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Items: 21

1.

Somatic HLA Mutations Expose the Role of Class I-Mediated Autoimmunity in Aplastic Anemia and its Clonal Complications.

Babushok DV, Duke JL, Xie HM, Stanley N, Atienza J, Perdigones N, Nicholas P, Ferriola D, Li Y, Huang H, Ye W, Morrissette JJD, Kearns J, Porter DL, Podsakoff GM, Eisenlohr LC, Biegel JA, Chou ST, Monos DS, Bessler M, Olson TS.

Blood Adv. 2017 Oct 10;1(22):1900-1910.

2.

AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes.

Hui DJ, Edmonson SC, Podsakoff GM, Pien GC, Ivanciu L, Camire RM, Ertl H, Mingozzi F, High KA, Basner-Tschakarjan E.

Mol Ther Methods Clin Dev. 2015 Sep 30;2:15029. doi: 10.1038/mtm.2015.29. eCollection 2015.

3.

Impaired Telomere Maintenance and Decreased Canonical WNT Signaling but Normal Ribosome Biogenesis in Induced Pluripotent Stem Cells from X-Linked Dyskeratosis Congenita Patients.

Gu BW, Apicella M, Mills J, Fan JM, Reeves DA, French D, Podsakoff GM, Bessler M, Mason PJ.

PLoS One. 2015 May 18;10(5):e0127414. doi: 10.1371/journal.pone.0127414. eCollection 2015.

4.

Emergence of clonal hematopoiesis in the majority of patients with acquired aplastic anemia.

Babushok DV, Perdigones N, Perin JC, Olson TS, Ye W, Roth JJ, Lind C, Cattier C, Li Y, Hartung H, Paessler ME, Frank DM, Xie HM, Cross S, Cockroft JD, Podsakoff GM, Monos D, Biegel JA, Mason PJ, Bessler M.

Cancer Genet. 2015 Apr;208(4):115-28. doi: 10.1016/j.cancergen.2015.01.007. Epub 2015 Feb 2.

5.

Single nucleotide polymorphism array analysis of bone marrow failure patients reveals characteristic patterns of genetic changes.

Babushok DV, Xie HM, Roth JJ, Perdigones N, Olson TS, Cockroft JD, Gai X, Perin JC, Li Y, Paessler ME, Hakonarson H, Podsakoff GM, Mason PJ, Biegel JA, Bessler M.

Br J Haematol. 2014 Jan;164(1):73-82. doi: 10.1111/bjh.12603. Epub 2013 Oct 14.

6.

Overcoming preexisting humoral immunity to AAV using capsid decoys.

Mingozzi F, Anguela XM, Pavani G, Chen Y, Davidson RJ, Hui DJ, Yazicioglu M, Elkouby L, Hinderer CJ, Faella A, Howard C, Tai A, Podsakoff GM, Zhou S, Basner-Tschakarjan E, Wright JF, High KA.

Sci Transl Med. 2013 Jul 17;5(194):194ra92. doi: 10.1126/scitranslmed.3005795.

7.

Ribosomal and hematopoietic defects in induced pluripotent stem cells derived from Diamond Blackfan anemia patients.

Garçon L, Ge J, Manjunath SH, Mills JA, Apicella M, Parikh S, Sullivan LM, Podsakoff GM, Gadue P, French DL, Mason PJ, Bessler M, Weiss MJ.

Blood. 2013 Aug 8;122(6):912-21. doi: 10.1182/blood-2013-01-478321. Epub 2013 Jun 6.

8.

Effects of immunosuppression on circulating adeno-associated virus capsid-specific T cells in humans.

Parzych EM, Li H, Yin X, Liu Q, Wu TL, Podsakoff GM, High KA, Levine MH, Ertl HC.

Hum Gene Ther. 2013 Apr;24(4):431-42. doi: 10.1089/hum.2012.246.

9.

Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans.

Candotti F, Shaw KL, Muul L, Carbonaro D, Sokolic R, Choi C, Schurman SH, Garabedian E, Kesserwan C, Jagadeesh GJ, Fu PY, Gschweng E, Cooper A, Tisdale JF, Weinberg KI, Crooks GM, Kapoor N, Shah A, Abdel-Azim H, Yu XJ, Smogorzewska M, Wayne AS, Rosenblatt HM, Davis CM, Hanson C, Rishi RG, Wang X, Gjertson D, Yang OO, Balamurugan A, Bauer G, Ireland JA, Engel BC, Podsakoff GM, Hershfield MS, Blaese RM, Parkman R, Kohn DB.

Blood. 2012 Nov 1;120(18):3635-46. doi: 10.1182/blood-2012-02-400937. Epub 2012 Sep 11.

10.

Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer.

Buchlis G, Podsakoff GM, Radu A, Hawk SM, Flake AW, Mingozzi F, High KA.

Blood. 2012 Mar 29;119(13):3038-41. doi: 10.1182/blood-2011-09-382317. Epub 2012 Jan 23. Erratum in: Blood. 2014 Mar 13;123(11):1768.

11.

Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report.

Engel BC, Podsakoff GM, Ireland JL, Smogorzewska EM, Carbonaro DA, Wilson K, Shah A, Kapoor N, Sweeney M, Borchert M, Crooks GM, Weinberg KI, Parkman R, Rosenblatt HM, Wu SQ, Hershfield MS, Candotti F, Kohn DB.

Blood. 2007 Jan 15;109(2):503-6. Epub 2006 Sep 14.

12.

Perspectives on gene therapy for immune deficiencies.

Podsakoff GM, Engel BC, Kohn DB.

Biol Blood Marrow Transplant. 2005 Dec;11(12):972-6. Review. No abstract available.

13.

Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34(+) cells.

Podsakoff GM, Engel BC, Carbonaro DA, Choi C, Smogorzewska EM, Bauer G, Selander D, Csik S, Wilson K, Betts MR, Koup RA, Nabel GJ, Bishop K, King S, Schmidt M, von Kalle C, Church JA, Kohn DB.

Mol Ther. 2005 Jul;12(1):77-86.

14.

Update on gene therapy of inherited immune deficiencies.

Engel BC, Kohn DB, Podsakoff GM.

Curr Opin Mol Ther. 2003 Oct;5(5):503-7.

PMID:
14601519
16.

Induction of immunity to antigens expressed by recombinant adeno-associated virus depends on the route of administration.

Brockstedt DG, Podsakoff GM, Fong L, Kurtzman G, Mueller-Ruchholtz W, Engleman EG.

Clin Immunol. 1999 Jul;92(1):67-75.

PMID:
10413654
17.

Treatment of lysosomal storage disease in MPS VII mice using a recombinant adeno-associated virus.

Watson GL, Sayles JN, Chen C, Elliger SS, Elliger CA, Raju NR, Kurtzman GJ, Podsakoff GM.

Gene Ther. 1998 Dec;5(12):1642-9.

18.

Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector.

Herzog RW, Yang EY, Couto LB, Hagstrom JN, Elwell D, Fields PA, Burton M, Bellinger DA, Read MS, Brinkhous KM, Podsakoff GM, Nichols TC, Kurtzman GJ, High KA.

Nat Med. 1999 Jan;5(1):56-63.

PMID:
9883840
19.

Recombinant adeno-associated virus mediates a high level of gene transfer but less efficient integration in the K562 human hematopoietic cell line.

Malik P, McQuiston SA, Yu XJ, Pepper KA, Krall WJ, Podsakoff GM, Kurtzman GJ, Kohn DB.

J Virol. 1997 Mar;71(3):1776-83.

20.

Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein.

Kessler PD, Podsakoff GM, Chen X, McQuiston SA, Colosi PC, Matelis LA, Kurtzman GJ, Byrne BJ.

Proc Natl Acad Sci U S A. 1996 Nov 26;93(24):14082-7.

21.

N-ethylmaleimide and the induction of phage lambda.

Podsakoff GM, Ho YL, Bullas LR, Harris TW.

Virology. 1976 Oct 15;74(2):531-4. No abstract available.

PMID:
790756

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