PBASE PROTEIN, FUSION PROTEIN, NUCLEIC ACID, GENE INTEGRATION SYSTEM AND USE

WIPO Patent Application WO/2024/094224

A2

Provided in the present invention are a PBase protein, a fusion protein, a nucleic acid, a gene integration system, a gene delivery system and the use. The PBase protein retains the abilities of binding to the ITR sequences of a Piggybac transposon and cleaving the Piggybac transposon to make the Piggybac transposon separate from a donor plasmid in a linear manner; and the PBase protein has a lower ability for binding to the double strands of DNA within 10 bp upstream and downstream of "TTAA". When the PBase protein is linked to a site-specific DNA nuclease to prepare the fusion protein, the PBase protein can be efficiently integrated with a long fragment gene of interest at the double strand breaks generated by the site-specific DNA nuclease, thereby achieving the effect of site-directed insertion of the long fragment gene.

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XU NAIQING (CN)
LIU JINGXIAN (CN)
XU TIAN (CN)

PCT/CN2023/138225

May 10, 2024

December 12, 2023

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GECELL THERAPEUTICS LLC (CN)

C12N9/12; A61K38/45; A61K38/46; A61K48/00; A61P17/00; C07K19/00; C12N9/22; C12N15/54; C12N15/55; C12N15/62; C12N15/85

SHANGHAI BESHINING LAW OFFICE (CN)

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