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Perinatal Gene Transfer to the Liver

McKay, TR; Rahim, AA; Buckley, SM; Ward, NJ; Chan, JK; Howe, SJ; Waddington, SN (2011) Perinatal Gene Transfer to the Liver. CURRENT PHARMACEUTICAL DESIGN, 17 (24). 2528 - 2541 (14). ISSN 1381-6128
SGUL Authors: McKay, Tristan Rowntree

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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.

Item Type: Article
Additional Information: PubMed ID: 21774770
Keywords: Adenoviridae, Animals, Dependovirus, Female, Fetal Diseases, Fetal Therapies, Gene Transfer Techniques, Genetic Therapy, Genetic Vectors, Humans, Infant, Newborn, Lentivirus, Liver, Pregnancy, Science & Technology, Life Sciences & Biomedicine, Pharmacology & Pharmacy, Fetal gene therapy, in utero gene therapy, neonatal gene therapy, lysosomal storage disease, mucopolysaccharidosis, hemophilia, glycogen storage disease, liver, viral vector, ORNITHINE TRANSCARBAMYLASE DEFICIENCY, LONG-TERM CORRECTION, DISEASE TYPE IA, MUCOPOLYSACCHARIDOSIS TYPE-VII, ADENOASSOCIATED VIRAL VECTORS, ALPHA-L-IDURONIDASE, PSEUDOTYPED RETROVIRAL VECTORS, DEPENDENT ADENOVIRAL VECTORS, LEBERS CONGENITAL AMAUROSIS, GLYCOGEN-STORAGE DISEASES
SGUL Research Institute / Research Centre: Academic Structure > Molecular and Clinical Sciences Research Institute (MCS)
Academic Structure > Molecular and Clinical Sciences Research Institute (MCS) > Cell Sciences (INCCCS)
ISSN: 1381-6128
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1 August 2011Published
Web of Science ID: WOS:000299627300004
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