Perinatal gene transfer to the liver.

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.

journal_name

Curr Pharm Des

authors

McKay TR,Rahim AA,Buckley SM,Ward NJ,Chan JK,Howe SJ,Waddington SN

doi

10.2174/138161211797247541

subject

Has Abstract

pub_date

2011-01-01 00:00:00

pages

2528-41

issue

24

eissn

1381-6128

issn

1873-4286

pii

BSP/CPD/E-Pub/000546

journal_volume

17

pub_type

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