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There are 9049 results for: content related to: Effective AAV-mediated gene therapy in a mouse model of ethylmalonic encephalopathy

  1. Biodistribution and safety assessment of AAV2-GAD following intrasubthalamic injection in the rat

    The Journal of Gene Medicine

    Volume 12, Issue 4, April 2010, Pages: 385–398, Helen L. Fitzsimons, Veronique Riban, Ross J. Bland, Jennifer L. Wendelken, Christine V. Sapan and Matthew J. During

    Version of Record online : 29 MAR 2010, DOI: 10.1002/jgm.1449

  2. Precision Fluorescent Labeling of an Adeno-Associated Virus Vector to Monitor the Viral Infection Pathway

    Biotechnology Journal

    Accepted manuscript online: 22 JAN 2018, Chuanling Zhang, Xueying Zhou, Tianzhuo Yao, Zhenyu Tian and Demin Zhou

    DOI: 10.1002/biot.201700374

  3. You have free access to this content
    Muscle-directed gene therapy for hemophilia B with more efficient and less immunogenic AAV vectors

    Journal of Thrombosis and Haemostasis

    Volume 9, Issue 10, October 2011, Pages: 2009–2019, L. WANG, J.-P. LOUBOUTIN, P. BELL, J. A. GREIG, Y. LI, D. WU and J. M. WILSON

    Version of Record online : 29 SEP 2011, DOI: 10.1111/j.1538-7836.2011.04491.x

  4. Site-Specific Modification of Adeno-Associated Viruses via a Genetically Engineered Aldehyde Tag


    Volume 9, Issue 3, February 11, 2013, Pages: 421–429, Yarong Liu, Yun Fang, Yu Zhou, Ebrahim Zandi, Chi-Lin Lee, Kye-Il Joo and Pin Wang

    Version of Record online : 5 OCT 2012, DOI: 10.1002/smll.201201661

  5. Gene delivery of neurturin to putamen and substantia nigra in Parkinson disease: A double-blind, randomized, controlled trial

    Annals of Neurology

    Volume 78, Issue 2, August 2015, Pages: 248–257, C. Warren Olanow, Raymond T. Bartus, Tiffany L. Baumann, Stewart Factor, Nicholas Boulis, Mark Stacy, Dennis A. Turner, William Marks, Paul Larson, Phillip A. Starr, Joseph Jankovic, Richard Simpson, Ray Watts, Barton Guthrie, Kathleen Poston, Jaimie M. Henderson, Matthew Stern, Gordon Baltuch, Christopher G. Goetz, Christopher Herzog, Jeffrey H. Kordower, Ron Alterman, Andres M. Lozano and Anthony E. Lang

    Version of Record online : 10 JUN 2015, DOI: 10.1002/ana.24436

  6. Long-term functional adeno-associated virus-microdystrophin expression in the dystrophic CXMDj dog

    The Journal of Gene Medicine

    Volume 13, Issue 9, September 2011, Pages: 497–506, Taeyoung Koo, Takashi Okada, Takis Athanasopoulos, Helen Foster, Shin'ichi Takeda and George Dickson

    Version of Record online : 29 SEP 2011, DOI: 10.1002/jgm.1602

  7. Long-term glycemic control with hepatic insulin gene therapy in streptozotocin-diabetic mice

    The Journal of Gene Medicine

    Volume 17, Issue 8-9, August-September 2015, Pages: 141–152, Peter M. Thulé, Adam G. Campbell, Dingwu Jia, Yulin Lin, Shou You, Sara Paveglio, Darin E. Olson and Miroslaw Kozlowski

    Version of Record online : 17 SEP 2015, DOI: 10.1002/jgm.2835

  8. You have free access to this content
    Transgene expression and effective gene silencing in vagal afferent neurons in vivo using recombinant adeno-associated virus vectors

    The Journal of Physiology

    Volume 588, Issue 21, November 2010, Pages: 4303–4315, M. Kollarik, M. J. Carr, F. Ru, C. J. A. Ring, V. J. Hart, P. Murdock, A. C. Myers, Y. Muroi and B. J. Undem

    Version of Record online : 29 OCT 2010, DOI: 10.1113/jphysiol.2010.192971

  9. Insight into the mechanisms of enhanced retinal transduction by the engineered AAV2 capsid variant -7m8

    Biotechnology and Bioengineering

    Volume 113, Issue 12, December 2016, Pages: 2712–2724, Hanen Khabou, Mélissa Desrosiers, Céline Winckler, Stéphane Fouquet, Gwenaëlle Auregan, Alexis-Pierre Bemelmans, José-Alain Sahel and Deniz Dalkara

    Version of Record online : 30 JUN 2016, DOI: 10.1002/bit.26031

  10. Gene therapy strategies for hemophilia: benefits versus risks

    The Journal of Gene Medicine

    Volume 12, Issue 10, October 2010, Pages: 797–809, Inge Petrus, Marinee Chuah and Thierry VandenDriessche

    Version of Record online : 16 SEP 2010, DOI: 10.1002/jgm.1500

  11. Antibody formation and mannose-6-phosphate receptor expression impact the efficacy of muscle-specific transgene expression in murine Pompe disease

    The Journal of Gene Medicine

    Volume 12, Issue 11, November 2010, Pages: 881–891, Baodong Sun, Songtao Li, Andrew Bird, Haiqing Yi, Alex Kemper, Beth L. Thurberg and Dwight D. Koeberl

    Version of Record online : 22 OCT 2010, DOI: 10.1002/jgm.1511

  12. Ectopic expression of TrKA in the adult rat basal ganglia induces both nerve growth factor-dependent and -independent neuronal responses

    Journal of Neuroscience Research

    Volume 90, Issue 8, August 2012, Pages: 1507–1521, Dimitra Giannakopoulou, Véronique Daguin-Nerrière, Adamantia Mitsacos, Elias D. Kouvelas, Isabelle Neveu, Panagiotis Giompres and Philippe Brachet

    Version of Record online : 15 MAR 2012, DOI: 10.1002/jnr.23031

  13. Adeno-associated virus mediated delivery of an engineered protein that combines the complement inhibitory properties of CD46, CD55 and CD59

    The Journal of Gene Medicine

    Volume 17, Issue 6-7, June-July 2015, Pages: 101–115, Derek Leaderer, Siobhan M. Cashman and Rajendra Kumar-Singh

    Version of Record online : 24 JUL 2015, DOI: 10.1002/jgm.2829

  14. Striatal delivery of CERE-120, an AAV2 vector encoding human neurturin, enhances activity of the dopaminergic nigrostriatal system in aged monkeys

    Movement Disorders

    Volume 22, Issue 8, 15 June 2007, Pages: 1124–1132, Christopher D. Herzog, Biplob Dass, James E. Holden, James Stansell III, Mehdi Gasmi, Mark H. Tuszynski, Raymond T. Bartus and Jeffrey H. Kordower

    Version of Record online : 18 APR 2007, DOI: 10.1002/mds.21503

  15. Pseudotyped adeno-associated viral vector tropism and transduction efficiencies in murine wound healing

    Wound Repair and Regeneration

    Volume 20, Issue 4, July-August 2012, Pages: 592–600, Sundeep G. Keswani, Swathi Balaji, Louis Le, Alice Leung, Foong-Yen Lim, Mounira Habli, Helen N. Jones, James M. Wilson and Timothy M. Crombleholme

    Version of Record online : 19 JUN 2012, DOI: 10.1111/j.1524-475X.2012.00810.x

  16. You have full text access to this OnlineOpen article
    Subretinal delivery of adeno-associated virus serotype 2 results in minimal immune responses that allow repeat vector administration in immunocompetent mice

    The Journal of Gene Medicine

    Volume 11, Issue 6, June 2009, Pages: 486–497, Susie E. Barker, Cathryn A. Broderick, Scott J. Robbie, Yanai Duran, Mythili Natkunarajah, Prateek Buch, Kamaljit S. Balaggan, Robert E. MacLaren, James W. B. Bainbridge, Alexander J. Smith and Robin R. Ali

    Version of Record online : 1 APR 2009, DOI: 10.1002/jgm.1327

  17. Synergistic effects of central nervous system-directed gene therapy and bone marrow transplantation in the murine model of infantile neuronal ceroid lipofuscinosis

    Annals of Neurology

    Volume 71, Issue 6, June 2012, Pages: 797–804, Shannon L. Macauley, Marie S. Roberts, Andrew M. Wong, Francesca McSloy, Adarsh S. Reddy, Jonathan D. Cooper and Mark S. Sands

    Version of Record online : 24 FEB 2012, DOI: 10.1002/ana.23545

  18. Bioactivity of AAV2-neurturin gene therapy (CERE-120): Differences between Parkinson's disease and nonhuman primate brains

    Movement Disorders

    Volume 26, Issue 1, January 2011, Pages: 27–36, Raymond T. Bartus, Christopher D. Herzog, Yaping Chu, Alistair Wilson, Lamar Brown, Joao Siffert, Eugene M. Johnson Jr., C. Warren Olanow, Elliott J. Mufson and Jeffrey H. Kordower

    Version of Record online : 18 NOV 2010, DOI: 10.1002/mds.23442

  19. You have free access to this content
    PKA/PrKX activity is a modulator of AAV/adenovirus interaction

    The EMBO Journal

    Volume 22, Issue 7, April 1, 2003, Pages: 1716–1724, Giovanni Di Pasquale and John A. Chiorini

    Version of Record online : 1 APR 2003, DOI: 10.1093/emboj/cdg153

  20. Adeno-associated virus-mediated gene transfer

    Journal of Cellular Biochemistry

    Volume 105, Issue 1, 1 September 2008, Pages: 17–24, Arun Srivastava

    Version of Record online : 23 MAY 2008, DOI: 10.1002/jcb.21819