Selection of appropriate non-clinical animal models to ensure translatability of novel AAV-gene therapies to the clinic

Author:

Singh MarkORCID,Brooks Andrew,Toofan Parto,McLuckie Keith

Publisher

Springer Science and Business Media LLC

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference97 articles.

1. EMA. EMA - Guideline on the quality, non-clinical and clinical aspects of gene therapy medicinal products. 2018; Available from: https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-quality-non-clinical-clinical-aspects-gene-therapy-medicinal-products_en.pdf.

2. EPAR - Luxturna [Internet]. 2019. Available from: https://www.ema.europa.eu/en/documents/assessment-report/luxturna-epar-public-assessment-report_en.pdf.

3. EPAR - Zolgensma [Internet]. 2020. Available from: https://www.ema.europa.eu/en/documents/assessment-report/zolgensma-epar-public-assessment-report_en.pdf.

4. EMA. EPAR - Glybera [Internet]. 2012. Available from: https://www.ema.europa.eu/en/documents/assessment-report/glybera-epar-public-assessment-report_en.pdf.

5. Roctavian-EMA [Internet]. 2022. Available from: https://www.ema.europa.eu/en/medicines/human/EPAR/roctavian-0.

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