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

    Catherine Cukras, Henry E. Wiley, Brett G. Jeffrey, H. Nida Sen, Amy Turriff, Yong Zeng, Camasamudram Vijayasarathy, Dario Marangoni, Lucia Ziccardi, Sten Kjellstrom, Tae Kwon Park, Suja Hiriyanna, J. Fraser Wright, Peter Colosi, Zhijian Wu, Ronald A. Bush, Lisa L. Wei & Paul A. Sieving, 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

    Shubhranshu Debnath, Pekka Jaako, Kavitha Siva, Michael Rothe, Jun Chen, Maria Dahl, H. Bobby Gaspar, Johan Flygare, Axel Schambach & 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

    Megan D Hoban, Dianne Lumaquin, Caroline Y Kuo, Zulema Romero, Joseph Long, Michelle Ho, Courtney S Young, Michelle Mojadidi, Sorel Fitz-Gibbon, Aaron R Cooper, Georgia R Lill, Fabrizia Urbinati, Beatriz Campo-Fernandez, Carmen Flores Bjurström, Matteo Pellegrini, Roger P Hollis & Donald B Kohn, 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

    K Taipale, I Liikanen, A Koski, R Heiskanen, A Kanerva, O Hemminki, M Oksanen, S Grönberg-Vähä-Koskela, K Hemminki, T Joensuu & A Hemminki, 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.

    Anna Kanerva, Anniina Koski, Ilkka Liikanen, Minna Oksanen, Timo Joensuu, Otto Hemminki, Juni Palmgren, Kari Hemminki & Akseli Hemminki, 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.

    Erik Cederfjäll, Lauren Broom & 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, Jan-Eric Månsson, André R A Marques, Mina Mirzaian, Johannes M Aerts, Mats Ehinger, Michael Rothe, Ute Modlich, Axel Schambach & 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, Giuseppe Manfre, Erika Elgstrand, Angrit Namislo, Christina Isaksson & 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

    Christopher Siatskas, Natalie Seach, Guizhi Sun, Ashley Emerson-Webber, Aude Silvain, Ban-Hock Toh, Frank Alderuccio, B Thomas Bäckström, Richard L Boyd & Claude C Bernard, 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

    Hemraj B. Dodiya, Tomas Björklund, James, III Stansell, Ronald J. Mandel, Deniz Kirik & Jeffrey H. Kordower, 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

    Noriko Miyake, Koichi Miyake, Stefan Karlsson & Takashi Shimada, 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

    Hongjie Wang, Ying Liu, Zongyi Li, Xiaolong Fan, Akseli Hemminki & Andre Lieber, 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.

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

    Research output: Contribution to journalArticle

  22. Fetal Gene Therapy for Acute Neuronopathic Gaucher Disease

    Ahad A. Rahim, Andrew M. Wong, Suzanne M. Buckley, Derralynn A. Hughes, Stefan Karlsson, Jonathan D. Cooper, Adrian J. Thrasher, Atul Mehta & Simon N. Waddington, 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

    Inger Gjertsson, Karen L. Laurie, James Devitt, Steven J. Howe, Adrian J. Thrasher, Rikard Holmdahl & Kenth Gustafsson, 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.

    Irene Kanter Schlifke, Biljana Georgievska, 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

    S Pezet, A Krzyzanowska, LF Wong, J Grist, ND Mazarakis, Biljana Georgievska & SB McMahon, 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.

    Koichi Miyake, Johan Flygare, Thomas Kiefer, Taiju Utsugisawa, Johan Richter, Zhi Ma, Maciej Wiznerowicz, Didier Trono & 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.

    Thomas Relander, Maria Askmyr, Karin Olsson, Yasuhiro Ikeda, Yasuhiro Takeuchi, Mary Collins & 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

    C Baum, C von Kalle, F J T Staal, Z X Li, B Fehse, M Schmidt, F Weerkamp, Stefan Karlsson, G Wagemaker & D A Williams, 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

    Isao Hamaguchi, Johan Flygare, Hiroshi Nishiura, Ann Brun, Andreas Ooka, Thomas Kiefer, Zhi Ma, N Dahl, Johan Richter & Stefan Karlsson, 2003, In: Molecular Therapy. 7, 5, p. 613-622

    Research output: Contribution to journalArticle

  36. 2002