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Preselection of retrovirally transduced bone marrow avoids subsequent stem cell gene silencing and age-dependent extinction of expression of human beta-globin in engrafted mice.
Kalberer CP, Pawliuk R, Imren S, Bachelot T, Takekoshi KJ, Fabry M, Eaves CJ, London IM, Humphries RK, Leboulch P. Kalberer CP, et al. Among authors: leboulch p. Proc Natl Acad Sci U S A. 2000 May 9;97(10):5411-5. doi: 10.1073/pnas.100082597. Proc Natl Acad Sci U S A. 2000. PMID: 10792053 Free PMC article.
Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia.
Thompson AA, Walters MC, Kwiatkowski J, Rasko JEJ, Ribeil JA, Hongeng S, Magrin E, Schiller GJ, Payen E, Semeraro M, Moshous D, Lefrere F, Puy H, Bourget P, Magnani A, Caccavelli L, Diana JS, Suarez F, Monpoux F, Brousse V, Poirot C, Brouzes C, Meritet JF, Pondarré C, Beuzard Y, Chrétien S, Lefebvre T, Teachey DT, Anurathapan U, Ho PJ, von Kalle C, Kletzel M, Vichinsky E, Soni S, Veres G, Negre O, Ross RW, Davidson D, Petrusich A, Sandler L, Asmal M, Hermine O, De Montalembert M, Hacein-Bey-Abina S, Blanche S, Leboulch P, Cavazzana M. Thompson AA, et al. Among authors: leboulch p. N Engl J Med. 2018 Apr 19;378(16):1479-1493. doi: 10.1056/NEJMoa1705342. N Engl J Med. 2018. PMID: 29669226 Free article. Clinical Trial.
In vivo gene delivery to synovium by lentiviral vectors.
Gouze E, Pawliuk R, Pilapil C, Gouze JN, Fleet C, Palmer GD, Evans CH, Leboulch P, Ghivizzani SC. Gouze E, et al. Among authors: leboulch p. Mol Ther. 2002 Apr;5(4):397-404. doi: 10.1006/mthe.2002.0562. Mol Ther. 2002. PMID: 11945066 Free article.
121 results