Gene therapy of metabolic diseases

Alain Fischer; Salima Hacein-Bey-Abina; Marina Cavazzana-Calvo

doi:10.1007/s10545-006-0270-7

Gene therapy of metabolic diseases

J Inherit Metab Dis. 2006 Apr-Jun;29(2-3):409-12. doi: 10.1007/s10545-006-0270-7.

Authors

Alain Fischer¹, Salima Hacein-Bey-Abina, Marina Cavazzana-Calvo

Affiliation

¹ Inserm, U768, Paris, France. [email protected]

PMID: 16763910
DOI: 10.1007/s10545-006-0270-7

Abstract

This review surveys the potential application of gene therapy to metabolic diseases. Proof of principle has been achieved in the treatment of two inherited immunodeficiency conditions. A significant safety issue has also been observed. Several strategies are being experimentally tested for a number of metabolic diseases (more than 20) and may provide a rationale for the future development of clinical trials.

Publication types

Review

MeSH terms

Gene Targeting
Gene Transfer Techniques
Genetic Therapy* / adverse effects
Humans
Metabolism, Inborn Errors / genetics
Metabolism, Inborn Errors / therapy*
Severe Combined Immunodeficiency / genetics
Severe Combined Immunodeficiency / therapy*
Treatment Outcome