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345 results

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Page 1
Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of β-thalassemia and sickle cell disease.
Negre O, Bartholomae C, Beuzard Y, Cavazzana M, Christiansen L, Courne C, Deichmann A, Denaro M, de Dreuzy E, Finer M, Fronza R, Gillet-Legrand B, Joubert C, Kutner R, Leboulch P, Maouche L, Paulard A, Pierciey FJ, Rothe M, Ryu B, Schmidt M, von Kalle C, Payen E, Veres G. Negre O, et al. Among authors: von kalle c. Curr Gene Ther. 2015;15(1):64-81. doi: 10.2174/1566523214666141127095336. Curr Gene Ther. 2015. PMID: 25429463 Free PMC article.
Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome.
Scaramuzza S, Biasco L, Ripamonti A, Castiello MC, Loperfido M, Draghici E, Hernandez RJ, Benedicenti F, Radrizzani M, Salomoni M, Ranzani M, Bartholomae CC, Vicenzi E, Finocchi A, Bredius R, Bosticardo M, Schmidt M, von Kalle C, Montini E, Biffi A, Roncarolo MG, Naldini L, Villa A, Aiuti A. Scaramuzza S, et al. Among authors: von kalle c. Mol Ther. 2013 Jan;21(1):175-84. doi: 10.1038/mt.2012.23. Epub 2012 Feb 28. Mol Ther. 2013. PMID: 22371846 Free PMC article.
Transgenic expression of human glial cell line-derived neurotrophic factor from integration-deficient lentiviral vectors is neuroprotective in a rodent model of Parkinson's disease.
Lu-Nguyen NB, Broadstock M, Schliesser MG, Bartholomae CC, von Kalle C, Schmidt M, Yáñez-Muñoz RJ. Lu-Nguyen NB, et al. Among authors: von kalle c. Hum Gene Ther. 2014 Jul;25(7):631-41. doi: 10.1089/hum.2014.003. Epub 2014 Apr 15. Hum Gene Ther. 2014. PMID: 24635742 Free PMC article.
Vector integration and tumorigenesis.
von Kalle C, Deichmann A, Schmidt M. von Kalle C, et al. Hum Gene Ther. 2014 Jun;25(6):475-81. doi: 10.1089/hum.2014.2525. Hum Gene Ther. 2014. PMID: 24950086 Free PMC article. No abstract available.
Lentiviral Vector Promoter is Decisive for Aberrant Transcript Formation.
Scholz SJ, Fronza R, Bartholomä CC, Cesana D, Montini E, von Kalle C, Gil-Farina I, Schmidt M. Scholz SJ, et al. Among authors: von kalle c. Hum Gene Ther. 2017 Oct;28(10):875-885. doi: 10.1089/hum.2017.162. Epub 2017 Aug 18. Hum Gene Ther. 2017. PMID: 28825370
Parallel assessment of globin lentiviral transfer in induced pluripotent stem cells and adult hematopoietic stem cells derived from the same transplanted β-thalassemia patient.
Tubsuwan A, Abed S, Deichmann A, Kardel MD, Bartholomä C, Cheung A, Negre O, Kadri Z, Fucharoen S, von Kalle C, Payen E, Chrétien S, Schmidt M, Eaves CJ, Leboulch P, Maouche-Chrétien L. Tubsuwan A, et al. Among authors: von kalle c. Stem Cells. 2013 Sep;31(9):1785-94. doi: 10.1002/stem.1436. Stem Cells. 2013. PMID: 23712774
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: von kalle c. 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.
345 results