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Lentiviral Gene Therapy with CD34+ Hematopoietic Cells for Hemophilia A.
Srivastava A, Abraham A, Aboobacker F, Singh G, Geevar T, Kulkarni U, Selvarajan S, Korula A, Dave RG, Shankar M, Singh AS, Jeba A, Kumaar N, Benjamin C, Lakshmi KM, Srivastava VM, Shaji RV, Nair SC, Brown HC, Denning G, Lollar P, Doering CB, Spencer T. Srivastava A, et al. N Engl J Med. 2025 Jan 30;392(5):450-457. doi: 10.1056/NEJMoa2410597. Epub 2024 Dec 9. N Engl J Med. 2025. PMID: 39655790 Free PMC article. Clinical Trial.
BACKGROUND: Severe hemophilia A is managed with factor VIII replacement or hemostatic products that stop or prevent bleeding. Data on gene therapy with hematopoietic stem-cell (HSC)-based expression of factor VIII for the treatment …
BACKGROUND: Severe hemophilia A is managed with factor VIII replacement or hemostatic products that stop or prevent bleeding. …
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
Autologous transplantation of gene-modified hematopoietic stem cells may provide a therapeutic strategy for several monogeneic disorders. In previous studies, retroviral gene transfer of coagulation factor VIII (FVIII) into FVIII(-/-) mou …
Autologous transplantation of gene-modified hematopoietic stem cells may provide a therapeutic strategy for several mon …
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.
The percentage of GFP-positive cells was consistently higher following lentiviral versus oncoretroviral transduction, consistent with increased GFP mRNA levels and increased gene transfer efficiency measured by polymerase chain reaction and Sout …
The percentage of GFP-positive cells was consistently higher following lentiviral versus oncoretroviral transduction …
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. ...The product consists of autologous CD34(+) cells transduced with a human immunodeficiency virus 1-based, monocyte lineage-res …
Genetically modified, autologous hematopoietic stem and progenitor cells (HSPCs) represent a new class of genetic medic …
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 …
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.
All these defects have been previously described either in WAS-knockout mice or in WAS patients, validating K562WASKO as a cell model for WAS. However, K562WASPKO cells showed also increased basal F-actin and adhesion, increased expression of CD61 and reduced exp …
All these defects have been previously described either in WAS-knockout mice or in WAS patients, validating K562WASKO as a cell model for WA …