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Evaluation of cytosine base editing and adenine base editing as a potential treatment for alpha-1 antitrypsin deficiency

Michael S. Packer, Vivek Chowdhary, Genesis Lung, Lo-I Cheng, Yvonne Aratyn-Schaus, Dominique Leboeuf, Sarah Smith, Aalok Shah, Delai Chen, Marina Zieger, Brian J. Cafferty, Bo Yan, Giuseppe Ciaramella, Francine M. Gregoire, Christian Mueller

2022Molecular Therapy42 citationsDOIOpen Access PDF

Topics & Concepts

CytosineAlpha (finance)Base (topology)Molecular biologyGeneticsBiologyDNAChemistryMedicineMathematicsPatient satisfactionNursingConstruct validityMathematical analysisCRISPR and Genetic EngineeringVirus-based gene therapy researchHerpesvirus Infections and Treatments
Evaluation of cytosine base editing and adenine base editing as a potential treatment for alpha-1 antitrypsin deficiency | Litcius