Kit for reparing fbn1t7498c mutation, combination for making and repairing mutation, and method of repairing thereof
AssigneeShanghaitech University
InventorsXingxu HUANG, Guanglei LI, Jianan LI
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Abstract
A reagent and a method for repairing an FBN1T7498C mutation by using base editing. A kit for efficiently repairing the FBN1T7498C mutation is characterized by comprising a base editor and a repair re-sgRNA directed to the FBN1T7498C site. A base editing technology is used to repair the mutation of FBN1T7498C through a precise CT single base mutation, thereby providing a method for treating a Marfan syndrome caused by such mutation.
Claims9
A kit for efficiently repairing an FBN1T7498C mutation, the kit comprising a base editor and a repair re-sgRNA directed to an FBN1T7498C site.
A combination for making a mutation and repairing the mutation, the combination comprising:
- at least one of a mutated mt-sgRNA designed according to an FBN1T7498C site and a correspondingly mutated ssODN, a repair re-sgRNA directed to the FBN1T7498C site, and a base editor.
A method for repairing a mutation by using base editing, the method comprising:
- in an FBN1T7498C-containing mutated cell, using a repair re-sgRNA directed to an FBN1T7498C site to guide a base editor to a mutation site to perform base editing repair and collect transfected cells.
Dependencies are inferred from the claim text.