Article
In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease.
Nature communications - 13 Jul 2021
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
Abstract excerpt
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 utero adeno-associated virus serotype 9 (AAV9) delivery of an adenine base editor (ABE) targeting the Idua G→A...
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