A tailored Cln3(Q352X) mouse model for testing therapeutic interventions in CLN3 Batten disease
CLN3 Batten disease (CLN3 disease) is a pediatric lysosomal storage disorder that presents with progressive blindness, motor and cognitive decline, seizures, and premature death. CLN3 disease results from mutations in CLN3 with the most prevalent mutation, a 966 bp deletion spanning exons 7–8, affec...
Autores principales: | , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group UK
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7324379/ https://www.ncbi.nlm.nih.gov/pubmed/32601357 http://dx.doi.org/10.1038/s41598-020-67478-5 |