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Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays

Inherited retinal dystrophies and optic neuropathies cause chronic disabling loss of visual function. The development of recombinant adeno-associated viral vectors (rAAV) gene therapies in all disease fields have been promising, but the translation to the clinic has been slow. The safety and efficac...

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Bibliografische gegevens
Gepubliceerd in:Int J Mol Sci
Hoofdauteurs: Buck, Thilo M., Wijnholds, Jan
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: MDPI 2020
Onderwerpen:
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7352801/
https://ncbi.nlm.nih.gov/pubmed/32545533
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms21124197
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