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Gene Therapy for Metabolic Diseases of the Liver.
- 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]
- Subjects :
- *LIVER disease treatment
*GENE therapy
Subjects
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