Single AAV-mediated CRISPR/Nme2Cas9 efficiently reduces mutant hTTR expression in a transgenic mouse model of transthyretin amyloidosis.
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L. Aagaard | Junjiu Huang | Puping Liang | Yuxi Chen | Tianqi Cao | Yanming Huang | Jinkun Wen | Jinni Wu | Jianxin Zhong | Shengyao Zhi | Peilin Zhen | Guanglan Wu