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  1. 1.   Telomere length and clonal chromosomal alterations in peripheral blood of patients with severe aplastic anaemia
  2. Strauss, Joshua D; Brown, Derek W; Zhou, Weiyin; Dagnall,Casey; Yuan, Jian-Min; Im, Annie; Savage, Sharon A; Wang, Youjin; Rafati, Maryam; Spellman, Stephen R; Gadalla, Shahinaz M
  3. British Journal of Haematology. 2024, Aug 05;
  1. 2.   Combined haploidentical and cord blood transplantation for refractory severe aplastic anaemia and hypoplastic myelodysplastic syndrome
  2. Childs, Richard W; Tian, Xin; Vo, Phuong; Purev, Enkhtsetseg; Kotecha, Ritesh R; Carlsten, Mattias; Clara, Joseph; Flegel, Willy A; Adams, Sharon D; Khuu, Hanh M; Stroncek, David F; Cook, Lisa; Worthy, Tat'yana; Geller, Nancy L; Wells, Brian; Wilder,Jennifer; Reger, Robert; Aue, Georg
  3. British Journal of Haematology. 2021, May 15; 193(5): 951-960.
  1. 3.   Pre-transplant short telomeres are associated with high mortality risk after unrelated donor haematopoietic cell transplant for severe aplastic anaemia
  2. Wang, Youjin; McReynolds, Lisa J; Dagnall,Casey; Katki, Hormuzd A; Spellman, Stephen R; Wang, Tao; Hicks,Belynda; Freedman, Neal D; Jones,Kristine; Lee, Stephanie J; Savage, Sharon A; Gadalla, Shahinaz M
  3. British journal of haematology. 2019, Aug 19;
  1. 4.   Identification of an Intronic Regulatory Element Necessary for Tissue-Specific Expression of Foxn1 in Thymic Epithelial Cells
  2. Larsen, Brian M; Cowan, Jennifer E; Wang, Yueqiang; Tanaka, Yu; Zhao, Yongge; Voisin, Benjamin; Constantinides, Michael G; Nagao, Keisuke; Belkaid, Yasmine; Awasthi,Roackie; Takahama, Yousuke; Bhandoola, Avinash
  3. Journal of immunology (Baltimore, Md. : 1950). 2019, AUG 1; 203(3): 686-695.
  1. 5.   CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease
  2. De Ravin, Suk See; Li, Linhong; Wu, Xiaolin; Choi, Uimook; Allen, Cornell; Koontz, Sherry; Lee, Janet; Theobald-Whiting, Narda; Chu, Jessica; Garofalo, Mary; Sweeney, Colin; Kardava, Lela; Moir, Susan; Viley, Angelia; Natarajan, Pachai; Su, Ling; Kuhns, Douglas; Zarember, Kol A.; Peshwa, Madhusudan V.; Malech, Harry L.
  3. SCIENCE TRANSLATIONAL MEDICINE. 2017, Jan 11; 9(372): eaah3480.
  1. 6.   Oncogenic IL7R gain-of-function mutations in childhood T-cell acute lymphoblastic leukemia
  2. Zenatti, P. P.; Ribeiro, D.; Li, W. Q.; Zuurbier, L.; Silva, M. C.; Paganin, M.; Tritapoe, J.; Hixon, J. A.; Silveira, A. B.; Cardoso, B. A.; Sarmento, L. M.; Correia, N.; Toribio, M. L.; Kobarg, J.; Horstmann, M.; Pieters, R.; Brandalise, S. R.; Ferrando, A. A.; Meijerink, J. P.; Durum, S. K.; Yunes, J. A.; Barata, J. T.
  3. Nature Genetics. 2011, Oct; 43(10): 932-U31.
  1. 7.   Association of HLA alleles with Plasmodium falciparum severity in Malian children
  2. Lyke, K. E.; Fernandez-Vina, M. A.; Cao, K.; Hollenbach, J.; Coulibaly, D.; Kone, A. K.; Guindo, A.; Burdett, L. A.; Hartzman, R. J.; Wahl, A. R.; Hildebrand, W. H.; Doumbo, O. K.; Plowe, C. V.; Sztein, M. B.
  3. Tissue Antigens. 2011, Jun; 77(6): 562-571.
  1. 8.   Suppression of the C/EBP family of transcription factors in adipose tissue causes lipodystrophy
  2. Chatterjee, R.; Bhattacharya, P.; Gavrilova, O.; Glass, K.; Moitra, J.; Myakishev, M.; Pack, S.; Jou, W.; Feigenbaum, L.; Eckhaus, M.; Vinson, C.
  3. Journal of Molecular Endocrinology. 2011, Jun; 46(3): 175-192.
  1. 9.   Role of IKK alpha in skin squamous cell carcinomas
  2. Park, E. M.; Liu, B. G.; Xia, X. J.; Zhu, F.; Jami, W. B.; Hu, Y. L.
  3. Future Oncology. 2011, Jan; 7(1): 123-134.
  1. 10.   Implications for Gene Therapy-Limiting Expression of IL-2R gamma(c) Delineate Differences in Signaling Thresholds Required for Lymphocyte Development and Maintenance
  2. Orr, S. J.; Roessler, S.; Quigley, L.; Chan, T.; Ford, J. W.; O'Connor, G. M.; McVicar, D. W.
  3. Journal of Immunology. 2010, Aug; 185(3): 1393-1403.
  1. 11.   Hypomorphic Rag mutations can cause destructive midline granulomatous disease
  2. De Ravin, S. S.; Cowen, E. W.; Zarember, K. A.; Whiting-Theobald, N. L.; Kuhns, D. B.; Sandler, N. G.; Douek, D. C.; Pittaluga, S.; Poliani, P. L.; Lee, Y. N.; Notarangelo, L. D.; Wang, L.; Alt, F. W.; Kang, E. M.; Milner, J. D.; Niemela, J. E.; Fontana-Penn, M.; Sinal, S. H.; Malech, H. L.
  3. Blood. 2010, Aug; 116(8): 1263-1271.
  1. 12.   Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils
  2. Kang, E. M.; Choi, U.; Theobald, N.; Linton, G.; Priel, D. A. L.; Kuhns, D.; Malech, H. L.
  3. Blood. 2010, Jan; 115(4): 783-791.
  1. 13.   Self-inactivating Retroviral Vector-mediated Gene Transfer Induces Oncogene Activation and Immortalization of Primary Murine Bone Marrow Cells
  2. Bosticardo, M.; Ghosh, A.; Du, Y.; Jenkins, N. A.; Copeland, N. G.; C, otti
  3. Molecular Therapy. 2009 17(11): 1910-1918.
  1. 14.   Sustained high-level polyclonal hematopoietic marking and transgene expression 4 years after autologous transplantation of rhesus macaques with SIV lentiviral vector-transduced CD34(+) cells
  2. Kim, Y. J.; Kim, Y. S.; Larochelle, A.; Renaud, G.; Wolfsberg, T. G.; Adler, R.; Donahue, R. E.; Hematti, P.; Hong, B. K.; Roayaei, J.; Akagi, K.; Riberdy, J. M.; Nienhuis, A. W.; Dunbar, C. E.; Persons, D. A.
  3. Blood. 2009 113(22): 5434-5443.
  1. 15.   IL-10 helps control pathogen load during high-level bacteremia
  2. Londono, D.; Marques, A.; Hornung, R. L.; Cadavid, D.
  3. Journal of Immunology. 2008 181(3): 2076-2083.
  1. 16.   An inducible and reversible mouse genetic rescue system
  2. Zeng, H. K.; Horie, K.; Madisen, L.; Pavlova, M. N.; Gragerova, G.; Rohde, A. D.; Schimpf, B. A.; Liang, Y. Q.; Ojala, E.; Kramer, F.; Roth, P.; Slobodskaya, O.; Dolka, I.; Southon, E. A.; Tessarollo, L.; Bornfeldt, K. E.; Gragerov, A.; Pavlakis, G.
  3. Plos Genetics. 2008 4(5): 1-13.
  1. 17.   Recombinant human activated protein C for the postexposure treatment of Ebola hemorrhagic fever
  2. Hensley, L. E.; Stevens, E. L.; Yan, S. B.; Geisbert, J. B.; Macias, W. L.; Larsen, T.; Daddario-DiCaprio, K. M.; Cassell, G. H.; Jahrling, P. B.; Geisbert, T. W.
  3. Journal of Infectious Diseases. 2007, Nov; 196: S390-S399.
  1. 18.   Natural products as sources of new drugs over the last 25 years
  2. Newman, D. J.; Cragg, G. M.
  3. Journal of Natural Products. 2007, Mar; 70(3): 461-477.
  1. 19.   Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1
  2. Ott, M. G.; Schmidt, M.; Schwarzwaelder, K.; Stein, S.; Siler, U.; Koehl, U.; Glimm, H.; Kuhlcke, K.; Schilz, A.; Kunkel, H.; Naundorf, S.; Brinkmann, A.; Deichmann, A.; Fischer, M.; Ball, C.; Pilz, I.; Dunbar, C.; Du, Y.; Jenkins, N. A.; Copeland, N. G.; Luthi, U.; Hassan, M.; Thrasher, A. J.; Hoelzer, D.; von Kalle, C.; Seger, R.; Grez, M.
  3. Nature Medicine. 2006, Apr; 12(4): 401-409.
  1. 20.   Stat5a/b are essential for normal lymphoid development and differentiation
  2. Yao, Z. J.; Cui, Y. Z.; Watford, W. T.; Bream, J. H.; Yamaoka, K.; Hissong, B. D.; Li, D.; Durum, S. K.; Jiang, Q. O.; Bhandoola, A.; Hennighausen, L.; O'Shea, J. J.
  3. Proceedings of the National Academy of Sciences of the United States of America. 2006, JAN 24; 103(4): 1000-1005.
  1. 21.   Retroviral transduction of IL-7R alpha into IL-7R alpha(-/-) bone marrow progenitors: correction of lymphoid deficiency and induction of neutrophilia
  2. Jiang, Q.; Li, W. Q.; Aiello, F. B.; Klarmann, K.; Keller, J. R.; Durum, S. K.
  3. Gene Therapy. 2005, DEC; 12(24): 1761-1768.
  1. 22.   Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells
  2. Calmels, B.; Ferguson, C.; Laukkanen, M. O.; Adler, R.; Faulhaber, M.; Kim, H. J.; Sellers, S.; Hematti, P.; Schmidt, M.; von Kalle, C.; Akagi, K.; Donahue, R. E.; Dunbar, C. E.
  3. Blood. 2005, OCT 1; 106(7): 2530-2533.
  1. 23.   Integration target site selection for retroviruses and transposable elements
  2. Wu, X.; Burgess, S. M.
  3. Cellular and Molecular Life Sciences. 2004, OCT; 61(19-20): 2588-2596.
  1. 24.   RBSP3 (HYA22) is a tumor suppressor gene implicated in major epithelial malignancies
  2. Kashuba, V. I.; Li, J. F.; Wang, F. L.; Senchenko, V. N.; Protopopov, A.; Malyukova, A.; Kutsenko, A. S.; Kadyrova, E.; Zabarovska, V. I.; Muravenko, O. V.; Zelenin, A. V.; Kisselev, L. L.; Kuzmin, I.; Minna, J. D.; Winberg, G.; Ernberg, I.; Braga, E.; Lerman, M. I.; Klein, G.; Zabarovsky, E. R.
  3. Proceedings of the National Academy of Sciences of the United States of America. 2004 101(14): 4906-4911.
  1. 25.   Gene therapy insertional mutagenesis insights
  2. Dave, U. P.; Jenkins, N. A.; Copeland, N. G.
  3. Science. 2004 303(5656): 333-333.
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