RNA METHODS AND COMPOSITIONS FOR TARGETED GENETIC MODIFICATION USING PAIRED GUIDE RNAs

• Main Authors: FRENDEWEY DAVID, MURPHY ANDREW J, YANCOPOULOS GEORGE D, LAI KA MAN VENUS, DROGUETT GUSTAVO, GAGLIARDI ANTHONY, MACDONALD LYNN, AUERBACH WOJTEK, VORONINA VERA, VALENZUELA DAVID M
• Format: Patent
• Language: English; Korean
• Published: 08.09.2017
• Subjects: AGRICULTURE; ANIMAL HUSBANDRY; BEER; BIOCHEMISTRY; CARE OF BIRDS, FISHES, INSECTS; CHEMISTRY; COMPOSITIONS OR TEST PAPERS THEREFOR; COMPOSITIONS THEREOF; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL ORENZYMOLOGICAL PROCESSES; CULTURE MEDIA; ENZYMOLOGY; FISHING; FORESTRY; HUMAN NECESSITIES; HUNTING; MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEICACIDS OR MICROORGANISMS; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; NEW BREEDS OF ANIMALS; PROCESSES OF PREPARING SUCH COMPOSITIONS; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; SPIRITS; TRAPPING; VINEGAR; WINE

세포 내의 게놈에 대한 이중대립유전자 표적화된 변형을 생성하고 촉진하기 위한, 그리고 변형된 게놈을 포함하는 비인간 동물을 생성하기 위한 조성물 및 방법이 제공된다. 또한, 대립유전자에 대해 이형접합성인 세포 내의 게놈을 세포가 그러한 대립유전자에 대해 동형접합성이 되도록 변형시키기 위한 조성물 및 방법이 제공된다. 본 방법은 상기 게놈 표적 유전자좌 내의 상이한 위치를 표적화하는 2개 이상의 가이드 RNA 및 Cas 단백질을 사용한다. 또한, 변형된 게놈을 갖는 세포를 확인하는 방법이 제공된다. Compositions and methods are provided for creating and promoting biallelic targeted modifications to genomes within cells and for producing non-human animals comprising the modified genomes. Also provided are compositions and methods for modifying a genome within a cell that is heterozygous for an allele to become homozygous for that allele. The methods make use of Cas proteins and two or more guide RNAs that target different locations within the same genomic target locus. Also provided are methods of identifying cells with modified genomes.