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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Publicado no:Nat Commun
Main Authors: 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: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group UK 2021
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC8277817/
https://ncbi.nlm.nih.gov/pubmed/34257302
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-021-24443-8
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