Modeling of congenital erythropoietic porphyria by RNA interference: a new tool for preclinical gene therapy evaluation.

Robert-Richard, Elodie

Modeling of congenital erythropoietic porphyria by RNA interference: a new tool for preclinical gene therapy evaluation. [electronic resource] - The journal of gene medicine Aug 2010 - 637-46 p. digital

Publication Type: Evaluation Study; Journal Article; Research Support, Non-U.S. Gov't

1521-2254

10.1002/jgm.1478 doi


Animals
Disease Models, Animal
Genetic Therapy--methods
Hematopoietic Stem Cells--metabolism
Humans
K562 Cells
Lentivirus--genetics
Mice
Porphyria, Erythropoietic--enzymology
RNA Interference
Uroporphyrinogen III Synthetase--genetics