Improved specificity and efficiency of in vivo adenine base editing therapies with hybrid guide RNAs.
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| Title: | Improved specificity and efficiency of in vivo adenine base editing therapies with hybrid guide RNAs. |
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| Authors: | Whittaker MN; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Department of Bioengineering, University of Pennsylvania, Philadelphia, Pennsylvania, USA.; These authors contributed equally: Madelynn N. Whittaker, Lauren C. Testa., Testa LC; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; These authors contributed equally: Madelynn N. Whittaker, Lauren C. Testa., Brooks DL; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA., Quigley A; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA., Said H; Department of Pathology and Laboratory Medicine, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.; Department of Bioengineering, George Mason University, Fairfax, Virginia, USA., Dwivedi G; Division of Infectious Diseases, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Penn Institute for RNA Innovation, Perelman School of Medicine, University of Pennsylvania; Philadelphia, Pennsylvania, USA., Jindal I; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA., Volpp D; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA., Qu P; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA., Wang JZ; Agilent Technologies, Inc., Santa Clara, California, USA., Levine MA; Division of Endocrinology and Diabetes, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA., Ahrens-Nicklas RC; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Division of Human Genetics and Metabolism, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA., Li Q; Department of Biochemistry and Molecular Biology, Sidney Kimmel Medical College, Jefferson Institute of Molecular Medicine, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.; PXE International Center of Excellence in Research and Clinical Care, Thomas Jefferson University, Philadelphia, PA, USA., Musunuru K; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Division of Human Genetics and Metabolism, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.; These authors jointly directed this work: Kiran Musunuru, Mohamad-Gabriel Alameh, William H. Peranteau, and Xiao Wang., Alameh MG; Department of Pathology and Laboratory Medicine, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.; Department of Bioengineering, George Mason University, Fairfax, Virginia, USA.; Penn Institute for RNA Innovation, Perelman School of Medicine, University of Pennsylvania; Philadelphia, Pennsylvania, USA.; Department of Pathology and Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; These authors jointly directed this work: Kiran Musunuru, Mohamad-Gabriel Alameh, William H. Peranteau, and Xiao Wang., Peranteau WH; The Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.; Division of Pediatric General, Thoracic, and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.; These authors jointly directed this work: Kiran Musunuru, Mohamad-Gabriel Alameh, William H. Peranteau, and Xiao Wang., Wang X; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.; These authors jointly directed this work: Kiran Musunuru, Mohamad-Gabriel Alameh, William H. Peranteau, and Xiao Wang. |
| Source: | BioRxiv : the preprint server for biology [bioRxiv] 2025 Apr 09. Date of Electronic Publication: 2025 Apr 09. |
| Publication Type: | Journal Article; Preprint |
| Journal Info: | Country of Publication: United States NLM ID: 101680187 Publication Model: Electronic Cited Medium: Internet ISSN: 2692-8205 (Electronic) Linking ISSN: 26928205 NLM ISO Abbreviation: bioRxiv Subsets: PubMed not MEDLINE |
| Database: | MEDLINE Ultimate |
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