CRISPR/Cas9-Mediated Gene Correction in Newborn Rabbits with Hereditary Tyrosinemia Type I

Patients with hereditary tyrosinemia type I (HT1) present acute and irreversible liver and kidney damage during infancy. CRISPR-Cas9-mediated gene correction during infancy may provide a promising approach to treat patients with HT1. However, all previous studies were performed on adult HT1 rodent m...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Li, Nan, Gou, Shixue, Wang, Jiaowei, Zhang, Quanjun, Huang, Xingyun, Xie, Jingke, Li, Li, Jin, Qin, Ouyang, Zhen, Chen, Fangbing, Ge, Weikai, Shi, Hui, Liang, Yanhui, Zhuang, Zhenpeng, Zhao, Xiaozhu, Lian, Meng, Ye, Yinghua, Quan, Longquan, Wu, Han, Lai, Liangxue, Wang, Kepin
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2021
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7934638/
https://ncbi.nlm.nih.gov/pubmed/33221434
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.11.023
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