Search International and National Patent Collections

1. (WO2017173004) A METHOD FOR IN VIVO PRECISE GENOME EDITING

Pub. No.:    WO/2017/173004    International Application No.:    PCT/US2017/024869
Publication Date: Fri Oct 06 01:59:59 CEST 2017 International Filing Date: Thu Mar 30 01:59:59 CEST 2017
IPC: A01K 67/027
C12N 9/22
C12N 15/90
Applicants: MIKUNI, Takayasu
NISHIYAMA, Jun
YASUDA, Ryohei
Inventors: MIKUNI, Takayasu
NISHIYAMA, Jun
YASUDA, Ryohei
Title: A METHOD FOR IN VIVO PRECISE GENOME EDITING
Abstract:
The present invention relates to a method for in vivo genome editing, comprising: (a) delivering a genome editing machinery (e.g. plasmid vectors encoding a Cas9 nuclease and a single-guide RNA; as well as a donor template polynucleotide), to mitotic progenitor cells of a non-human embryo through in utero electroporation; and (b) modifying at least one target sequence (i.e. a DNA sequence within the genome of the progenitor cells) by using homology-directed repair (HDR). The genome editing machinery may further comprise a control of transfection, comprising a transposon, and a polynucleotide encoding a transposase. Another aspect of the invention relates to a non-human embryo or a non-human animal carrying a modified target sequence in its genome, wherein said non-human embryo or non-human animal is produced by the in vivo genome editing method of the invention. A further aspect of the in vivo genome editing method of the invention relates to a modified cell obtained from said non-human embryo or from said non-human animal.