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Summary of the clinical trials and AAV-based experimental gene therapies.
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Image Search Results


Summary of the clinical trials and AAV-based experimental gene therapies.

Journal: Frontiers in Genetics

Article Title: Clinical, genetic, and experimental research of hyperphenylalaninemia

doi: 10.3389/fgene.2022.1051153

Figure Lengend Snippet: Summary of the clinical trials and AAV-based experimental gene therapies.

Article Snippet: AAV Gene Therapy Study for Subjects With PKU , BMN 307: AAV2/8 gene therapy infusion , Systemic delivery , Phase 1/2 Active, not recruiting , NCT04480567 , BioMarin Pharmaceutical , 24 September 2020 , December 2027 , 15 Years and older.

Techniques: Clinical Proteomics, Plasmid Preparation, Expressing, Transplantation Assay, Drug discovery