Molecular Therapy, 1525-0024

Journal

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  1. 2018
  2. Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery

    Cukras, C., Wiley, H. E., Jeffrey, B. G., Sen, H. N., Turriff, A., Zeng, Y., Vijayasarathy, C., Marangoni, D., Ziccardi, L., Kjellstrom, S., Park, T. K., Hiriyanna, S., Wright, J. F., Colosi, P., Wu, Z., Bush, R. A., Wei, L. L. & Sieving, P. A., 2018 Sep 5, In : Molecular Therapy. 26, 9, p. 2282-2294 13 p.

    Research output: Contribution to journalArticle

  3. 2017
  4. Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia

    Debnath, S., Jaako, P., Siva, K., Rothe, M., Jun Chen, Maria Dahl, Gaspar, H. B., Johan Flygare, Schambach, A. & Stefan Karlsson, 2017, In : Molecular Therapy. 25, 8, p. 1805-1814

    Research output: Contribution to journalArticle

  5. 2016
  6. CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells

    Hoban, M. D., Lumaquin, D., Kuo, C. Y., Romero, Z., Long, J., Ho, M., Young, C. S., Mojadidi, M., Fitz-Gibbon, S., Cooper, A. R., Lill, G. R., Urbinati, F., Campo-Fernandez, B., Flores Bjurström, C., Pellegrini, M., Hollis, R. P. & Kohn, D. B., 2016 Sep, In : Molecular Therapy. 24, 9, p. 1561-9 9 p.

    Research output: Contribution to journalArticle

  7. Predictive and prognostic clinical variables in cancer patients treated with adenoviral oncolytic immunotherapy

    Taipale, K., Liikanen, I., Koski, A., Heiskanen, R., Kanerva, A., Hemminki, O., Oksanen, M., Grönberg-Vähä-Koskela, S., K Hemminki, Joensuu, T. & Hemminki, A., 2016 Jul, In : Molecular Therapy. 24, 7, p. 1323–1332

    Research output: Contribution to journalArticle

  8. 2015
  9. Case-control estimation of the impact of oncolytic adenovirus on the survival of patients with refractory solid tumors.

    Kanerva, A., Koski, A., Liikanen, I., Oksanen, M., Joensuu, T., Hemminki, O., Palmgren, J., Kari Hemminki & Hemminki, A., 2015, In : Molecular Therapy. 23, 2, p. 321-329

    Research output: Contribution to journalArticle

  10. Controlled striatal DOPA production from a gene delivery system in a rodent model of Parkinson's disease.

    Cederfjäll, E., Broom, L. & Deniz Kirik, 2015, In : Molecular Therapy. 23, 5, p. 896-906

    Research output: Contribution to journalArticle

  11. Lentiviral gene therapy using cellular promoters cures type 1 Gaucher disease in mice.

    Maria Dahl, Alexander Doyle, Karin Olsson, Månsson, J-E., Marques, A. R. A., Mirzaian, M., Aerts, J. M., Mats Ehinger, Rothe, M., Modlich, U., Schambach, A. & Stefan Karlsson, 2015, In : Molecular Therapy. 23, 5, p. 835-844

    Research output: Contribution to journalArticle

  12. 2013
  13. Functional Neuroprotection and Efficient Regulation of GDNF Using Destabilizing Domains in a Rodent Model of Parkinson's Disease

    Luis Quintino, Manfre, G., Elgstrand, E., Namislo, A., Isaksson, C. & Cecilia Lundberg, 2013, In : Molecular Therapy. 21, 12, p. 2169-2180

    Research output: Contribution to journalArticle

  14. 2012
  15. Thymic gene transfer of myelin oligodendrocyte glycoprotein ameliorates the onset but not the progression of autoimmune demyelination

    Siatskas, C., Seach, N., Sun, G., Emerson-Webber, A., Silvain, A., Toh, B-H., Alderuccio, F., B Thomas Bäckström, Boyd, R. L. & Bernard, C. C., 2012 Jul, In : Molecular Therapy. 20, 7, p. 1349-59

    Research output: Contribution to journalArticle

  16. 2010
  17. Differential Transduction Following Basal Ganglia Administration of Distinct Pseudotyped AAV Capsid Serotypes in Nonhuman Primates

    Dodiya, H. B., Tomas Björklund, Stansell, J. III., Mandel, R. J., Deniz Kirik & Kordower, J. H., 2010, In : Molecular Therapy. 18, 3, p. 579-587

    Research output: Contribution to journalArticle

  18. Successful Treatment of Metachromatic Leukodystrophy Using Bone Marrow Transplantation of HoxB4 Overexpressing Cells

    Miyake, N., Miyake, K., Stefan Karlsson & Shimada, T., 2010, In : Molecular Therapy. 18, 7, p. 1373-1378

    Research output: Contribution to journalArticle

  19. 2009
  20. A Recombinant Adenovirus Type 35 Fiber Knob Protein Sensitizes Lymphoma Cells to Rituximab Therapy

    Wang, H., Liu, Y., Li, Z., Xiaolong Fan, Hemminki, A. & Lieber, A., 2009, In : Molecular Therapy. 17, p. 240

    Research output: Contribution to journalPublished meeting abstract

  21. Dose Optimization for Long-term rAAV-mediated RNA Interference in the Nigrostriatal Projection Neurons.

    Ulusoy, A., Gurdal Sahin, Tomas Björklund, Aebischer, P. & Deniz Kirik, 2009, In : Molecular Therapy. 17, p. 1574-1584

    Research output: Contribution to journalArticle

  22. Fetal Gene Therapy for Acute Neuronopathic Gaucher Disease

    Rahim, A. A., Wong, A. M., Buckley, S. M., Hughes, D. A., Stefan Karlsson, Cooper, J. D., Thrasher, A. J., Mehta, A. & Waddington, S. N., 2009, In : Molecular Therapy. 17, p. S357-S357

    Research output: Contribution to journalPublished meeting abstract

  23. Tolerance Induction Using Lentiviral Gene Delivery Delays Onset and Severity of Collagen II Arthritis

    Gjertsson, I., Laurie, K. L., Devitt, J., Howe, S. J., Thrasher, A. J., Holmdahl, R. & Gustafsson, K., 2009, In : Molecular Therapy. 17, 4, p. 632-640

    Research output: Contribution to journalArticle

  24. 2007
  25. Seizure Suppression by GDNF Gene Therapy in Animal Models of Epilepsy.

    Kanter Schlifke, I., Georgievska, B., Deniz Kirik & Merab Kokaia, 2007, In : Molecular Therapy. 15, p. 1106-1113

    Research output: Contribution to journalArticle

  26. 2006
  27. Lentiviral Vectors for Use in the Central Nervous System.

    Johan Jakobsson & Cecilia Lundberg, 2006, In : Molecular Therapy. 13, 3, p. 484-493

    Research output: Contribution to journalReview article

  28. Reversal of neurochemical changes and pain-related behavior in a model of neuropathic pain using modified lentiviral vectors expressing GDNF

    Pezet, S., Krzyzanowska, A., Wong, LF., Grist, J., Mazarakis, ND., Georgievska, B. & McMahon, SB., 2006, In : Molecular Therapy. 13, 6, p. 1101-1109

    Research output: Contribution to journalArticle

  29. 2005
  30. Development of cellular models for ribosomal protein S19 (RPS19)-deficient diamond-blackfan anemia using inducible expression of siRNA against RPS19.

    Miyake, K., Johan Flygare, Kiefer, T., Utsugisawa, T., Johan Richter, Ma, Z., Wiznerowicz, M., Trono, D. & Stefan Karlsson, 2005, In : Molecular Therapy. 11, 4, p. 627-637

    Research output: Contribution to journalArticle

  31. Gene Transfer to Repopulating Human CD34(+) Cells Using Amphotropic-, GALV-, or RD114-Pseudotyped HIV-1-Based Vectors from Stable Producer Cells.

    Relander, T., Askmyr, M., Karin Olsson, Ikeda, Y., Takeuchi, Y., Collins, M. & Johan Richter, 2005, In : Molecular Therapy. 11, 3, p. 452-459

    Research output: Contribution to journalArticle

  32. 2004
  33. Chance or necessity? Insertional mutagenesis in gene therapy and its consequences

    Baum, C., von Kalle, C., Staal, F. J. T., Li, Z. X., Fehse, B., Schmidt, M., Weerkamp, F., Stefan Karlsson, Wagemaker, G. & Williams, D. A., 2004, In : Molecular Therapy. 9, 1, p. 5-13

    Research output: Contribution to journalReview article

  34. 2003
  35. Proliferation deficiency of multipotent hematopoietic progenitors in ribosomal protein S19 (RPS19)-deficient Diamond-Blackfan anemia improves following RPS19 gene transfer

    Hamaguchi, I., Johan Flygare, Nishiura, H., Brun, A., Ooka, A., Kiefer, T., Ma, Z., Dahl, N., Johan Richter & Stefan Karlsson, 2003, In : Molecular Therapy. 7, 5, p. 613-622

    Research output: Contribution to journalArticle

  36. 2002