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Publication

Promoterless, Nuclease-Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia.

Randy J Chandler | Leah E Venturoni | Jing Liao | Brandon T Hubbard | Jessica L Schneller | Victoria Hoffmann | Susana Gordo | Shengwen Zang | Chih-Wei Ko | Nelson Chau | Kyle Chiang | Mark A Kay | Adi Barzel | Charles P Venditti
Hepatology (Baltimore, Md.) | 2021

Adeno-associated viral (AAV) gene therapy has shown great promise as an alternative treatment for metabolic disorders managed using liver transplantation, but remains limited by transgene loss and genotoxicity. Our study aims to test an AAV vector with a promoterless integrating cassette, designed to provide sustained hepatic transgene expression and reduced toxicity in comparison to canonical AAV therapy.

Pubmed ID: 32976669

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Associated grants

  • Agency: NHLBI NIH HHS, United States
    Id: R01 HL064274
  • Agency: Intramural NIH HHS, United States
    Id: ZIA HG200318

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RRID:SCR_008986

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