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In vivo genome editing for hemophilia B therapy by the combination of rebalancing and therapeutic gene knockin using a viral and non-viral vector.
Lee, Jeong Hyeon; Han, Jeong Pil; Song, Dong Woo; Lee, Geon Seong; Choi, Beom Seok; Kim, MinJeong; Lee, Yeji; Kim, Seokjoong; Lee, Hyukjin; Yeom, Su Cheong.
Afiliação
  • Lee JH; Graduate School of International Agricultural Technology and Institute of GreenBio Science and Technology, Seoul National University, 1447 Pyeongchang-Ro, Daewha, Pyeongchang, Kangwon 25354, Korea.
  • Han JP; Graduate School of International Agricultural Technology and Institute of GreenBio Science and Technology, Seoul National University, 1447 Pyeongchang-Ro, Daewha, Pyeongchang, Kangwon 25354, Korea.
  • Song DW; Aavatar Therapeutics, Misadaero, Hanam, Gyeonggi-do, 12925, Korea.
  • Lee GS; Graduate School of International Agricultural Technology and Institute of GreenBio Science and Technology, Seoul National University, 1447 Pyeongchang-Ro, Daewha, Pyeongchang, Kangwon 25354, Korea.
  • Choi BS; Toolgen, Inc., 172 Magokjungang-Ro, Gangseo, Seoul 07788, Korea.
  • Kim M; Department of Biochemistry, Simmons Comprehensive Cancer Center, The University of Texas Southwestern Medical Center, Dallas, TX, USA.
  • Lee Y; College of Pharmacy, Graduate School of Pharmaceutical Sciences, Ewha Womans University, Seoul, 03760, Korea.
  • Kim S; Toolgen, Inc., 172 Magokjungang-Ro, Gangseo, Seoul 07788, Korea.
  • Lee H; College of Pharmacy, Graduate School of Pharmaceutical Sciences, Ewha Womans University, Seoul, 03760, Korea.
  • Yeom SC; Graduate School of International Agricultural Technology and Institute of GreenBio Science and Technology, Seoul National University, 1447 Pyeongchang-Ro, Daewha, Pyeongchang, Kangwon 25354, Korea.
Mol Ther Nucleic Acids ; 32: 161-172, 2023 Jun 13.
Article em En | MEDLINE | ID: mdl-37064777
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Mol Ther Nucleic Acids Ano de publicação: 2023 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Mol Ther Nucleic Acids Ano de publicação: 2023 Tipo de documento: Article