Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing

[1]  S. Zolotukhin,et al.  AAV capsid design: A Goldilocks challenge. , 2022, Trends in molecular medicine.

[2]  Miguel R. Chuapoco,et al.  AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset , 2021, Nature Neuroscience.

[3]  R. McKenna,et al.  Structurally Mapping Antigenic Epitopes of Adeno-associated Virus 9: Development of Antibody Escape Variants , 2021, Journal of virology.

[4]  Asher Mullard Gene therapy community grapples with toxicity issues, as pipeline matures , 2021, Nature Reviews Drug Discovery.

[5]  A. Wagers,et al.  Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species , 2021, Cell.

[6]  R. McKenna,et al.  Receptor Switching in Newly Evolved Adeno-associated Viruses , 2021, Journal of virology.

[7]  A. Asokan,et al.  The membrane associated accessory protein is an adeno-associated viral egress factor , 2021, Nature Communications.

[8]  Angela L McCall,et al.  Glycogen accumulation in smooth muscle of a Pompe disease mouse model , 2021, Journal of smooth muscle research = Nihon Heikatsukin Gakkai kikanshi.

[9]  Johannes C. M. van der Loo,et al.  The clinical landscape for AAV gene therapies , 2021, Nature Reviews Drug Discovery.

[10]  J. Mendell,et al.  Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the mdx Mouse Model of Duchenne Muscular Dystrophy , 2021, Human gene therapy.

[11]  S. Boye,et al.  Current Clinical Applications of In Vivo Gene Therapy with AAVs , 2020, Molecular therapy : the journal of the American Society of Gene Therapy.

[12]  D. Grimm,et al.  Identification of a myotropic AAV by massively parallel in vivo evaluation of barcoded capsid variants , 2020, Nature Communications.

[13]  A. Asokan,et al.  Rescuing AAV gene transfer from neutralizing antibodies with an IgG-degrading enzyme , 2020, JCI insight.

[14]  Mingyao Li,et al.  Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology. , 2020, Human gene therapy.

[15]  High-dose AAV gene therapy deaths , 2020, Nature Biotechnology.

[16]  M. Agbandje-McKenna,et al.  Coevolution of Adeno-associated Virus Capsid Antigenicity and Tropism through a Structure-Guided Approach , 2020, Journal of Virology.

[17]  T. Flotte,et al.  Moving forward after two deaths in a gene therapy trial of myotubular myopathy. , 2020, Human gene therapy.

[18]  J. Mendell,et al.  Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy , 2020, JAMA neurology.

[19]  C. Gersbach,et al.  Immunity to Cas9 as an Obstacle to Persistent Genome Editing. , 2020, Molecular therapy : the journal of the American Society of Gene Therapy.

[20]  F. Zhang,et al.  CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors , 2020, Cell.

[21]  R. Samulski,et al.  Engineering adeno-associated virus vectors for gene therapy , 2020, Nature Reviews Genetics.

[22]  B. Byrne,et al.  Reduction of Autophagic Accumulation in Pompe Disease Mouse Model Following Gene Therapy. , 2019, Current gene therapy.

[23]  E. Olson,et al.  CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells , 2019, Science Advances.

[24]  Christine L. Boutros,et al.  Delivering genes across the blood-brain barrier: LY6A, a novel cellular receptor for AAV-PHP.B capsids , 2019, bioRxiv.

[25]  G. Gao,et al.  Adeno-associated virus vector as a platform for gene therapy delivery , 2019, Nature Reviews Drug Discovery.

[26]  Charles A. Gersbach,et al.  Long-term Evaluation of AAV-CRISPR Genome Editing for Duchenne Muscular Dystrophy , 2018, Nature Medicine.

[27]  Keqing Zhang,et al.  AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice. , 2018, JCI insight.

[28]  Qiang Wang,et al.  The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice. , 2018, Molecular therapy : the journal of the American Society of Gene Therapy.

[29]  James M. Wilson,et al.  Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN. , 2018, Human gene therapy.

[30]  Brian K. Kaspar,et al.  Single‐Dose Gene‐Replacement Therapy for Spinal Muscular Atrophy , 2017, The New England journal of medicine.

[31]  A. Asokan,et al.  Mapping the Structural Determinants Required for AAVrh.10 Transport across the Blood-Brain Barrier. , 2017, Molecular therapy : the journal of the American Society of Gene Therapy.

[32]  Thomas Voit,et al.  Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy , 2017, Nature Communications.

[33]  M. Agbandje-McKenna,et al.  Structure-guided evolution of antigenically distinct adeno-associated virus variants for immune evasion , 2017, Proceedings of the National Academy of Sciences.

[34]  Douglas R Martin,et al.  In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy. , 2016, Molecular therapy : the journal of the American Society of Gene Therapy.

[35]  George M. Church,et al.  In vivo gene editing in dystrophic mouse muscle and muscle stem cells , 2016, Science.

[36]  Sripriya Ravindra Kumar,et al.  Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain , 2015, Nature Biotechnology.

[37]  H. Nakai,et al.  Drawing a high-resolution functional map of adeno-associated virus capsid by massively parallel sequencing , 2014, Nature Communications.

[38]  R. Samulski,et al.  Engraftment of a Galactose Receptor Footprint onto Adeno-associated Viral Capsids Improves Transduction Efficiency* , 2013, The Journal of Biological Chemistry.

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

[40]  Jana L Phillips,et al.  Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle , 2010, Nature Biotechnology.

[41]  Allan R. Jones,et al.  A robust and high-throughput Cre reporting and characterization system for the whole mouse brain , 2009, Nature Neuroscience.

[42]  Shahragim Tajbakhsh,et al.  Distinct regulatory cascades govern extraocular and pharyngeal arch muscle progenitor cell fates. , 2009, Developmental cell.

[43]  G. Rubanyi,et al.  Transcriptional silencing is associated with extensive methylation of the CMV promoter following adenoviral gene delivery to muscle , 2004, The journal of gene medicine.

[44]  N. Raben,et al.  Targeted Disruption of the Acid α-Glucosidase Gene in Mice Causes an Illness with Critical Features of Both Infantile and Adult Human Glycogen Storage Disease Type II* , 1998, The Journal of Biological Chemistry.

[45]  Mary Kay Gugerty,et al.  The Goldilocks Challenge , 2018 .