Effective Gene Therapy of Mice with Congenital Erythropoietic Porphyria Is Facilitated by a Survival Advantage of Corrected Erythroid Cells

2008/01/01 by Elodie Robert-Richard, Élodie Richard, François Moreau-Gaudry +11 · 2 citations
Biochemistry, Genetics and Molecular Biology · Medicine · #Biology #CRISPR and Genetic Engineering #Cancer research #Gene #Genetic enhancement #Genetics #Haematopoiesis #Hematopoietic stem cell transplantation #Immunology #Internal medicine #Medicine #Phenotype #Porphyrin Metabolism and Disorders #Stem cell #Transplantation #Virus-based gene therapy research

paper · pdf · doi:10.1016/j.ajhg.2007.09.007

crossref issued 2008/01/01 · crossref published 2008/01/01 · crossref published-print 2008/01/01 · openalex publication_date 2008/01/01 · crossref created 2008/01/28 · crossref deposited 2020/05/01 · openalex created_date 2025/10/10 · crossref indexed 2026/07/15 · openalex updated_date 2026/07/28

Citations

• Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1
• Point mutations in the uroporphyrinogen III synthase gene in congenital erythropoietic porphyria (Gunther's disease)

Cited by

• A management algorithm for congenital erythropoietic porphyria derived from a study of 29 cases
• Gunther disease [wikipedia]