405. Hindlimb and Forelimb Strength Assessment in a Canine Model of X-Linked Myotubular Myopathy Following AAV-Mediated Gene Replacement    Molecular.

Slides:



Advertisements
Similar presentations
Gene Preference in Maple Syrup Urine Disease Mary M. Nellis, Dean J. Danner The American Journal of Human Genetics Volume 68, Issue 1, Pages (January.
Advertisements

Jennifer G. Cheung, Krystyna Konopka, Nejat Düzgünes
Volume 20, Issue 2, (February 2012)
414. A Novel Clinical Severity Scoring Measure in Canine X-Linked Myotubular Myopathy: Results Following Systemic AAV Gene Replacement    Molecular Therapy 
209. Use of Helicobacter pyroli Neutrophil Activating Protein (NAP) as an Immune- Modulatory Agent To Enhance the Efficacy of Oncolytic Adenovirus Therapy.
446. Gender Differences in the Osteogenic & Chondrogenic Potential of Human Muscle- Derived Stem Cells In Vitro  Alex C. Scibetta, Andrea B. Liebowitz,
Man's Best Friend: Utilizing Naturally Occurring Tumors in Dogs to Improve Chimeric Antigen Receptor T-cell Therapy for Human Cancers  Melinda Mata, Stephen.
Engineering Natural Killer Cells for Cancer Immunotherapy
Genome-editing Technologies for Gene and Cell Therapy
How to Treat MDS without Stem Cell Transplantation
627. Non-Invasive, Multimodal Imaging of Microvesicles with Metabolically Biotinylated, Membrane-Bound Gaussia Luciferase    Molecular Therapy  Volume.
Elizabeth Morris, Alex Scibetta, Aiping Lu, Xueqin Gao, Johnny Huard
162. Stability of Polymer/Plasmid DNA Complexes In Vitro and In Vivo
281. Rapid Generation of Induced Pluripotent Stem Cells (iPSCs) from the Urine of a Patient with Duchenne Muscular Dystrophy    Molecular Therapy  Volume.
Direct Conversion of Skin Cells into Blood: Alchemy or Science?
Molecular Therapy  Volume 20, Pages S261-S262 (May 2012) DOI: /S (16)
Evolving Gene Therapy in Primary Immunodeficiency
343. Benchtop DNA Synthesizer: Oligo-Templated Polymerization (OTP)
FIX It in One Go: Enhanced Factor IX Gene Therapy for Hemophilia B
Section 5: Dialysis Interventions for Treatment of AKI
Engineering Natural Killer Cells for Cancer Immunotherapy
637. Effect of PEG Grafting Degree on Morphology and Transfection Efficiency of Polycation-g-PEG/DNA Nanoparticles    Molecular Therapy  Volume 20, (May.
Escaping the Valley of Death
220. AAV-Mediated miR122 Overexpression Rescues Hepatic Inflammatory Response Induced by Acute Iron-Overload  Min Li, Yuxiao Tang, Lusha Wu, Fengfeng.
Francesco Paolo Schena  Kidney International 
498. Prolonged Benefit from Systemic rAAV8 in a Canine Model of Myotubular Myopathy  Matthew Elverman, David Mack, Melissa Goddard, Jessica Snyder, Robert.
John R. Martin, Christopher E. Nelson, Mukesh K
Volume 26, Issue 2, Pages (February 2018)
Volume 25, Issue 1, Pages 5-7 (January 2017)
Chen Ling, Baozheng Li, Wenqin Ma, George Aslanidi, Arun Srivastava
The Other Face of Chimeric Antigen Receptors
Follicular Lymphoma: Prognostic Factors, Conventional Therapies, and Hematopoietic Cell Transplantation  Laurie H. Sehn, Timothy S. Fenske, Ginna G. Laport 
724. Blood-Derived Endothelial Progenitor Cells Can Be Isolated With a Novel Method of Diluted Whole Blood Incubation with AMD3100-Induced Mobilization.
Genome-editing Technologies for Gene and Cell Therapy
Premal Lulla, Carlos A. Ramos  Molecular Therapy 
Molecular Therapy  Volume 20, (May 2012) DOI: /S (16)
Molecular Therapy  Volume 7, Issue 5, (May 2003) DOI: /S (16)
547. AAV Preparations Contain Contamination from DNA Sequences in Production Plasmids Directly Outside of the ITRs  Mark A. Brimble, Junfang Zhou, Christopher.
847. Eradication of Therapy-Resistant Human Prostate Tumors Using an Ultrasound Guided Site-Specific Cancer Terminator Virus Delivery Approach    Molecular.
611. Alpha-Dystrobrevin-3 Prevents Myopathy and Restores Diaphragm Function in the Alpha-Dystrobrevin Null Mouse  Guy L. Odom, Glen B. Banks, Marvin E.
Designer Lipids Advance Systemic siRNA Delivery
133. Survival with Normal Neurological Development of the Juvenile Lethal Urea Cycle Defect Arginase Deficient Mouse with AAV Gene Therapy    Molecular.
660. Bowel/Bladder Sensation and Control in Patients with Spinal Cord Injury Treated with Human Embryonic Stem Cell Therapy  Geeta Shroff  Molecular Therapy 
Volume 26, Issue 2, Pages (February 2018)
68. Site Specific Intra-Placental Gene Transfer Corrects Fetal Growth Restriction in a Novel Mouse Model of Placental Insufficiency (PI)    Molecular.
521. Multiplex Genome Editing of TCR°/CD52 Genes as a Platform for “Off the Shelf” Adoptive T-Cell Immunotherapies    Molecular Therapy  Volume 22, Pages.
Jennifer G. Cheung, Krystyna Konopka, Nejat Düzgünes
Chae-lyul Limg, Yeon-Ju Lee, Sujeong Kim
335. High Content Analysis Platform for Optimization of Lipid Mediated CRISPR-Cas9 Delivery Strategies in Human Cells  Benjamin Steyer, Jared Carlson-Stevermer,
Volume 26, Issue 4, Pages (April 2018)
Gerard Socié, K. Scott Baker, Smita Bhatia 
674. Molecular, Biochemical and Biomechanical Analysis of Articular Cartilage Repaired with Genetically Modified Chondrocytes Expressing Insulin-Like.
Molecular Therapy  Volume 18, Pages S260-S261 (May 2010) DOI: /S (16)
In This Issue Molecular Therapy Volume 16, Issue 4, (April 2008)
372. Targeted Mutagenesis of Ubiquitin-Binding Lysine Residues on the Adeno- Associated Virus (AAV)2 Capsid Improves Its Transduction Efficiency    Molecular.
521. AAV Immunotherapy Induces Functional Antigen Specific Regulatory T-Cells to a Neuroantigen: A Potential Treatment for MS  Brad E. Hoffman  Molecular.
483. In Vivo DMD Gene Editing in Muscles and Muscle Stem Cells of Dystrophic Mice  Mohammadsharif Tabebordbar, Kexian Zhu, Jason Cheng, Jeffrey Widrick,
773. Detection of RNA Interference (RNAi) Mediated mRNA Cleavage in Fresh Injected Tumor Tissue from Patients in a Phase I Trial of pbi-shRNA™ Lipoplex.
740. Prevention of Radiation-Induced Lung Injury by Administration of Gene-Modified Mesenchymal Stem Cells    Molecular Therapy  Volume 20, Pages S285-S286.
Molecular Therapy  Volume 21, Pages S247-S248 (May 2013)
Shigeki Yagyu, Malcolm K. Brenner
History of Oncolytic Viruses: Genesis to Genetic Engineering
86. A Highly Compact Epitope-Based Marker Suicide Gene for Safer and Easier Adoptive T-Cell Gene Therapy    Molecular Therapy  Volume 20, Pages S35-S36.
297. Minimally Invasive Implantation of Autologous Chondrocytes Transduced with rAAV5-IGF-I Improves Longterm Cartilage Repair in Full-Thickness Chondral.
441. Direct Comparison of EF1α and PGK Promoters Reveals Superior Performance of the PGK Promoter for Expression of the Common Gamma Chain in a Canine.
10. High-Efficiency Transduction of Primary Human CD34+ Hematopoietic Stem/Progenitor Cells by AAV6 Serotype Vectors: Strategies for Overcoming Donor.
Sensing Bad: Are Co-stimulatory CAR-Expressing γδ T Cells Safer?
Jingxian Feng, Jiawen Wang, Junsheng Lin, Yong Diao
Shirley Wong, Roderick A. Slavcev
624. Randomized Phase II Trial of Adjuvant Autologous Tumor Cell Vaccine (FANG™) for High Risk Stage III/IV Ovarian Cancer: Preliminary Results    Molecular.
Presentation transcript:

405. Hindlimb and Forelimb Strength Assessment in a Canine Model of X-Linked Myotubular Myopathy Following AAV-Mediated Gene Replacement    Molecular Therapy  Volume 22, Pages S154-S155 (May 2014) DOI: 10.1016/S1525-0016(16)35418-1 Copyright © 2014 The American Society of Gene & Cell Therapy Terms and Conditions

Figure 1 a) Comparison of all forelimb extension torque measurements to hindlimb flexion torque measurements. b) Comparison of forelimb extension torque measurements to hindlimb flexion torque measurements separated by treatment group. Molecular Therapy 2014 22, S154-S155DOI: (10.1016/S1525-0016(16)35418-1) Copyright © 2014 The American Society of Gene & Cell Therapy Terms and Conditions