Titlebook: Base Editors; Methods and Protocol Sangsu Bae,Beomjong Song Book 2023 The Editor(s) (if applicable) and The Author(s), under exclusive lice

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https://doi.org/10.1007/978-3-030-28475-6 inherited retinal disease. The majority of retinal genome editing requires intravitreal and subretinal injection delivery of the therapeutic vector in order to transduce the target cells. Here, we provide an application guide of base editor as performed in the mouse retina.

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Use of the Representative Base Editing Tool Target-AID to Introduce Pathogenic Mutations into Micete genetically modified animals that harbor disease-causing pathogenic point mutations. In this chapter, I describe the basic protocol used to introduce disease-relevant pathogenic mutations into mice by Target-AID.

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Targeted Mutagenesis in Mice Using a Base Editorools for introducing point mutations, such as C-to-T and A-to-G conversions. The enhanced base editor, a C-to-G base editor (CGBE), can perform other nucleotide substitutions, such as C-to-G conversions. Here, we introduce a method for generating mouse models with point mutations using a base editing system.

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Ex Vivo Base Editing Therapy with Chemically Derived Hepatic Progenitorss a safe and efficient strategy for ex vivo gene therapy. Here, we described how to generate hepatic progenitors from terminally differentiated hepatocytes, deliver base/prime editors into the cells, select corrected hepatic progenitors, and transplant them into mice of inborn error of metabolism.

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Application of Base Editor-Mediated Genome Editing in Mouse Retina inherited retinal disease. The majority of retinal genome editing requires intravitreal and subretinal injection delivery of the therapeutic vector in order to transduce the target cells. Here, we provide an application guide of base editor as performed in the mouse retina.

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