Ribozyme rescue of photoreceptor cells in P23H transgenic rats: Long-term survival and late-stage therapy

Ribozyme-directed cleavage of mutant mRNAs appears to be a potentially effective therapeutic measure for dominantly inherited diseases. We previously demonstrated that two ribozymes targeted to the P23H mutation in rhodopsin slow photoreceptor degeneration in transgenic rats for up to 3 months of ag...

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Auteurs principaux: LaVail, Matthew M., Yasumura, Douglas, Matthes, Michael T., Drenser, Kimberly A., Flannery, John G., Lewin, Alfred S., Hauswirth, William W.
Format: Artigo
Langue:Inglês
Publié: The National Academy of Sciences 2000
Sujets:
Biological Sciences
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC17227/
https://ncbi.nlm.nih.gov/pubmed/11005848
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