Gene Therapy
Combination Therapies and Personalized Medicine
RNA Splicing
Alternative RNA Splicing
Conservative Site-specific Recombination and Phase Variation
Single Nucleotide Polymorphisms-SNPs
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Jinkuk Kim1,2,3,4, Sijae Woo5, Claudio M de Gusmao6,7
1Graduate School of Medical Science and Engineering, Korea Advanced Institute of Science and Technology (KAIST), Daejeon, Republic of Korea. jinkuk@kaist.ac.kr.
全基因组测序确定了具有遗传性疾病的个体,比如 - 角质切换,易受拼接切换抗意义寡核酸 (ASO) 的影响. 这些ASO成功地纠正了患者细胞的拼接缺陷,并在临床试验中显示出安全性.
10:06Engineering Artificial Factors to Specifically Manipulate Alternative Splicing in Human Cells
Published on: April 26, 2017
10:07A Standard Methodology to Examine On-site Mutagenicity As a Function of Point Mutation Repair Catalyzed by CRISPR/Cas9 and SsODN in Human Cells
Published on: August 25, 2017
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