Atomic structure of a rationally engineered gene delivery vector, AAV2.5.

[1]  D. Sousa,et al.  Atomic Resolution Structures of Human Bufaviruses Determined by Cryo-Electron Microscopy , 2018, Viruses.

[2]  C. Gersbach,et al.  Genome engineering: a new approach to gene therapy for neuromuscular disorders , 2017, Nature Reviews Neurology.

[3]  Jing-Zhang Wang,et al.  The AAV-mediated and RNA-guided CRISPR/Cas9 system for gene therapy of DMD and BMD , 2017, Brain and Development.

[4]  Jean-Paul Iyombe-Engembe and Jacques P Tremblay.  The advances and challenges of Gene Therapy for Duchenne Muscular Dystrophy , 2017 .

[5]  D. Sousa,et al.  Structural Insights into Human Bocaparvoviruses , 2017, Journal of Virology.

[6]  Dongsheng Duan,et al.  In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy , 2016, Science.

[7]  Scott M Stagg,et al.  The influence of frame alignment with dose compensation on the quality of single particle reconstructions. , 2015, Journal of structural biology.

[8]  R. McKenna,et al.  Structure of neurotropic adeno-associated virus AAVrh.8. , 2015, Journal of structural biology.

[9]  Tetsushi Sakuma,et al.  Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9 , 2014, Stem cell reports.

[10]  R. McKenna,et al.  The structure of AAVrh32.33, a novel gene delivery vector. , 2014, Journal of structural biology.

[11]  Marco Biasini,et al.  SWISS-MODEL: modelling protein tertiary and quaternary structure using evolutionary information , 2014, Nucleic Acids Res..

[12]  M. Agbandje-McKenna,et al.  Adeno-associated virus structural biology as a tool in vector development. , 2013, Future virology.

[13]  J. Chiorini,et al.  Structural Insights into Adeno-Associated Virus Serotype 5 , 2013, Journal of Virology.

[14]  Charles A Gersbach,et al.  Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients , 2013, Molecular therapy : the journal of the American Society of Gene Therapy.

[15]  R. Samulski,et al.  Global CNS Gene Delivery and Evasion of Anti-AAV Neutralizing Antibodies by Intrathecal AAV Administration in Non-Human Primates , 2012, Gene Therapy.

[16]  Conrad C. Huang,et al.  UCSF Chimera, MODELLER, and IMP: an integrated modeling system. , 2012, Journal of structural biology.

[17]  R. Samulski,et al.  Single Amino Acid Modification of Adeno-Associated Virus Capsid Changes Transduction and Humoral Immune Profiles , 2012, Journal of Virology.

[18]  B. Byrne,et al.  Structural Insight into the Unique Properties of Adeno-Associated Virus Serotype 9 , 2012, Journal of Virology.

[19]  M. Agbandje-McKenna,et al.  Parvoviruses: structure and infection , 2012 .

[20]  J. Grieger,et al.  Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector. , 2012, Molecular therapy : the journal of the American Society of Gene Therapy.

[21]  J. Mendell,et al.  Dystrophin immunity in Duchenne's muscular dystrophy. , 2010, The New England journal of medicine.

[22]  Kristin N. Parent,et al.  Structural Characterization of the Dual Glycan Binding Adeno-Associated Virus Serotype 6 , 2010, Journal of Virology.

[23]  M. S. Chapman,et al.  The structure of adeno-associated virus serotype 3B (AAV-3B): insights into receptor binding and immune evasion. , 2010, Virology.

[24]  P. Emsley,et al.  Features and development of Coot , 2010, Acta crystallographica. Section D, Biological crystallography.

[25]  B. Byrne,et al.  Structure of Adeno-Associated Virus Serotype 8, a Gene Therapy Vector , 2007, Journal of Virology.

[26]  J. Chiorini,et al.  Structurally Mapping the Diverse Phenotype of Adeno-Associated Virus Serotype 4 , 2006, Journal of Virology.

[27]  Shangzhen Zhou,et al.  Mutations on the External Surfaces of Adeno-AssociatedVirus Type 2 Capsids That Affect Transduction andNeutralization , 2006, Journal of Virology.

[28]  M. S. Chapman,et al.  The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy , 2002, Proceedings of the National Academy of Sciences of the United States of America.

[29]  M. Agbandje-McKenna,et al.  AAV capsid structure and cell interactions. , 2011, Methods in molecular biology.

[30]  W. Delano.  The PyMOL Molecular Graphics System , 2002 .