Article
Correction of the pathogenic mutation in TGM1 gene by adenine base editing in mutant embryos.
Molecular therapy : the journal of the American Society of Gene Therapy - 5 Jan 2022
Dang Lu, Zhou Xueliang, Zhong Xiufang, Yu Wenxia, Huang Shisheng, Liu Hanyan, Chen Yuanyuan, Zhang Wuwen, Yuan Lihua, Li Lei, Huang Xingxu, Li Guanglei, Liu Jianqiao, Tong Guoqing
Abstract excerpt
A couple diagnosed as carriers for lamellar ichthyosis, an autosomal recessive rare disease, encountered two pregnancy losses. Their blood samples showed the same heterozygous c.607C>T mutation in the TGM1 gene. However, we found that about 98.4% of the sperm had mutations, suggesting possible de novo germline mutation. To explore the probability of correcting this mutation, we used two different adenine base...
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