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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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Bibliographische Detailangaben
Veröffentlicht in:Int J Mol Sci
Hauptverfasser: Buck, Thilo M., Wijnholds, Jan
Format: Artigo
Sprache:Inglês
Veröffentlicht: MDPI 2020
Schlagworte:
Online Zugang: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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