Dong Woo Song
Publications 3
#1Dong Hyun Jo (Seoul National University Hospital)H-Index: 13
#2Dong Woo SongH-Index: 1
Last.Jungmin Lee (Handong Global University)H-Index: 4
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Leber congenital amaurosis (LCA), one of the leading causes of childhood-onset blindness, is caused by autosomal recessive mutations in several genes including RPE65. In this study, we performed CRISPR-Cas9–mediated therapeutic correction of a disease-associated nonsense mutation in Rpe65 in rd12 mice, a model of human LCA. Subretinal injection of adeno-associated virus carrying CRISPR-Cas9 and donor DNA resulted in >1% homology-directed repair and ~1.6% deletion of the pathogenic stop codon in ...
#2Ji-Su LeeH-Index: 4
Last.Hee Sook BaeH-Index: 1
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#1Eunji KimH-Index: 15
#2Taeyoung KooH-Index: 15
Last.Jin-Soo KimH-Index: 53
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The amount of genetic material that can be packaged in AAV vectors used for genome editing is limited. Here the authors show that the smallest known Cas9 orthologue, cjCas9, can be packaged in a single AAV vector along with sgRNA and a marker gene, and demonstrate efficient gene editing in mice.
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