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Gene therapy prevents photoreceptor death and preserves retinal function in a Bardet-Biedl syndrome mouse model

Patients with Bardet-Biedl syndrome (BBS) experience severe retinal degeneration as a result of impaired photoreceptor transport processes that are not yet fully understood. To date, there is no effective treatment for BBS-associated retinal degeneration, and blindness is imminent by the second deca...

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Detalhes bibliográficos
Main Authors: Simons, David L., Boye, Sanford L., Hauswirth, William W., Wu, Samuel M.
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 2011
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3076852/
https://ncbi.nlm.nih.gov/pubmed/21444805
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1019222108
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