Format
Sort by
Items per page

Send to

Choose Destination

Links from PubMed

Items: 1 to 20 of 112

1.

Preclinical studies for a phase 1 clinical trial of autologous hematopoietic stem cell gene therapy for sickle cell disease.

Urbinati F, Wherley J, Geiger S, Fernandez BC, Kaufman ML, Cooper A, Romero Z, Marchioni F, Reeves L, Read E, Nowicki B, Grassman E, Viswanathan S, Wang X, Hollis RP, Kohn DB.

Cytotherapy. 2017 Sep;19(9):1096-1112. doi: 10.1016/j.jcyt.2017.06.002. Epub 2017 Jul 18.

PMID:
28733131
2.

Enrichment of human hematopoietic stem/progenitor cells facilitates transduction for stem cell gene therapy.

Baldwin K, Urbinati F, Romero Z, Campo-Fernandez B, Kaufman ML, Cooper AR, Masiuk K, Hollis RP, Kohn DB.

Stem Cells. 2015 May;33(5):1532-42. doi: 10.1002/stem.1957.

3.

Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of β-thalassemia and sickle cell disease.

Negre O, Bartholomae C, Beuzard Y, Cavazzana M, Christiansen L, Courne C, Deichmann A, Denaro M, de Dreuzy E, Finer M, Fronza R, Gillet-Legrand B, Joubert C, Kutner R, Leboulch P, Maouche L, Paulard A, Pierciey FJ, Rothe M, Ryu B, Schmidt M, von Kalle C, Payen E, Veres G.

Curr Gene Ther. 2015;15(1):64-81.

4.

Potentially therapeutic levels of anti-sickling globin gene expression following lentivirus-mediated gene transfer in sickle cell disease bone marrow CD34+ cells.

Urbinati F, Hargrove PW, Geiger S, Romero Z, Wherley J, Kaufman ML, Hollis RP, Chambers CB, Persons DA, Kohn DB, Wilber A.

Exp Hematol. 2015 May;43(5):346-351. doi: 10.1016/j.exphem.2015.01.009. Epub 2015 Feb 12.

5.

Bone Marrow as a Hematopoietic Stem Cell Source for Gene Therapy in Sickle Cell Disease: Evidence from Rhesus and SCD Patients.

Uchida N, Fujita A, Hsieh MM, Bonifacino AC, Krouse AE, Metzger ME, Donahue RE, Tisdale JF.

Hum Gene Ther Clin Dev. 2017 Sep;28(3):136-144. doi: 10.1089/humc.2017.029. Epub 2017 Apr 17.

6.

Transduction of human hematopoietic stem cells by lentiviral vectors pseudotyped with the RD114-TR chimeric envelope glycoprotein.

Di Nunzio F, Piovani B, Cosset FL, Mavilio F, Stornaiuolo A.

Hum Gene Ther. 2007 Sep;18(9):811-20.

PMID:
17824830
7.

Safety and efficacy of a tCD25 preselective combination anti-HIV lentiviral vector in human hematopoietic stem and progenitor cells.

Barclay SL, Yang Y, Zhang S, Fong R, Barraza A, Nolta JA, Torbett BE, Abedi M, Bauer G, Anderson JS.

Stem Cells. 2015 Mar;33(3):870-9. doi: 10.1002/stem.1919.

8.

Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease.

Poletti V, Urbinati F, Charrier S, Corre G, Hollis RP, Campo Fernandez B, Martin S, Rothe M, Schambach A, Kohn DB, Mavilio F.

Mol Ther Methods Clin Dev. 2018 Nov 1;11:167-179. doi: 10.1016/j.omtm.2018.10.014. eCollection 2018 Dec 14.

9.

Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD34+ cells from Fanconi anemia patients.

Río P, Navarro S, Guenechea G, Sánchez-Domínguez R, Lamana ML, Yañez R, Casado JA, Mehta PA, Pujol MR, Surrallés J, Charrier S, Galy A, Segovia JC, Díaz de Heredia C, Sevilla J, Bueren JA.

Blood. 2017 Sep 28;130(13):1535-1542. doi: 10.1182/blood-2017-03-774174. Epub 2017 Aug 11.

10.
11.

Therapeutic hemoglobin levels after gene transfer in β-thalassemia mice and in hematopoietic cells of β-thalassemia and sickle cells disease patients.

Breda L, Casu C, Gardenghi S, Bianchi N, Cartegni L, Narla M, Yazdanbakhsh K, Musso M, Manwani D, Little J, Gardner LB, Kleinert DA, Prus E, Fibach E, Grady RW, Giardina PJ, Gambari R, Rivella S.

PLoS One. 2012;7(3):e32345. doi: 10.1371/journal.pone.0032345. Epub 2012 Mar 27.

12.

Sustained high-level polyclonal hematopoietic marking and transgene expression 4 years after autologous transplantation of rhesus macaques with SIV lentiviral vector-transduced CD34+ cells.

Kim YJ, Kim YS, Larochelle A, Renaud G, Wolfsberg TG, Adler R, Donahue RE, Hematti P, Hong BK, Roayaei J, Akagi K, Riberdy JM, Nienhuis AW, Dunbar CE, Persons DA.

Blood. 2009 May 28;113(22):5434-43. doi: 10.1182/blood-2008-10-185199. Epub 2009 Apr 1.

13.

Prostaglandin E2 Increases Lentiviral Vector Transduction Efficiency of Adult Human Hematopoietic Stem and Progenitor Cells.

Heffner GC, Bonner M, Christiansen L, Pierciey FJ, Campbell D, Smurnyy Y, Zhang W, Hamel A, Shaw S, Lewis G, Goss KA, Garijo O, Torbett BE, Horton H, Finer MH, Gregory PD, Veres G.

Mol Ther. 2018 Jan 3;26(1):320-328. doi: 10.1016/j.ymthe.2017.09.025. Epub 2017 Oct 5.

14.

Advances in stem cell transplantation and gene therapy in the β-hemoglobinopathies.

Payen E, Leboulch P.

Hematology Am Soc Hematol Educ Program. 2012;2012:276-83. doi: 10.1182/asheducation-2012.1.276. Review.

PMID:
23233592
15.

Hematopoietic stem cell gene transfer for the treatment of hemoglobin disorders.

Persons DA.

Hematology Am Soc Hematol Educ Program. 2009:690-7. doi: 10.1182/asheducation-2009.1.690. Review.

PMID:
20008255
16.

Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome.

Scaramuzza S, Biasco L, Ripamonti A, Castiello MC, Loperfido M, Draghici E, Hernandez RJ, Benedicenti F, Radrizzani M, Salomoni M, Ranzani M, Bartholomae CC, Vicenzi E, Finocchi A, Bredius R, Bosticardo M, Schmidt M, von Kalle C, Montini E, Biffi A, Roncarolo MG, Naldini L, Villa A, Aiuti A.

Mol Ther. 2013 Jan;21(1):175-84. doi: 10.1038/mt.2012.23. Epub 2012 Feb 28. Erratum in: Mol Ther. 2016 Jun;24(6):1159.

17.

Generation of an HIV-1-resistant immune system with CD34(+) hematopoietic stem cells transduced with a triple-combination anti-HIV lentiviral vector.

Walker JE, Chen RX, McGee J, Nacey C, Pollard RB, Abedi M, Bauer G, Nolta JA, Anderson JS.

J Virol. 2012 May;86(10):5719-29. doi: 10.1128/JVI.06300-11. Epub 2012 Mar 7.

18.

Gene Therapy Approaches to Hemoglobinopathies.

Ferrari G, Cavazzana M, Mavilio F.

Hematol Oncol Clin North Am. 2017 Oct;31(5):835-852. doi: 10.1016/j.hoc.2017.06.010. Review.

PMID:
28895851
19.

Improving Gene Therapy Efficiency through the Enrichment of Human Hematopoietic Stem Cells.

Masiuk KE, Brown D, Laborada J, Hollis RP, Urbinati F, Kohn DB.

Mol Ther. 2017 Sep 6;25(9):2163-2175. doi: 10.1016/j.ymthe.2017.05.023. Epub 2017 Jun 27.

20.

Comparison of transduction efficiency among various lentiviruses containing GFP reporter in bone marrow hematopoietic stem cell transplantation.

Wang N, Rajasekaran N, Hou T, Lisowski L, Mellins ED.

Exp Hematol. 2013 Nov;41(11):934-43. doi: 10.1016/j.exphem.2013.07.002. Epub 2013 Aug 14.

Supplemental Content

Support Center