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Genome Editing for Rare Diseases

PURPOSE OF THE REVIEW: Significant numbers of patients worldwide are affected by various rare diseases, but the effective treatment options to these individuals are limited. Rare diseases remain underfunded compared to more common diseases, leading to significant delays in research progress and ulti...

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Detalhes bibliográficos
Publicado no:Curr Stem Cell Rep
Main Authors: Pradhan, Arun, Kalin, Tanya V., Kalinichenko, Vladimir V.
Formato: Artigo
Idioma:Inglês
Publicado em: 2020
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7653884/
https://ncbi.nlm.nih.gov/pubmed/33184603
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s40778-020-00175-1
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