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Preservation of Photoreceptor Morphology and Function in P23H Rats Using an Allele Independent Ribozyme

To develop an allele independent ribozyme for the treatment of Autosomal Dominant Retinitis Pigmentosa (ADRP) associated with mutations in the rhodopsin (RHO) gene. A ribozyme targeting dog, mouse, human but not rat rhodopsin (RHO) mRNA was designed and tested in vitro. Activity of this ribozyme was...

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Autores principales: Gorbatyuk, M., Justilien, V., Liu, J., Hauswirth, W.W., Lewin, A.S
Formato: Artigo
Lenguaje:Inglês
Publicado: 2006
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Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC1778459/
https://ncbi.nlm.nih.gov/pubmed/17083931
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.exer.2006.08.014
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