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Current Pharmaceutical Design

Editor-in-Chief

ISSN (Print): 1381-6128
ISSN (Online): 1873-4286

Perinatal Gene Transfer to the Liver

Author(s): Tristan R. McKay, Ahad A. Rahim, Suzanne M.K. Buckley, Natalie J. Ward, Jerry K.Y.Chan, Steven J. Howe and Simon N. Waddington

Volume 17, Issue 24, 2011

Page: [2528 - 2541] Pages: 14

DOI: 10.2174/138161211797247541

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.

Keywords: Fetal gene therapy, in utero gene therapy, neonatal gene therapy, lysosomal storage disease, mucopolysaccharidosis, hemophilia, glycogen storage disease, liver, viral vector, Coagulation factor X


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