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Gene Therapy for Metabolic Diseases of the Liver.

Authors :
Promrat, K.
Wu, G.Y.
Wu, C.H.
Source :
BioDrugs. 2000, Vol. 13 Issue 3, p177-188. 12p.
Publication Year :
2000

Abstract

Significant advances have been made in the field of liver-directed gene therapy. Many diseases are potential targets for gene therapy, including diseases that have exclusive liver involvement and those with systemic manifestations as a result of defective protein synthesis from the liver. Examples are Crigler-Najjar syndrome type 1, α-antitrypsin deficiency and haemophilia A and B. Strategies for gene delivery include the use of viral and nonviral vectors. In addition to previously developed viral vectors, such as retroviruses, adenoviruses and adeno-associated viruses, new viral vectors such as lentiviruses are being investigated extensively. Nonviral vectors for gene delivery include liposomes and receptor-mediated gene therapy. A strategy to correct gene defects has been developed using chimaeric RNA/DNA oligonucleotides, and methods to inhibit aberrant or deleterious gene expression using ribozymes, antisense oligonucleotides and dominant-negative gene products are being developed. However, more research focusing on more efficient gene expression and safety will be required before gene therapy can be routinely applicable. [ABSTRACT FROM AUTHOR]

Details

Language :
English
ISSN :
11738804
Volume :
13
Issue :
3
Database :
Academic Search Index
Journal :
BioDrugs
Publication Type :
Academic Journal
Accession number :
9522711
Full Text :
https://doi.org/10.2165/00063030-200013030-00003