BR112021021744A8 - Métodos para tratar hemoglobinopatia e para determinar se um indivíduo sofrendo de hemoglobinopatia é adequado ou inadequado para tratar com uma segunda população das hspcs positivas em cd34 modificadas - Google Patents
Métodos para tratar hemoglobinopatia e para determinar se um indivíduo sofrendo de hemoglobinopatia é adequado ou inadequado para tratar com uma segunda população das hspcs positivas em cd34 modificadasInfo
- Publication number
- BR112021021744A8 BR112021021744A8 BR112021021744A BR112021021744A BR112021021744A8 BR 112021021744 A8 BR112021021744 A8 BR 112021021744A8 BR 112021021744 A BR112021021744 A BR 112021021744A BR 112021021744 A BR112021021744 A BR 112021021744A BR 112021021744 A8 BR112021021744 A8 BR 112021021744A8
- Authority
- BR
- Brazil
- Prior art keywords
- modified
- hemoglobinopathy
- population
- positive
- inappropriate
- Prior art date
Links
- 208000034737 hemoglobinopathy Diseases 0.000 title abstract 8
- 238000000034 method Methods 0.000 title abstract 6
- 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 abstract 6
- 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 Proteins 0.000 abstract 6
- 101001105486 Homo sapiens Proteasome subunit alpha type-7 Proteins 0.000 abstract 5
- 102100021201 Proteasome subunit alpha type-7 Human genes 0.000 abstract 5
- 102100022976 B-cell lymphoma/leukemia 11A Human genes 0.000 abstract 3
- 101000903703 Homo sapiens B-cell lymphoma/leukemia 11A Proteins 0.000 abstract 3
- 238000011156 evaluation Methods 0.000 abstract 2
- 210000003958 hematopoietic stem cell Anatomy 0.000 abstract 2
- 102000001554 Hemoglobins Human genes 0.000 abstract 1
- 108010054147 Hemoglobins Proteins 0.000 abstract 1
- 230000004069 differentiation Effects 0.000 abstract 1
- 230000001605 fetal effect Effects 0.000 abstract 1
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Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN2019085116 | 2019-04-30 | ||
| PCT/CN2020/087766 WO2020221291A1 (zh) | 2019-04-30 | 2020-04-29 | 一种血红蛋白病治疗有效性预测方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| BR112021021744A2 BR112021021744A2 (enExample) | 2021-12-28 |
| BR112021021744A8 true BR112021021744A8 (pt) | 2023-03-21 |
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| BR112021021744A BR112021021744A8 (pt) | 2019-04-30 | 2020-04-29 | Métodos para tratar hemoglobinopatia e para determinar se um indivíduo sofrendo de hemoglobinopatia é adequado ou inadequado para tratar com uma segunda população das hspcs positivas em cd34 modificadas |
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| US (1) | US20220193142A1 (enExample) |
| EP (1) | EP3964579A4 (enExample) |
| JP (1) | JP2022531269A (enExample) |
| KR (1) | KR20220002565A (enExample) |
| CN (1) | CN113785059A (enExample) |
| AU (1) | AU2020265060A1 (enExample) |
| BR (1) | BR112021021744A8 (enExample) |
| CA (1) | CA3138029A1 (enExample) |
| CL (1) | CL2021002854A1 (enExample) |
| CO (1) | CO2021016058A2 (enExample) |
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| IL (1) | IL287672A (enExample) |
| MA (1) | MA55817A (enExample) |
| MX (1) | MX2021013363A (enExample) |
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| CR20210572A (es) | 2019-04-15 | 2022-04-07 | Univ Beijing | Métodos y composiciones para editar ácido ribonucleicos (arn) |
| MX2022000497A (es) | 2019-07-12 | 2022-02-03 | Univ Beijing | Edicion de acido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actua sobre acido ribonucleico endogeno (adar) utilizando acidos ribonucleicos (arn) modificados geneticamente. |
| BR112023019773A2 (pt) * | 2021-04-02 | 2024-03-12 | Univ Shanghai Technology | Método para promover a produção de gama-globina em uma célula humana, polinucleotídeos codificando uma proteína associada a um crispr (cas), uma nucleobase desaminase, um rna de guia único (sgrna) e um rna de guia único auxiliar (hsgrna), e, proteína de fusão |
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| SG11201405103SA (en) * | 2012-02-24 | 2014-09-26 | Hutchinson Fred Cancer Res | Compositions and methods for the treatment of hemoglobinopathies |
| MY189533A (en) | 2012-05-25 | 2022-02-16 | Univ California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
| KR20230065381A (ko) | 2012-07-25 | 2023-05-11 | 더 브로드 인스티튜트, 인코퍼레이티드 | 유도 dna 결합 단백질 및 게놈 교란 도구 및 이의 적용 |
| WO2014065596A1 (en) | 2012-10-23 | 2014-05-01 | Toolgen Incorporated | Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof |
| PT3502240T (pt) * | 2012-11-27 | 2021-08-11 | Childrens Medical Ct Corp | Elementos reguladores distais de bcl11a como alvo para a reindução de hemoglobina fetal |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| WO2016135558A2 (en) * | 2015-02-23 | 2016-09-01 | Crispr Therapeutics Ag | Materials and methods for treatment of hemoglobinopathies |
| SG11201805217XA (en) * | 2015-12-28 | 2018-07-30 | Novartis Ag | Compositions and methods for the treatment of hemoglobinopathies |
| TW201839136A (zh) * | 2017-02-06 | 2018-11-01 | 瑞士商諾華公司 | 治療血色素異常症之組合物及方法 |
| US11788087B2 (en) * | 2017-05-25 | 2023-10-17 | The Children's Medical Center Corporation | BCL11A guide delivery |
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2020
- 2020-04-29 WO PCT/CN2020/087766 patent/WO2020221291A1/zh not_active Ceased
- 2020-04-29 KR KR1020217038930A patent/KR20220002565A/ko not_active Withdrawn
- 2020-04-29 CN CN202080028977.8A patent/CN113785059A/zh active Pending
- 2020-04-29 PH PH1/2021/552732A patent/PH12021552732A1/en unknown
- 2020-04-29 BR BR112021021744A patent/BR112021021744A8/pt not_active Application Discontinuation
- 2020-04-29 EP EP20798490.7A patent/EP3964579A4/en not_active Withdrawn
- 2020-04-29 US US17/607,796 patent/US20220193142A1/en not_active Abandoned
- 2020-04-29 AU AU2020265060A patent/AU2020265060A1/en not_active Abandoned
- 2020-04-29 CA CA3138029A patent/CA3138029A1/en active Pending
- 2020-04-29 JP JP2021564630A patent/JP2022531269A/ja active Pending
- 2020-04-29 CR CR20210591A patent/CR20210591A/es unknown
- 2020-04-29 EA EA202192931A patent/EA202192931A1/ru unknown
- 2020-04-29 MX MX2021013363A patent/MX2021013363A/es unknown
- 2020-04-29 SG SG11202112072QA patent/SG11202112072QA/en unknown
- 2020-04-29 MA MA055817A patent/MA55817A/fr unknown
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2021
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- 2021-10-29 CL CL2021002854A patent/CL2021002854A1/es unknown
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|---|---|
| CA3138029A1 (en) | 2020-11-05 |
| WO2020221291A1 (zh) | 2020-11-05 |
| PH12021552732A1 (en) | 2023-01-16 |
| IL287672A (en) | 2021-12-01 |
| EA202192931A1 (ru) | 2022-02-22 |
| MA55817A (fr) | 2022-03-09 |
| JP2022531269A (ja) | 2022-07-06 |
| CN113785059A (zh) | 2021-12-10 |
| US20220193142A1 (en) | 2022-06-23 |
| CR20210591A (es) | 2022-03-11 |
| KR20220002565A (ko) | 2022-01-06 |
| SG11202112072QA (en) | 2021-12-30 |
| PE20220003A1 (es) | 2022-01-05 |
| CL2021002854A1 (es) | 2022-08-12 |
| EP3964579A4 (en) | 2023-07-26 |
| BR112021021744A2 (enExample) | 2021-12-28 |
| AU2020265060A1 (en) | 2021-12-16 |
| MX2021013363A (es) | 2021-11-18 |
| EP3964579A1 (en) | 2022-03-09 |
| CO2021016058A2 (es) | 2021-12-10 |
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