A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

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7. High-density brush-shaped polymer lipids reduce anti-PEG antibody binding for repeated administration of mRNA therapeutics
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13. In Vivo Genome Editing as a Therapeutic Approach
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14. CRISPR-Cas9 genome engineering: Treating inherited retinal degeneration
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15. AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice
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16. Gene Therapy Intervention in Neovascular Eye Disease: A Recent Update
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17. CRISPR/Cas9 system in breast cancer therapy: advancement, limitations and future scope
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18. Strategies for In Vivo Genome Editing in Nondividing Cells
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19. Amelioration of Alpha-1 Antitrypsin Deficiency Diseases with Genome Editing in Transgenic Mice
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20. Advances with using CRISPR/Cas-mediated gene editing to treat infections with hepatitis B virus and hepatitis C virus
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21. Different Methods of Delivering CRISPR/Cas9 Into Cells
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22. Poly(Beta-Amino Ester) Nanoparticles Enable Nonviral Delivery of CRISPR-Cas9 Plasmids for Gene Knockout and Gene Deletion
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24. Safe CRISPR: Challenges and Possible Solutions
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29. Targeted Gene Editing of Glia Maturation Factor in Microglia: a Novel Alzheimer’s Disease Therapeutic Target
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31. The Development of a Viral Mediated CRISPR/Cas9 System with Doxycycline Dependent gRNA Expression for Inducible In vitro and In vivo Genome Editing
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32. Creating cell and animal models of human disease by genome editing using CRISPR/Cas9
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34. Delivery of CRISPR/Cas9 by Novel Strategies for Gene Therapy
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35. Rare Opportunities: CRISPR/Cas-Based Therapy Development for Rare Genetic Diseases
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36. Engineer chimeric Cas9 to expand PAM recognition based on evolutionary information
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37. Design of AAV Vectors for Delivery of Large or Multiple Transgenes
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38. Efficacy and long-term safety of CRISPR/Cas9 genome editing in the SOD1-linked mouse models of ALS
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39. ON-Target Adverse Events of CRISPR-Cas9 Nuclease: More Chaotic than Expected
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40. Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9
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42. Inhibition of HBV Expression in HBV Transgenic Mice Using AAV-Delivered CRISPR-SaCas9
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44. Promoterless gene targeting without nucleases rescues lethality of a Crigler‐Najjar syndrome mouse model
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45. The rapidly advancing Class 2 CRISPR‐Cas technologies: A customizable toolbox for molecular manipulations
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46. Delivery Approaches for Therapeutic Genome Editing and Challenges
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47. Coupling AAV-mediated promoterless gene targeting to SaCas9 nuclease to efficiently correct liver metabolic diseases
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49. CRISPR-Cas9-Mediated In Vivo Gene Integration at the Albumin Locus Recovers Hemostasis in Neonatal and Adult Hemophilia B Mice
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53. Progress and challenges towards CRISPR/Cas clinical translation
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55. Targeted in vivo knock-in of human alpha-1-antitrypsin cDNA using adenoviral delivery of CRISPR/Cas9
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56. Engineering designer beta cells with a CRISPR-Cas9 conjugation platform
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63. AAV9-NPC1 significantly ameliorates Purkinje cell death and behavioral abnormalities in mouse NPC disease
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64. Genome editing for inborn errors of metabolism: advancing towards the clinic
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65. The Hope and Hype of CRISPR-Cas9 Genome Editing
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67. In Situ Modification of Tissue Stem and Progenitor Cell Genomes
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69. Aptamer Chimeras for Therapeutic Delivery: The Challenging Perspectives
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70. Electrosprayed Alginate Nanoparticles as CRISPR Plasmid DNA Delivery Carrier: Preparation, Optimization, and Characterization
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76. Applications of Genome Editing Technology in Animal Disease Modeling and Gene Therapy
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77. Prevention of acquired sensorineural hearing loss in mice by in vivo Htra2 gene editing
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81. Treatment of monogenic and digenic dominant genetic hearing loss by CRISPR-Cas9 ribonucleoprotein delivery in vivo
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83. AAV11 enables efficient retrograde targeting of projection neurons and enhances astrocyte-directed transduction
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84. Genome Editing in Cardiovascular Biology
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