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Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity.
Braun CJ, Boztug K, Paruzynski A, Witzel M, Schwarzer A, Rothe M, Modlich U, Beier R, Göhring G, Steinemann D, Fronza R, Ball CR, Haemmerle R, Naundorf S, Kühlcke K, Rose M, Fraser C, Mathias L, Ferrari R, Abboud MR, Al-Herz W, Kondratenko I, Maródi L, Glimm H, Schlegelberger B, Schambach A, Albert MH, Schmidt M, von Kalle C, Klein C. Braun CJ, et al. Among authors: paruzynski a. Sci Transl Med. 2014 Mar 12;6(227):227ra33. doi: 10.1126/scitranslmed.3007280. Sci Transl Med. 2014. PMID: 24622513 Clinical Trial.
Mapping Active Gene-Regulatory Regions in Human Repopulating Long-Term HSCs.
Wünsche P, Eckert ESP, Holland-Letz T, Paruzynski A, Hotz-Wagenblatt A, Fronza R, Rath T, Gil-Farina I, Schmidt M, von Kalle C, Klein C, Ball CR, Herbst F, Glimm H. Wünsche P, et al. Among authors: paruzynski a. Cell Stem Cell. 2018 Jul 5;23(1):132-146.e9. doi: 10.1016/j.stem.2018.06.003. Cell Stem Cell. 2018. PMID: 29979988 Free article.
The balance between the intronic miR-342 and its host gene Evl determines hematopoietic cell fate decision.
Herbst F, Lang TJL, Eckert ESP, Wünsche P, Wurm AA, Kindinger T, Laaber K, Hemmati S, Hotz-Wagenblatt A, Zavidij O, Paruzynski A, Lu J, von Kalle C, Zenz T, Klein C, Schmidt M, Ball CR, Glimm H. Herbst F, et al. Among authors: paruzynski a. Leukemia. 2021 Oct;35(10):2948-2963. doi: 10.1038/s41375-021-01267-5. Epub 2021 May 21. Leukemia. 2021. PMID: 34021250 Free PMC article.
Successful Combination of Sequential Gene Therapy and Rescue Allo-HSCT in Two Children with X-CGD - Importance of Timing.
Siler U, Paruzynski A, Holtgreve-Grez H, Kuzmenko E, Koehl U, Renner ED, Alhan C, de Loosdrecht AA, Schwäble J, Pfluger T, Tchinda J, Schmugge M, Jauch A, Naundorf S, Kühlcke K, Notheis G, Güngor T, Kalle CV, Schmidt M, Grez M, Seger R, Reichenbach J. Siler U, et al. Among authors: paruzynski a. Curr Gene Ther. 2015;15(4):416-27. doi: 10.2174/1566523215666150515145255. Curr Gene Ther. 2015. PMID: 25981636 Clinical Trial.
Comprehensive genomic access to vector integration in clinical gene therapy.
Gabriel R, Eckenberg R, Paruzynski A, Bartholomae CC, Nowrouzi A, Arens A, Howe SJ, Recchia A, Cattoglio C, Wang W, Faber K, Schwarzwaelder K, Kirsten R, Deichmann A, Ball CR, Balaggan KS, Yáñez-Muñoz RJ, Ali RR, Gaspar HB, Biasco L, Aiuti A, Cesana D, Montini E, Naldini L, Cohen-Haguenauer O, Mavilio F, Thrasher AJ, Glimm H, von Kalle C, Saurin W, Schmidt M. Gabriel R, et al. Among authors: paruzynski a. Nat Med. 2009 Dec;15(12):1431-6. doi: 10.1038/nm.2057. Epub 2009 Nov 22. Nat Med. 2009. PMID: 19966782
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