias, lysosomal storage diseases including mucopolysaccharidoses, glycogen storage diseases and bile metabolism. We conclude by assessing the advantages and disadvantages associated with fetal and neonatal liver gene transfer. © 2011 Bentham Science Publishers.
Source Title: Current Pharmaceutical Design
ISSN: 13816128
DOI: 10.2174/138161211797247541
Appears in Collections:Staff Publications

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