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- W2951784992 abstract "Parkinson's disease (PD) is the second most common neurodegenerative disorder. It is characterised by selective loss of dopaminergic neurons in the substantia nigra pars compacta, which results in dopamine depletion, leading to a number of motor and non-motor symptoms. In recent years, the development of new animal models using nuclease-based genome-editing technology (ZFN, TALEN, and CRISPR/Cas9 nucleases) has enabled the introduction of custom-made modifications into the genome to replicate key features of PD, leading to significant advances in our understanding of the pathophysiology of the disease. We review the most recent studies on this new generation of in vitro and in vivo PD models, which replicate the most relevant symptoms of the disease and enable better understanding of the aetiology and mechanisms of PD. This may be helpful in the future development of effective treatments to halt or slow disease progression. La enfermedad de Parkinson (EP) es el segundo trastorno neurodegenerativo más común, caracterizado por la pérdida selectiva de neuronas dopaminérgicas en la substancia nigra pars compacta, produciendo depleción en los niveles de dopamina y dando como resultado las manifestaciones clínicas de la enfermedad que se pueden clasificar como síntomas motrices y no motrices. En los últimos años, la generación de nuevos modelos animales basados en los sistemas de edición genética por nucleasas: ZFN, TALEN, CRISPR/Cas9, permiten la realización de modificaciones personalizadas en el genoma, replicando características clave que definen a la EP y consecuentemente avances significativos en la comprensión del proceso fisiopatológico de este trastorno. En esta revisión recopilamos los estudios más novedosos de esta nueva generación de modelos in vitro e in vivo de la EP que permiten emular síntomas clave y tener una mayor comprensión de la etiología o los mecanismos involucrados en el proceso de iniciación o desarrollo de la enfermedad y la futura prueba de terapias realmente eficaces para detener o ralentizar su progresión." @default.
- W2951784992 created "2019-06-27" @default.
- W2951784992 creator A5031377682 @default.
- W2951784992 creator A5034098285 @default.
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- W2951784992 creator A5046746256 @default.
- W2951784992 creator A5067698498 @default.
- W2951784992 creator A5007370356 @default.
- W2951784992 date "2020-09-01" @default.
- W2951784992 modified "2023-10-01" @default.
- W2951784992 title "New transgenic models of Parkinson's disease using genome editing technology" @default.
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