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Single AAV-mediated CRISPR-Nme2Cas9 efficiently reduces mutant hTTR expression in a transgenic mouse model of transthyretin amyloidosis |
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Titel: |
Single AAV-mediated CRISPR-Nme2Cas9 efficiently reduces mutant hTTR expression in a transgenic mouse model of transthyretin amyloidosis |
Auteur: |
Wen, Jinkun Cao, Tianqi Wu, Jinni Chen, Yuxi Zhi, Shengyao Huang, Yanming Zhen, Peilin Wu, Guanglan Aagaard, Lars Zhong, Jianxin Liang, Puping Huang, Junjiu |
Verschenen in: |
Molecular therapy |
Paginering: |
Jaargang 30 () nr. 1 pagina's 164-174 |
Jaar: |
2022 |
Inhoud: |
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Uitgever: |
The American Society of Gene and Cell Therapy |
Bronbestand: |
Elektronische Wetenschappelijke Tijdschriften |
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