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Increasing the Specificity of AAV-Based Gene Editing through Self-Targeting and Short-Promoter Strategies.
Breton, Camilo; Furmanak, Thomas; Avitto, Alexa N; Smith, Melanie K; Latshaw, Caitlin; Yan, Hanying; Greig, Jenny A; Wilson, James M.
Afiliação
  • Breton C; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
  • Furmanak T; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
  • Avitto AN; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
  • Smith MK; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
  • Latshaw C; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
  • Yan H; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
  • Greig JA; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
  • Wilson JM; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA. Electronic address: wilsonjm@upenn.edu.
Mol Ther ; 29(3): 1047-1056, 2021 03 03.
Article em En | MEDLINE | ID: mdl-33359790

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Regiões Promotoras Genéticas / Dependovirus / Endonucleases / Edição de Genes / Vetores Genéticos / Inibidores de PCSK9 / Lipoproteínas LDL Limite: Animals / Humans Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Estados Unidos

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Regiões Promotoras Genéticas / Dependovirus / Endonucleases / Edição de Genes / Vetores Genéticos / Inibidores de PCSK9 / Lipoproteínas LDL Limite: Animals / Humans Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Estados Unidos