論文 - 詳細
| RRC ID | 58135 |
|---|---|
| 著者 | Morishige S, Mizuno S, Ozawa H, Nakamura T, Mazahery A, Nomura K, Seki R, Mouri F, Osaki K, Yamamura K, Okamura T, Nagafuji K. |
| タイトル | CRISPR/Cas9-mediated gene correction in hemophilia B patient-derived iPSCs. |
| ジャーナル | Int J Hematol |
| Abstract |
The clustered regulatory interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) system is an efficient genome-editing tool that holds potential for gene therapy. Here, we report an application of this system for gene repair in hemophilia B (HB) using induced pluripotent stem cells (iPSCs). We prepared targeting plasmids with homology arms containing corrected sequences to repair an in-frame deletion in exon 2 of the factor IX (F9) gene and transfected patient-derived iPSCs with the Cas9 nuclease and a guide RNA expression vector. To validate the expression of corrected F9, we attempted to induce the differentiation of iPSCs toward hepatocyte-like cells (HLCs) in vitro. We successfully repaired a disease-causing mutation in HB in patient-derived iPSCs. The transcription product of corrected F9 was confirmed in HLCs differentiated from gene-corrected iPSCs. Although further research should be undertaken to obtain completely functional hepatocytes with secretion of coagulation factor IX, our study provides a proof-of-principle for HB gene therapy using the CRISPR/Cas9 system. |
| 巻・号 | 111(2) |
| ページ | 225-233 |
| 公開日 | 2020-2-1 |
| DOI | 10.1007/s12185-019-02765-0 |
| PII | 10.1007/s12185-019-02765-0 |
| PMID | 31664646 |
| MeSH | CRISPR-Cas Systems / genetics* Gene Editing* Genetic Therapy / methods* Hemophilia B / genetics* Hemophilia B / therapy* Humans Induced Pluripotent Stem Cells* |
| IF | 2.245 |
| 引用数 | 1 |
| オルトメトリクス指標 |
オルトメトリクス指標項目
|
| 最多言及媒体 | X(Twitter) |
| 各媒体での言及数の合計 | 2 |
| 過去6か月間でのオルトメトリクス指標の変動値 | 0.0 |
| リソース情報 | |
| ヒト・動物細胞 | 201B7(HPS0063) |