Supramolecular nanosubstrate-mediated delivery system enables CRISPR-Cas9 knockin of hemoglobin beta gene for hemoglobinopathies
文献类型： 外文期刊
作者： Yang, Peng 1 ; Chou, Shih-Jie 3 ; Li, Jindian 5 ; Hui, Wenqiao 6 ; Liu, Wenfei 7 ; Sun, Na 2 ; Zhang, Ryan Y. 2 ; Zhu, Ya 1 ;
作者机构： 1.Anhui Univ, Ctr Stem Cell & Translat Med, Sch Life Sci, Hefei 230601, Anhui, Peoples R China
2.Univ Calif Los Angeles, Crump Inst Mol Imaging CIMI, Calif NanoSyst Inst CNSI, Dept Mol & Med Pharmacol, Los Angeles, CA 90095 USA
3.Taipei Vet Gen Hosp, Dept Med Res, Div Basic Res, Taipei, Taiwan
4.Natl Yang Ming Univ, Sch Med, Inst Pharmacol, Taipei, Taiwan
5.Univ Southern Calif, Mol Imaging Ctr, Keck Sch Med, Dept Radiol, Los Angeles, CA 90033 USA
6.Anhui Acad Agr Sci, Inst Anim Husb & Vet Med, Hefei 230031, Peoples R China
7.Univ Calif Los Angeles, Dept Mat Sci & Engn, Dept Chem & Biochem, Dept Bioengn, Los Angeles, CA 90095 USA
8.Univ Calif Los Angeles, Calif NanoSyst Inst CNSI, Los Angeles, CA 90095 USA
9.Univ Calif Los Angeles, Eli & Edythe Broad Ctr Regenerat Med & Stem Cell, Dept Microbiol Immunol & Mol Genet,Jonsson Compre, DGSOM,Div Pediat Hematol Oncol,Dept Pediat,David, Los Angeles, CA 90095 USA
10.Foshan Univ, Sch Stomatol & Med, Foshan 528000, Peoples R China
11.Sun Yat Sen Univ, Affiliated Hosp 1, Inst Precis Med, Dept Pathol, Guangzhou 510080, Peoples R China
12.Southern Univ, Shenzhen Peoples Hosp, Clin Med Res Ctr, Affiliated Hosp 1, Shenzhen 518020, Peoples R China
13.Natl Chung Hsing Univ NCHU, Dept Chem, iCtr Adv Sci & Technol, Innovat & Dev Ctr Sustainable Agr, Xingda Rd,South, Taichung 402, Taiwan
14.Univ Calif Los Angeles, Eli & Edythe Broad Ctr Regenerat Med & Stem Cell, Childrens Discovery & Innovat Inst, Calif Nanosyst Inst CNSI,Dept Pediat,David Geffen, Los Angeles, CA 90095 USA
15.Taipei Vet Gen Hosp, Dept Med Res, Taipei, Taiwan
16.Acad Sinica, Genom Res Ctr, Taipei, Taiwan
期刊名称：SCIENCE ADVANCES （ 影响因子：14.136； 五年影响因子：16.446 ）
ISSN： 2375-2548
年卷期： 2020 年 6 卷 43 期
页码：
收录情况： SCI
摘要： Leveraging the endogenous homology-directed repair (HDR) pathway, the CRISPR-Cas9 gene-editing system can be applied to knock in a therapeutic gene at a designated site in the genome, offering a general therapeutic solution for treating genetic diseases such as hemoglobinopathies. Here, a combined supramolecular nanoparticle (SMNP)/supramolecular nanosubstrate-mediated delivery (SNSMD) strategy is used to facilitate CRISPR-Cas9 knockin of the hemoglobin beta (HBB) gene into the adeno-associated virus integration site 1 (AAVS1) safe-harbor site of an engineered K562 3.21 cell line harboring the sickle cell disease mutation. Through stepwise treatments of the two SMNP vectors encapsulating a Cas9 center dot single-guide RNA (sgRNA) complex and an HBB/green fluorescent protein (GFP)-encoding plasmid, CRISPR-Cas9 knockin was successfully achieved via HDR. Last, the HBB/GFP-knockin K562 3.21 cells were introduced into mice via intraperitoneal injection to show their in vivo proliferative potential. This proof-of-concept demonstration paves the way for general gene therapeutic solutions for treating hemoglobinopathies.
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