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Development of Lead Hammerhead Ribozyme Candidates against Human Rod Opsin mRNA for Retinal Degeneration Therapy

To identify lead candidate allele-independent hammerhead ribozymes (hhRz) for the treatment of autosomal dominant mutations in the human rod opsin (RHO) gene, we tested a series of hhRzs for potential to significantly knockdown human RHO gene expression in a human cell expression system. Multiple co...

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Detalhes bibliográficos
Main Authors: Abdelmaksoud, Heba E., Yau, Edwin H., Zuker, Michael, Sullivan, Jack M.
Formato: Artigo
Idioma:Inglês
Publicado em: 2008
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3388947/
https://ncbi.nlm.nih.gov/pubmed/19094986
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.exer.2008.11.018
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