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Retroviral transfer of the glucocerebrosidase gene into CD34+ cells from patients with Gaucher disease: in vivo detection of transduced cells without myeloablation.

Dunbar CE, Kohn DB, Schiffmann R, Barton NW, Nolta JA, Esplin JA, Pensiero M, Long Z, Lockey C, Emmons RV, Csik S, Leitman S, Krebs CB, Carter C, Brady RO, Karlsson S.

Hum Gene Ther. 1998 Nov 20;9(17):2629-40.


Cytokine mobilization of peripheral blood stem cells in patients with Gaucher disease with a view to gene therapy.

Nimgaonkar M, Mierski J, Beeler M, Kemp A, Lancia J, Mannion-Henderson J, Mohney T, Bahnson A, Rice E, Ball ED, et al.

Exp Hematol. 1995 Dec;23(14):1633-41. Erratum in: Exp Hematol 1996 Feb;24(2):398.


Growth factors and stromal support generate very efficient retroviral transduction of peripheral blood CD34+ cells from Gaucher patients.

Xu LC, Kluepfel-Stahl S, Blanco M, Schiffmann R, Dunbar C, Karlsson S.

Blood. 1995 Jul 1;86(1):141-6.


Correction of the enzyme deficiency in hematopoietic cells of Gaucher patients using a clinically acceptable retroviral supernatant transduction protocol.

Xu L, Stahl SK, Dave HP, Schiffmann R, Correll PH, Kessler S, Karlsson S.

Exp Hematol. 1994 Feb;22(2):223-30.


A bicistronic therapeutic retroviral vector enables sorting of transduced CD34+ cells and corrects the enzyme deficiency in cells from Gaucher patients.

Medin JA, Migita M, Pawliuk R, Jacobson S, Amiri M, Kluepfel-Stahl S, Brady RO, Humphries RK, Karlsson S.

Blood. 1996 Mar 1;87(5):1754-62.


Selection of transduced CD34+ progenitors and enzymatic correction of cells from Gaucher patients, with bicistronic vectors.

Migita M, Medin JA, Pawliuk R, Jacobson S, Nagle JW, Anderson S, Amiri M, Humphries RK, Karlsson S.

Proc Natl Acad Sci U S A. 1995 Dec 19;92(26):12075-9.


Retroviral gene transduction of adult peripheral blood or marrow-derived CD34+ cells for six hours without growth factors or on autologous stroma does not improve marking efficiency assessed in vivo.

Emmons RV, Doren S, Zujewski J, Cottler-Fox M, Carter CS, Hines K, O'Shaughnessy JA, Leitman SF, Greenblatt JJ, Cowan K, Dunbar CE.

Blood. 1997 Jun 1;89(11):4040-6.


Comparison of methods for retroviral mediated transfer of glucocerebrosidase gene to CD34+ hematopoietic progenitor cells.

Takiyama N, Mohney T, Swaney W, Bahnson AB, Rice E, Beeler M, Scheirer-Fochler S, Ball ED, Barranger JA.

Eur J Haematol. 1998 Jul;61(1):1-6.


Retroviral-mediated transfer of the human glucocerebrosidase gene into cultured Gaucher bone marrow.

Nolta JA, Yu XJ, Bahner I, Kohn DB.

J Clin Invest. 1992 Aug;90(2):342-8. Erratum in: J Clin Invest 1992 Oct;90(4):following 1634.


Improved retroviral gene transfer into murine and Rhesus peripheral blood or bone marrow repopulating cells primed in vivo with stem cell factor and granulocyte colony-stimulating factor.

Dunbar CE, Seidel NE, Doren S, Sellers S, Cline AP, Metzger ME, Agricola BA, Donahue RE, Bodine DM.

Proc Natl Acad Sci U S A. 1996 Oct 15;93(21):11871-6.


Transduction of CD34+ enriched cord blood and Gaucher bone marrow cells by a retroviral vector carrying the glucocerebrosidase gene.

Bahnson AB, Nimgaonkar M, Fei Y, Boggs SS, Robbins PD, Ohashi T, Dunigan J, Li J, Ball ED, Barranger JA.

Gene Ther. 1994 May;1(3):176-84.


Transduction of mobilized peripheral blood CD34+ cells with the glucocerebrosidase cDNA.

Nimgaonkar MT, Bahnson AB, Boggs SS, Ball ED, Barranger JA.

Gene Ther. 1994 May;1(3):201-7.


Cell-surface marking of CD(34+)-restricted phenotypes of human hematopoietic progenitor cells by retrovirus-mediated gene transfer.

Ruggieri L, Aiuti A, Salomoni M, Zappone E, Ferrari G, Bordignon C.

Hum Gene Ther. 1997 Sep 1;8(13):1611-23.


No discrepancy between in vivo gene marking efficiency assessed in peripheral blood populations compared with bone marrow progenitors or CD34+ cells.

Sellers SE, Tisdale JF, Bodine DM, Williams DA, Karlsson S, Meztger M, Donahue RE, Dunbar CE.

Hum Gene Ther. 1999 Mar 1;10(4):633-40.


Toward gene therapy for Gaucher disease.

Kohn DB, Nolta JA, Weinthal J, Bahner I, Yu XJ, Lilley J, Crooks GM.

Hum Gene Ther. 1991 Summer;2(2):101-5.


Long-term in vivo expression of the human glucocerebrosidase gene in nonhuman primates after CD34+ hematopoietic cell transduction with cell-free retroviral vector preparations.

Xu LC, Karlsson S, Byrne ER, Kluepfel-Stahl S, Kessler SW, Agricola BA, Sellers S, Kirby M, Dunbar CE, Brady RO, et al.

Proc Natl Acad Sci U S A. 1995 May 9;92(10):4372-6.

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