Single residue AAV capsid mutation improves transduction of photoreceptors in the Abca4(-/-) mouse and bipolar cells in the rd1 mouse and human retina ex-vivo

Gene therapy using adeno-associated viral vectors (AAV) for the treatment of retinal degenerations has shown safety and efficacy in clinical trials. However, very high levels of vector expression may be necessary for the treatment of conditions such as Stargardt disease where a dual vector approach...

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Udgivet i:Gene Ther
Main Authors: De Silva, Samantha R., Charbel Issa, Peter, Singh, Mandeep S., Lipinski, Daniel M., Barnea-Cramer, Alona O., Walker, Nathan J., Barnard, Alun R., Hankins, Mark W., MacLaren, Robert E.
Format: Artigo
Sprog:Inglês
Udgivet: 2016
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5097463/
https://ncbi.nlm.nih.gov/pubmed/27416076
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2016.54
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