Liver-directed gene therapy

Promises, problems and prospects at the turn of the century

S. S. Ghosh, M. Takahashi, N. R. Thummala, B. Parashar, N. Roy Chowdhury, Jayanta Roy-Chowdhury

Research output: Contribution to journalArticle

46 Citations (Scopus)

Abstract

Although liver-directed gene therapy arrived later than gene therapy directed at bone marrow cells, intrinsic advantages of the liver as a target organ make it likely that gene therapy for liver diseases will be among the first therapeutically relevant applications of this treatment modality at the onset of the 21st century. Vectorology for gene transfer to the liver is advancing rapidly, and it is safe to predict that gene therapy vehicles that will be in clinical use a decade from now, have not yet been developed. None of the currently available modes of gene transfer to the liver is optimal for all types of applications. Nonetheless, the concerted effort of many investigators has provided a wide choice of non-viral and viral vectors for gene transfer to the liver for use in specific situations. Original strategies for liver-directed gene therapy included substitution of missing gene products, overexpression of intrinsic or extrinsic genes and inhibition of expression of specific genes. To the list is now added the possibility of site-specific correction or generation of mutations within specific genes in somatic cells of living adult animals. Thus, despite some initial faux pas, liver-directed gene therapy is poised to make an important impact on health care in the year 2000 and beyond.

Original languageEnglish (US)
Pages (from-to)238-252
Number of pages15
JournalJournal of Hepatology, Supplement
Volume32
Issue number1
StatePublished - 2000

Fingerprint

Genetic Therapy
Liver
Genes
Viral Genes
Bone Marrow Cells
Liver Diseases
Research Personnel
Delivery of Health Care
Gene Expression
Mutation

Keywords

  • Gene therapy
  • Liver diseases
  • Site-directed gene repair
  • Vectors, viral, non-viral

ASJC Scopus subject areas

  • Hepatology
  • Gastroenterology

Cite this

Ghosh, S. S., Takahashi, M., Thummala, N. R., Parashar, B., Roy Chowdhury, N., & Roy-Chowdhury, J. (2000). Liver-directed gene therapy: Promises, problems and prospects at the turn of the century. Journal of Hepatology, Supplement, 32(1), 238-252.

Liver-directed gene therapy : Promises, problems and prospects at the turn of the century. / Ghosh, S. S.; Takahashi, M.; Thummala, N. R.; Parashar, B.; Roy Chowdhury, N.; Roy-Chowdhury, Jayanta.

In: Journal of Hepatology, Supplement, Vol. 32, No. 1, 2000, p. 238-252.

Research output: Contribution to journalArticle

Ghosh, SS, Takahashi, M, Thummala, NR, Parashar, B, Roy Chowdhury, N & Roy-Chowdhury, J 2000, 'Liver-directed gene therapy: Promises, problems and prospects at the turn of the century', Journal of Hepatology, Supplement, vol. 32, no. 1, pp. 238-252.
Ghosh, S. S. ; Takahashi, M. ; Thummala, N. R. ; Parashar, B. ; Roy Chowdhury, N. ; Roy-Chowdhury, Jayanta. / Liver-directed gene therapy : Promises, problems and prospects at the turn of the century. In: Journal of Hepatology, Supplement. 2000 ; Vol. 32, No. 1. pp. 238-252.
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