Effective gene therapy of mice with congenital erythropoietic porphyria is facilitated by a survival advantage of corrected erythroid cells. [electronic resource]
- American journal of human genetics Jan 2008
- 113-24 p. digital
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
1537-6605
10.1016/j.ajhg.2007.09.007 doi
Animals Cell Survival Disease Models, Animal Erythrocytes Female Genetic Therapy Genetic Vectors Hematopoietic Stem Cells Lentivirus Male Mice Mice, Inbred BALB C Porphyria, Erythropoietic--genetics Uroporphyrinogen III Synthetase--genetics