Human Gene Therapy, 1043-0342

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  1. 2019
  2. Hematopoietic stem cell-targeted neonatal gene therapy with a clinically applicable lentiviral vector corrects osteopetrosis in oc/oc Mice

    Löfvall, H., Rothe, M., Schambach, A., Henriksen, K., Johan Richter & Ilana Moscatelli, 2019, In : Human Gene Therapy. 30, 11, p. 1395-1404 10 p.

    Research output: Contribution to journalArticle

  3. 2018
  4. Targeting NSG Mice Engrafting Cells with a Clinically Applicable Lentiviral Vector Corrects Osteoclasts in Infantile Malignant Osteopetrosis

    Ilana Moscatelli, Löfvall, H., Schneider Thudium, C., Rothe, M., Montano, C., Kertész, Z., Sirin, M., Schulz, A., Schambach, A., Henriksen, K. & Johan Richter, 2018, In : Human Gene Therapy. 29, 8, p. 938-949

    Research output: Contribution to journalArticle

  5. 2016
  6. Preclinical Dose-Escalation Study of Intravitreal AAV-RS1 Gene Therapy in a Mouse Model of X-linked Retinoschisis: Dose-Dependent Expression and Improved Retinal Structure and Function

    Bush, R. A., Zeng, Y., Colosi, P., Kjellström, S., Hiriyanna, S., Vijayasarathy, C., Santos, M., Li, J., Wu, Z. & Sieving, P. A., 2016, In : Human Gene Therapy. 27, 5, p. 376-389 14 p.

    Research output: Contribution to journalArticle

  7. 2013
  8. Lentivirus-Induced Dendritic Cells for Immunization Against High-Risk WT1(+) Acute Myeloid Leukemia

    Sundarasetty, B. S., Singh, V. K., Salguero, G., Geffers, R., Rickmann, M., Macke, L., Borchers, S., Figueiredo, C., Schambach, A., Urban Gullberg, Provasi, E., Bonini, C., Ganser, A., Woelfel, T. & Stripecke, R., 2013, In : Human Gene Therapy. 24, 2, p. 220-237

    Research output: Contribution to journalArticle

  9. 2011
  10. 2010
  11. A Human Model of IMO by Lentiviral Mediated shRNA Knock-down of Tcirg1 in CD34+Cells

    Ilana Moscatelli, Thudium, C. S., Flores, C., Henriksen, K. & Richter, J., 2010, In : Human Gene Therapy. 21, 10, p. 1458-1459

    Research output: Contribution to journalPublished meeting abstract

  12. Development of Novel Therapies in Murine Models for Gaucher Disease

    Stefan Karlsson, 2010, In : Human Gene Therapy. 21, 4, p. 490-491

    Research output: Contribution to journalPublished meeting abstract

  13. 2008
  14. Neonatal hematopoietic stem cell transplantation following low-dose busulphan conditioning reverses osteopetrosis and preserves vision in oc/oc mice

    Flores Bjurström, C., Askmyr, M., Holmberg, J., Mats Ehinger, Hjalt, T. & Johan Richter, 2008, In : Human Gene Therapy. 19, 10, p. 1183-1184

    Research output: Contribution to journalPublished meeting abstract

  15. 2004
  16. Regulated delivery of glial cell line-derived neurotrophic factor into rat striatum, using a tetracycline-dependent lentiviral vector.

    Georgievska, B., Johan Jakobsson, Persson, E., Ericson, C., Deniz Kirik & Cecilia Lundberg, 2004, In : Human Gene Therapy. 15, 10, p. 934-944

    Research output: Contribution to journalArticle

  17. 2002
  18. 2001
  19. Glucose-regulated insulin expression in diabetic rats

    Barry, S. C., Ramesh, N., Lejnieks, D. V., Simonson, W. T., Kemper, L., Å Lernmark & Osborne, W. R. A., 2001 Jan 20, In : Human Gene Therapy. 12, 2, p. 131-139 9 p.

    Research output: Contribution to journalArticle

  20. 1996
  21. Retroviral display of antibody fragments; interdomain spacing strongly influences vector infectivity

    Ager, S., B H Nilson, Morling, F. J., Peng, K. W., Cosset, F. L. & Russell, S. J., 1996 Nov 10, In : Human Gene Therapy. 7, 17, p. 2157-2164

    Research output: Contribution to journalArticle