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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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Detalhes bibliográficos
Publicado no:Int J Mol Sci
Main Authors: Buck, Thilo M., Wijnholds, Jan
Formato: Artigo
Idioma:Inglês
Publicado em: MDPI 2020
Assuntos:
Acesso em linha: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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