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In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease.
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.
Affiliation
  • Bose SK; Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • White BM; Division of General, Thoracic and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Kashyap MV; Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Dave A; Division of General, Thoracic and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • De Bie FR; Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Li H; Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Singh K; Division of General, Thoracic and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Menon P; Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Wang T; Division of General, Thoracic and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Teerdhala S; Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Swaminathan V; Division of General, Thoracic and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Hartman HA; Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Jayachandran S; Division of General, Thoracic and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Chandrasekaran P; Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Musunuru K; Division of General, Thoracic and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Jain R; Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Frank DB; Division of General, Thoracic and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Zoltick P; Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Peranteau WH; Division of General, Thoracic and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Nat Commun ; 12(1): 4291, 2021 07 13.
Article in En | MEDLINE | ID: mdl-34257302
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Lysosomal Storage Diseases Limits: Animals / Humans Language: En Journal: Nat Commun Journal subject: BIOLOGIA / CIENCIA Year: 2021 Document type: Article Affiliation country: Estados Unidos Country of publication: Reino Unido
Fulltext
Add to My VHL
Print
XML
PubMed Links
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Lysosomal Storage Diseases Limits: Animals / Humans Language: En Journal: Nat Commun Journal subject: BIOLOGIA / CIENCIA Year: 2021 Document type: Article Affiliation country: Estados Unidos Country of publication: Reino Unido