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Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease.
Brendel C, Rothe M, Santilli G, Charrier S, Stein S, Kunkel H, Abriss D, Müller-Kuller U, Gaspar B, Modlich U, Galy A, Schambach A, Thrasher AJ, Grez M. Brendel C, et al. Among authors: galy a. Hum Gene Ther Clin Dev. 2018 Jun;29(2):69-79. doi: 10.1089/humc.2017.245. Epub 2018 Apr 17. Hum Gene Ther Clin Dev. 2018. PMID: 29664709
Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models.
Marangoni F, Bosticardo M, Charrier S, Draghici E, Locci M, Scaramuzza S, Panaroni C, Ponzoni M, Sanvito F, Doglioni C, Liabeuf M, Gjata B, Montus M, Siminovitch K, Aiuti A, Naldini L, Dupré L, Roncarolo MG, Galy A, Villa A. Marangoni F, et al. Among authors: galy a. Mol Ther. 2009 Jun;17(6):1073-82. doi: 10.1038/mt.2009.31. Epub 2009 Mar 3. Mol Ther. 2009. PMID: 19259069 Free PMC article.
Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting.
Tolar J, Adair JE, Antoniou M, Bartholomae CC, Becker PS, Blazar BR, Bueren J, Carroll T, Cavazzana-Calvo M, Clapp DW, Dalgleish R, Galy A, Gaspar HB, Hanenberg H, Von Kalle C, Kiem HP, Lindeman D, Naldini L, Navarro S, Renella R, Rio P, Sevilla J, Schmidt M, Verhoeyen E, Wagner JE, Williams DA, Thrasher AJ. Tolar J, et al. Among authors: galy a. Mol Ther. 2011 Jul;19(7):1193-8. doi: 10.1038/mt.2011.78. Epub 2011 May 3. Mol Ther. 2011. PMID: 21540837 Free PMC article. Review.
Gene therapy for the Wiskott-Aldrich syndrome.
Galy A, Thrasher AJ. Galy A, et al. Curr Opin Allergy Clin Immunol. 2011 Dec;11(6):545-50. doi: 10.1097/ACI.0b013e32834c230c. Curr Opin Allergy Clin Immunol. 2011. PMID: 21971332 Review.
210 results