Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8

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3. Potential of AAV vectors in the treatment of metabolic disease
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4. A muscle-targeting peptide displayed on AAV2 improves muscle tropism on systemic delivery
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5. Cardiac I-1c Overexpression With Reengineered AAV Improves Cardiac Function in Swine Ischemic Heart Failure
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7. Life-Long Correction of Hyperbilirubinemia with a Neonatal Liver-Specific AAV-Mediated Gene Transfer in a Lethal Mouse Model of Crigler–Najjar Syndrome
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8. Adeno-associated Virus 9 Mediated FKRP Gene Therapy Restores Functional Glycosylation of α-dystroglycan and Improves Muscle Functions
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9. Manipulation of mtDNA heteroplasmy in all striated muscles of newborn mice by AAV9-mediated delivery of a mitochondria-targeted restriction endonuclease
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10. Gene and cell‐mediated therapies for muscular dystrophy
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11. Percutaneous Transendocardial Delivery of Self-complementary Adeno-associated Virus 6 Achieves Global Cardiac Gene Transfer in Canines
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12. Muscle and cardiac therapeutic strategies for Duchenne muscular dystrophy: past, present, and future
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13. Terminal differentiation of cardiac and skeletal myocytes induces permissivity to AAV transduction by relieving inhibition imposed by DNA damage response proteins
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14. Hypoxia-induced H19/YB-1 cascade modulates cardiac remodeling after infarction
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15. Genetic treatments in muscular dystrophies
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16. Emerging strategies for cell and gene therapy of the muscular dystrophies
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17. AAV-mediated in vivo functional selection of tissue-protective factors against ischaemia
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18. Principles of Optogenetic Methods and Their Application to Cardiac Experimental Systems
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19. Gene Therapy-Mediated Partial Reprogramming Extends Lifespan and Reverses Age-Related Changes in Aged Mice
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20. Rescue of bilirubin‐induced neonatal lethality in a mouse model of Crigler‐Najjar syndrome type I by AAV9‐mediated gene transfer
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21. AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice
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22. Myocardial gene delivery using molecular cardiac surgery with recombinant adeno-associated virus vectors in vivo
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23. Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems
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24. Efficient Whole-body Transduction with Trans-splicing Adeno-associated Viral Vectors
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25. A Potential Role of Distinctively Delayed Blood Clearance of Recombinant Adeno-associated Virus Serotype 9 in Robust Cardiac Transduction
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26. Correction of Multiple Striated Muscles in Murine Pompe Disease Through Adeno-associated Virus–mediated Gene Therapy
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27. AAV-Mediated TAZ Gene Replacement Restores Mitochondrial and Cardioskeletal Function in Barth Syndrome
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28. Genomic expression profiling of human inflammatory cardiomyopathy (DCMi) suggests novel therapeutic targets
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29. Genome engineering: a new approach to gene therapy for neuromuscular disorders
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30. The next step in gene delivery: Molecular engineering of adeno-associated virus serotypes
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31. AAV Vectors for Cardiac Gene Transfer: Experimental Tools and Clinical Opportunities
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32. Heart-targeted adeno-associated viral vectors selected by in vivo biopanning of a random viral display peptide library
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33. An emerging phenotype of central nervous system involvement in Pompe disease: from bench to bedside and beyond
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34. The Role of DNA-PKcs and Artemis in Opening Viral DNA Hairpin Termini in Various Tissues in Mice
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35. Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I
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36. Cardiac-targeted RNA interference mediated by an AAV9 vector improves cardiac function in coxsackievirus B3 cardiomyopathy
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37. Gene Therapy Delivery Systems for Enhancing Viral and Nonviral Vectors for Cardiac Diseases: Current Concepts and Future Applications
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38. Deregulation of XBP1 expression contributes to myocardial vascular endothelial growth factor‐A expression and angiogenesis during cardiac hypertrophy in vivo
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39. Adeno-Associated Virus Mediated Gene Delivery: Implications for Scalable in vitro and in vivo Cardiac Optogenetic Models
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40. Standard screening methods underreport AAV-mediated transduction and gene editing
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41. Current Pharmacological Strategies for Duchenne Muscular Dystrophy
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42. An enhancer-based gene-therapy strategy for spatiotemporal control of cargoes during tissue repair
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43. In vivo genetic engineering of murine pancreatic beta cells mediated by single-stranded adeno-associated viral vectors of serotypes 6, 8 and 9
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44. Current Landscape of Heart Failure Gene Therapy
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47. Glycan Binding Avidity Determines the Systemic Fate of Adeno-Associated Virus Type 9
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48. Genetically Encoded Ca 2+ Indicators in Cardiac Myocytes
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49. Systemic injection of AAV9 carrying a periostin promoter targets gene expression to a myofibroblast-like lineage in mouse hearts after reperfused myocardial infarction
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50. Efficient Gene Transfer to Kidney Mesenchymal Cells Using a Synthetic Adeno-Associated Viral Vector
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51. Efficient Whole-body Transduction with Trans-splicing Adeno-associated Viral Vectors
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52. Augmentation of AAV-mediated cardiac gene transfer after systemic administration in adult rats
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53. Gene Therapy in Large Animal Models of Muscular Dystrophy
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54. Inhibition of miR-155 reduces impaired autophagy and improves prognosis in an experimental pancreatitis mouse model
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55. Next-generation muscle-directed gene therapy by in silico vector design
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56. AAV8-mediated Gene Therapy Prevents Induced Biochemical Attacks of Acute Intermittent Porphyria and Improves Neuromotor Function
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57. Designing Heart Performance by Gene Transfer
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58. Comparison of adeno-associated virus serotypes and delivery methods for cardiac gene transfer
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59. Next Step in Gene Delivery: Modern Approaches and Further Perspectives of AAV Tropism Modification
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60. Augmentation of Left Ventricular Mechanics by Recirculation-Mediated AAV2/1–SERCA2a Gene Delivery in Experimental Heart Failure
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61. Expansive Gene Transfer in the Rat CNS Rapidly Produces Amyotrophic Lateral Sclerosis Relevant Sequelae When TDP-43 is Overexpressed
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67. Improvement of cardiac fibrosis in dystrophic mice by rAAV9-mediated microdystrophin transduction
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68. Prevention of cardiomyopathy in -sarcoglycan knockout mice after systemic transfer of targeted adeno-associated viral vectors
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71. Efficient Retrograde Transport of Adeno-Associated Virus Type 8 to Spinal Cord and Dorsal Root Ganglion After Vector Delivery in Muscle
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72. Adeno-associated virus serotype 9 efficiently targets ischemic skeletal muscle following systemic delivery
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73. Cardiovascular gene therapy for myocardial infarction
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74. Duchenne muscular dystrophy gene therapy: Lost in translation?
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75. Highly Efficient Ultracentrifugation-free Chromatographic Purification of Recombinant AAV Serotype 9
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76. Gene delivery methods in cardiac gene therapy
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77. Circulating microRNAs predispose to takotsubo syndrome following high-dose adrenaline exposure
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81. Expanding Adeno-associated Viral Vector Capacity: A Tale of Two Vectors
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93. Adipose Tissue: An Emerging Target for Adeno-associated Viral Vectors
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