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Gene therapy of metabolic diseases
Author(s) -
Fischer Alain,
HaceinBeyAbina Salima,
CavazzanaCalvo Marina
Publication year - 2006
Publication title -
journal of inherited metabolic disease
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.462
H-Index - 102
eISSN - 1573-2665
pISSN - 0141-8955
DOI - 10.1007/s10545-006-0270-7
Subject(s) - genetic enhancement , human genetics , medicine , metabolic disease , clinical trial , bioinformatics , intensive care medicine , computational biology , biology , gene , genetics
Summary 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.