Adeno Associated Virus (AAV) for Cell and Gene Therapy
Type
Blog Post
...trials involve identifying a therapeutic gene, finding a means to deliver it and identifying a suitable...significant clinical efficacy against a form of blindness known as Leber’s congenital amurosis (LCA). This...RPE65. It is characterized by childhood onset of blindness and was thought to be untreatable until 2008. ...their efficacy:
Pre-existing immunity: Many individuals already have wt AAV in their bodies and may therefore...interfere with virus-cell interactions and seriously hinder gene therapy outcomes.
Anti-AAV antibodies developed...decline in vision with gene therapy in childhood blindness." New England Journal of Medicine 372.20 (2015...Quality Control Process
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