Gene therapy

General Adeno-associated Viral Vector (AAV) Platforms


  • Customized recombinant AAV (rAAV) genome construction and validation
  • General AAV packaging, purification and tittering
  • Premade AAV control vectors
  • In vitro and in vivo transduction

Fig 1. Customized recombinant AAV (rAAV) genome construction


Fig 2. In vitro validation by two clinically-relevant rAAV vectors



AAV-mediated Gene Replacement Therapy


    • Customized therapeutic rAAV vector design, construction and packaging
    • In vitro validation of the therapeutic effect
    • Dose-escalation long-term efficacy and safety evaluation in vivo including wild type and disease models


Fig 3. Schematic diagram of AAV-mediated Gene Replacement Therapy


Fig4. Efficacy and safety evaluation of AAV8-Gene A in vivo.
a. Biodistribution of AAV8-GFP in mice.
b. Transduction efficiency of AAV8-GFP vector in liver.
c. Gene A expression levels in mice at week 1, 2, 4, 6 and 8 post viral delivery.
d. No alanine transaminase (ALT) change demonstrate the safety of the gene delivery.  



AAV-mediated In Vivo Genome Editing and Therapy


  • Customized rAAV vector design , construction and packaging for in vivo genome editing
  • In vitro validation of genome editing
  • In vivo genome editing efficiency and functional evaluation


Fig 5. Schematic diagram of in vivo genome editing and therapy


Fig 6. Validation of in vivo KO by AAV8-sgGene B. a, Editing efficiency of AAV8-sgGene B. b, Representative indels formation in liver tissue.



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