Aav2-mediated gene therapy for bietti crystalline dystrophy provides functional cyp4v2 in multiple relevant cell models

HIGHLIGHTS

• who: Jiang-Hui Wang from the CentreThe University have published the Article: AAV2-mediated gene therapy for Bietti crystalline dystrophy provides functional CYP4V2 in multiple relevant cell models, in the Journal: Scientific Reports Scientific Reports of 24/Dec/2021
• what: This study has demonstrated that gene therapy using both wild_type (AAV2.wtCYP4V2) and codon-optimised (AAV2.coCYP4V2) CYP4V2 vectors enhanced the level of CYP4V2 protein and function in multiple human cells, including a human iPSCs-derived RPE cells.
• how: The results showed that AAV2 encoding codon-optimised CYP4V2 achieved the effective transduction in . . .
  

   

  

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