CN114222816A - 一种高效修复环状铁粒幼细胞性贫血基因突变的方法 - Google Patents
一种高效修复环状铁粒幼细胞性贫血基因突变的方法 Download PDFInfo
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Abstract
提供一种利用基因编辑技术高效修复导致环状铁粒幼红细胞性贫血的ALAS‑2基因突变的方法，其包含利用基因编辑技术，高效安全地基因修饰患者造血干细胞的ALAS‑2基因特异点突变，恢复ALAS‑2因表达，使亚铁血红素合成以及红细胞成熟恢复至正常水平，实现治疗疾病的目的。
Description
PCT国内申请，说明书已公开。
Claims (25)
- PCT国内申请，权利要求书已公开。
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CNPCT/CN2019/103235 | 2019-08-29 | ||
| CN2019103235 | 2019-08-29 | ||
| PCT/CN2020/112227 WO2021037234A1 (zh) | 2019-08-29 | 2020-08-28 | 一种高效修复环状铁粒幼细胞性贫血基因突变的方法 |
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|---|---|
| CN114222816A true CN114222816A (zh) | 2022-03-22 |
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| CN202080056107.1A Pending CN114222816A (zh) | 2019-08-29 | 2020-08-28 | 一种高效修复环状铁粒幼细胞性贫血基因突变的方法 |
Country Status (2)
| Country | Link |
|---|---|
| CN (1) | CN114222816A (zh) |
| WO (1) | WO2021037234A1 (zh) |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2002010454A2 (en) * | 2000-07-28 | 2002-02-07 | Genaissance Pharmaceuticals, Inc. | Haplotypes of the alas2 gene |
| CN107949641A (zh) * | 2015-06-17 | 2018-04-20 | Uab研究基金会 | 用于基因组编辑的crispr/cas9复合物 |
| CN109735574A (zh) * | 2017-10-27 | 2019-05-10 | 博雅辑因（北京）生物科技有限公司 | 一种提高胎儿血红蛋白表达的方法 |
Family Cites Families (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11123430B2 (en) * | 2015-11-04 | 2021-09-21 | Acceleron Pharma Inc. | Methods for increasing red blood cell levels and treating ineffective erythropoiesis |
-
2020
- 2020-08-28 WO PCT/CN2020/112227 patent/WO2021037234A1/zh active Application Filing
- 2020-08-28 CN CN202080056107.1A patent/CN114222816A/zh active Pending
Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2002010454A2 (en) * | 2000-07-28 | 2002-02-07 | Genaissance Pharmaceuticals, Inc. | Haplotypes of the alas2 gene |
| CN107949641A (zh) * | 2015-06-17 | 2018-04-20 | Uab研究基金会 | 用于基因组编辑的crispr/cas9复合物 |
| CN109735574A (zh) * | 2017-10-27 | 2019-05-10 | 博雅辑因（北京）生物科技有限公司 | 一种提高胎儿血红蛋白表达的方法 |
| CN109735497A (zh) * | 2017-10-27 | 2019-05-10 | 博雅辑因（北京）生物科技有限公司 | 一种提高胎儿血红蛋白表达水平的方法 |
Non-Patent Citations (1)
| Title |
|---|
| ZHANG，Y.C.等: "Intron 1 GATA site enhances ALAS2 expression indispensably during erythroid differentiation" * |
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| WO2021037234A1 (zh) | 2021-03-04 |
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