Recapture Lysosomal Enzyme Deficiency via Targeted Gene Disruption in the Human Near-Haploid Cell Line HAP1

Background: Advancement in genome engineering enables rapid and targeted disruption of any coding sequences to study gene functions or establish human disease models. We explored whether this approach can be used to study Gaucher disease, one of the most common types of lysosomal storage diseases (L...

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Detalles Bibliográficos
Autores principales: Brown, Annie, Zhang, Jiayi, Lawler, Brendan, Lu, Biao
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8308024/
https://www.ncbi.nlm.nih.gov/pubmed/34356092
http://dx.doi.org/10.3390/genes12071076

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8308024/
https://www.ncbi.nlm.nih.gov/pubmed/34356092
http://dx.doi.org/10.3390/genes12071076

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