Protospacer modification improves base editing of a canonical splice site variant and recovery of CFTR function in human airway epithelial cells

Autor: Joynt, Anya T., Kavanagh, Erin W., Newby, Gregory A., Mitchell, Shakela, Eastman, Alice C., Paul, Kathleen C., Bowling, Alyssa D., Osorio, Derek L., Merlo, Christian A., Patel, Shivani U., Raraigh, Karen S., Liu, David R., Sharma, Neeraj, Cutting, Garry R.

Publikováno v: In Molecular Therapy - Nucleic Acids June 2023

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Establishment of a conditionally reprogrammed primary eccrine sweat gland culture for evaluation of tissue-specific CFTR function.

Autor: Eastman AC; Johns Hopkins School of Medicine, Department of Human Genetics, Miller Research Building, 733N Broadway, Baltimore, MD 21205, USA., Rosson G; Johns Hopkins Medicine, Department of Plastic and Reconstructive Surgery, 601N Caroline St, Baltimore, MD 21287, USA., Kim N; Johns Hopkins School of Medicine, Department Medicine, Division of Dermatology, 601N Caroline St, Baltimore, MD 21287, USA., Kang S; Johns Hopkins School of Medicine, Department Medicine, Division of Dermatology, 601N Caroline St, Baltimore, MD 21287, USA., Raraigh K; Johns Hopkins School of Medicine, Department of Human Genetics, Miller Research Building, 733N Broadway, Baltimore, MD 21205, USA., Goff LA; Johns Hopkins School of Medicine, Department of Human Genetics, Miller Research Building, 733N Broadway, Baltimore, MD 21205, USA., Merlo C; Johns Hopkins University School of Medicine, Department of Medicine, Division of Pulmonary and Critical Care Medicine, 1800 Orleans St, Baltimore, MD 21287, USA., Lechtzin N; Johns Hopkins University School of Medicine, Department of Medicine, Division of Pulmonary and Critical Care Medicine, 1800 Orleans St, Baltimore, MD 21287, USA., Cutting GR; Johns Hopkins School of Medicine, Department of Human Genetics, Miller Research Building, 733N Broadway, Baltimore, MD 21205, USA., Sharma N; Johns Hopkins School of Medicine, Department of Human Genetics, Miller Research Building, 733N Broadway, Baltimore, MD 21205, USA. Electronic address: nsharma5@jhmi.edu.

Publikováno v: Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society [J Cyst Fibros] 2024 Jul 04. Date of Electronic Publication: 2024 Jul 04.