In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease

In utero base editing has the potential to correct disease-causing mutations before the onset of pathology. Mucopolysaccharidosis type I (MPS-IH, Hurler syndrome) is a lysosomal storage disease (LSD) affecting multiple organs, often leading to early postnatal cardiopulmonary demise. We assessed in u...

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Detalles Bibliográficos
Autores principales: Bose, Sourav K., White, Brandon M., Kashyap, Meghana V., Dave, Apeksha, De Bie, Felix R., Li, Haiying, Singh, Kshitiz, Menon, Pallavi, Wang, Tiankun, Teerdhala, Shiva, Swaminathan, Vishal, Hartman, Heather A., Jayachandran, Sowmya, Chandrasekaran, Prashant, Musunuru, Kiran, Jain, Rajan, Frank, David B., Zoltick, Philip, Peranteau, William H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8277817/
https://www.ncbi.nlm.nih.gov/pubmed/34257302
http://dx.doi.org/10.1038/s41467-021-24443-8

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8277817/
https://www.ncbi.nlm.nih.gov/pubmed/34257302
http://dx.doi.org/10.1038/s41467-021-24443-8

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