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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Udgivet i: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.
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group UK 2021
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Article
Online adgang: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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