Article
In vivo correction of murine tyrosinemia type I by DNA-mediated transposition.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Dec 2002
Montini Eugenio, Held Patrice K, Noll Meenakshi, Morcinek Nicolas, Al-Dhalimy Muhsen, Finegold Milton, Yant Stephen R, Kay Mark A, Grompe Markus
Abstract excerpt
Gene therapy applications of naked DNA constructs for genetic disorders have been limited because of lack of permanent transgene expression. This limitation, however, can be overcome by the Sleeping Beauty (SB) transposable element, which can achieve permanent transgene expression through genomic integration from plasmid DNA. To date, only one example of an in vivo gene therapy application of this system has been...
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