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Preclinical Development of a Hematopoietic Stem and Progenitor Cell Bioengineered Factor VIII Lentiviral Vector Gene Therapy for Hemophilia A.
Doering CB, Denning G, Shields JE, Fine EJ, Parker ET, Srivastava A, Lollar P, Spencer HT. Doering CB, et al. Hum Gene Ther. 2018 Oct;29(10):1183-1201. doi: 10.1089/hum.2018.137. Hum Gene Ther. 2018. PMID: 30160169 Free PMC article.
Genetically modified, autologous hematopoietic stem and progenitor cells (HSPCs) represent a new class of genetic medicine. Following this therapeutic paradigm, we are developing a product candidate, designated CD68-ET3-LV CD34(+), for the treatment of …
Genetically modified, autologous hematopoietic stem and progenitor cells (HSPCs) represent a new class of genetic medic …
Recombinant factor VIII expression in hematopoietic cells following lentiviral transduction.
Tiede A, Eder M, von Depka M, Battmer K, Luther S, Kiem HP, Ganser A, Scherr M. Tiede A, et al. Gene Ther. 2003 Oct;10(22):1917-25. doi: 10.1038/sj.gt.3302093. Gene Ther. 2003. PMID: 14502221
In previous studies, retroviral gene transfer of coagulation factor VIII (FVIII) into FVIII(-/-) mouse bone marrow (BM) cells did not result in detectable plasma FVIII levels. ...Here, we used lentiviral vectors to study the ability of various …
In previous studies, retroviral gene transfer of coagulation factor VIII (FVIII) into FVIII(-/-) mouse bone marrow (BM) …
Efficient lentiviral transduction and improved engraftment of human bone marrow mesenchymal cells.
Van Damme A, Thorrez L, Ma L, Vandenburgh H, Eyckmans J, Dell'Accio F, De Bari C, Luyten F, Lillicrap D, Collen D, VandenDriessche T, Chuah MK. Van Damme A, et al. Stem Cells. 2006 Apr;24(4):896-907. doi: 10.1634/stemcells.2003-0106. Epub 2005 Dec 9. Stem Cells. 2006. PMID: 16339997 Free article.
Human bone marrow (BM) mesenchymal stem/progenitor cells are potentially attractive targets for ex vivo gene therapy. The potential of lentiviral vectors for transducing BM mesenchymal cells was examined using a self-inactivating vector that
Human bone marrow (BM) mesenchymal stem/progenitor cells are potentially attractive targets for ex vivo gene therapy. T
Use of zinc-finger nucleases to knock out the WAS gene in K562 cells: a human cellular model for Wiskott-Aldrich syndrome.
Toscano MG, Anderson P, Muñoz P, Lucena G, Cobo M, Benabdellah K, Gregory PD, Holmes MC, Martin F. Toscano MG, et al. Dis Model Mech. 2013 Mar;6(2):544-54. doi: 10.1242/dmm.010652. Epub 2013 Jan 11. Dis Model Mech. 2013. PMID: 23324327 Free PMC article.
However, K562WASPKO cells showed also increased basal F-actin and adhesion, increased expression of CD61 and reduced expression of TGFβ and Factor VIII, defects that have never been described before for WAS-deficient cells. ...All phenoty …
However, K562WASPKO cells showed also increased basal F-actin and adhesion, increased expression of CD61 and reduced expres
In vivo enrichment of genetically manipulated platelets corrects the murine hemophilic phenotype and induces immune tolerance even using a low multiplicity of infection.
Schroeder JA, Chen Y, Fang J, Wilcox DA, Shi Q. Schroeder JA, et al. J Thromb Haemost. 2014 Aug;12(8):1283-93. doi: 10.1111/jth.12633. Epub 2014 Jul 17. J Thromb Haemost. 2014. PMID: 24931217 Free PMC article.
BACKGROUND: Our previous studies have demonstrated that platelet-specific gene delivery to hematopoietic stem cells can induce sustained therapeutic levels of platelet factor VIII (FVIII) expression in mice with hemophilia A. ...Platelet …
BACKGROUND: Our previous studies have demonstrated that platelet-specific gene delivery to hematopoietic stem cells can …
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