TWI851534B - 用於治療α-１抗胰蛋白酶缺乏症之組合物及方法 - Google Patents
用於治療α-１抗胰蛋白酶缺乏症之組合物及方法 Download PDFInfo
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| EP3850088A4 (en) | 2018-09-07 | 2023-07-19 | Beam Therapeutics, Inc. | Compositions and methods for improving base editing |
| WO2020082047A1 (en) | 2018-10-18 | 2020-04-23 | Intellia Therapeutics, Inc. | Compositions and methods for treating alpha-1 antitrypsin deficiencey |
| MX2021004278A (es) * | 2018-10-18 | 2021-09-08 | Intellia Therapeutics Inc | Composiciones y metodos para la expresion de transgenes a partir de un locus de albumina. |
| EP3867380A2 (en) | 2018-10-18 | 2021-08-25 | Intellia Therapeutics, Inc. | Compositions and methods for expressing factor ix |
| US20220160847A1 (en) * | 2020-11-20 | 2022-05-26 | Mark Egly | Method of preventing and/or treating a plurality of diseases |
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| AU2014281028B2 (en) * | 2013-06-17 | 2020-09-10 | Massachusetts Institute Of Technology | Delivery and use of the CRISPR-Cas systems, vectors and compositions for hepatic targeting and therapy |
| EP4012031A1 (en) | 2013-07-03 | 2022-06-15 | Dicerna Pharmaceuticals, Inc. | Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded rna |
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| EP3386550B1 (en) * | 2015-12-07 | 2021-01-20 | Arc Bio, LLC | Methods for the making and using of guide nucleic acids |
| EP3433364A1 (en) * | 2016-03-25 | 2019-01-30 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency |
| CN109475646A (zh) * | 2016-03-30 | 2019-03-15 | 因特利亚治疗公司 | 用于crispr/cas成分的脂质纳米颗粒制剂 |
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Non-Patent Citations (2)
| Title |
|---|
| 期刊 Cory Smith et al., Efficient and allele-specific genome editing of disease loci in human iPSCs. Molecular Therapy. 23(3): 2015 Mar; Epub 2014 Nov 24. 570-577.;期刊 Kosuke Yusa et al., Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells. Nature. 478(7369): doi: 10.1038/nature10424. 2011 Oct 12; 391-396. * |
| 期刊 Kosuke Yusa et al., Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells. Nature. 478(7369): doi: 10.1038/nature10424. 2011 Oct 12; 391-396. |
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