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Transcript of Gn) 7 G#ÝFÔG F¸ 4G $SFþ$S Â0 Â'¼Fû2(FéG …...Kang, X.et al. CRISPR-Cas9 p \J Assist...
Genome Editing ( )
SpCas9
Streptococcus pyogenes
PAM: protospacer adjacent motifsgRNA: single guide RNA
NHEJ: Non Homologous End JoiningHDR: Homology Directed Repair
Nature Review Cancer (2015) 15: 387–395.
Base Editing
C→T
A→G
https://benchling.com/pub/liu-base-editor
Tang, L. et al. CRISPR-Cas9 Mol Reprod Dev 85, 461-463, (2018).
Zhou, C. et al. Base Editing Protein Cell 8, 772-775, (2017).
Tang, L. et al. CRISPR-Cas9 Mol Genet Genomics 292, 525-533, (2017).
Ma, H. et al. CRISPR-Cas9 Nature 548, 413-419, (2017).
Liang, P. et al. Base Editing Protein Cell 8, 811-822, (2017).
Li, G. et al. Base Editing Protein Cell 8, 776-779, (2017).
Fogarty, N. et al. CRISPR-Cas9 Nature 550, 67-73, (2017).
Kang, X. et al. CRISPR-Cas9 J Assist Reprod Genet 33, 581-588, (2016).
Liang, P et al. CRISPR-Cas9 Protein Cell 6, 363-372, (2015).
CRISPR-Cas9 6 1, 1, 4Base Editing 3 3 /9
OCT4
Nature 550, 67-73 (2017)
100
BBChttps://www.bbc.com/news/health-41269200
IVF
Oregon Health & Science University
Nature 548, 414-419 (2017)
WT:Mut: 4bpNHEJ: Mos:
DNAM II DNA
S
>10,000 , 2%Vector A.McKusick , Mendelian Inheritance in Man
14, 1-9 (2004)
VIII, IX
5000 1 1 .
VIII IX2
https://www.hemophilia-navi.jp/disease/index.html
1
NF1
1/3000 .
http://pathology.or.jp/corepictures2010/20/c06/01.html
BBChttps://www.bbc.com/news/uk-england-leicestershire-36478601
1862 –1890
Ray Crundwell / QMUL
huntingtin
5~6 /100 . 1
30~40 . 10~20
https://labs.uab.edu/mccgray/research-areas/huntington-s-disease
NATURE CELL BIOLOGY 18, 700-708 (2016)NATURE 533, 251-254 (2016)
Nature 564, 183-185 (2018)
Human Reproduction 31, 312–323 2016 Dev Cell 38, 621-34 (2016)
NATURE 522, 221-225 (2016)
IFITM1: interferon-induced transmembrane protein 1
HRVK: Human endogenous retrovirus K
Cell 160, 253-268 (2015)Dev Cell 39, 169-185 (2016)
SOX17ES
aminion
(NCB: Nuffield Council on Bioethics)
1991 Nuffield1994
Medical Research CouncilWellcome Trust
BBC https://www.bbc.com/news/health-44849034
,
‘heritable genome editing interventions’,
, , ,
Nuffield Council on Bioethicshttp://nuffieldbioethics.org/project/genome-editing-human-reproduction
CRISPR-Cas9 Base Editor
, 1, 7
10,000
2018 7
