CL2021002854A1 - Método para la predicción de eficacia del tratamiento de hemoglobinopatía - Google Patents
Método para la predicción de eficacia del tratamiento de hemoglobinopatíaInfo
- Publication number
- CL2021002854A1 CL2021002854A1 CL2021002854A CL2021002854A CL2021002854A1 CL 2021002854 A1 CL2021002854 A1 CL 2021002854A1 CL 2021002854 A CL2021002854 A CL 2021002854A CL 2021002854 A CL2021002854 A CL 2021002854A CL 2021002854 A1 CL2021002854 A1 CL 2021002854A1
- Authority
- CL
- Chile
- Prior art keywords
- modified
- individual
- treatment
- hemoglobinopathy
- population
- Prior art date
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- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
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- A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
- A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
- A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
- A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
- A61K31/7076—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines containing purines, e.g. adenosine, adenylic acid
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- A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
- A61K38/1774—Immunoglobulin superfamily (e.g. CD2, CD4, CD8, ICAM molecules, B7 molecules, Fc-receptors, MHC-molecules)
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Abstract
La presente invención se refiere a un método para el tratamiento de hemoglobinopatía en un individuo, que comprende: (a) un paso de evaluación: el paso de evaluación comprende evaluar la capacidad de una primera población de células madre hematopoyéticas positivas a CD34 modificadas/células progenitoras para producir un nivel deseado de ?-globina o hemoglobina fetal después de la diferenciación, HSPCs positivas a CD34 modificadas de la primera población se derivan del individuo y que se modifican para reducir la función de BCL11A; y (b) un paso de tratamiento: el paso de tratamiento comprende administrar al individuo una segunda población de HSPCs positivas a CD34 modificadas, las HSPCs positivas a CD34 modificadas se derivan del individuo y se modifican para reducir la función de BCL11A; al mismo tiempo, la invención también se refiere a un método para el tratamiento de hemoglobinopatía en individuos, un método para seleccionar individuos que sufren de hemoglobinopatía para tratamiento utilizando HSPCs positivas a CD34 modificadas de la segunda población, y un método para determinar si un individuo que sufre de hemoglobinopatía es adecuado o inadecuado para tratamiento utilizando la segunda población de HSPCs positivas a CD34 modificadas derivadas del individuo y modificadas para reducir la función de BCL11A.
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
CN2019085116 | 2019-04-30 |
Publications (1)
Publication Number | Publication Date |
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CL2021002854A1 true CL2021002854A1 (es) | 2022-08-12 |
Family
ID=73028604
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
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CL2021002854A CL2021002854A1 (es) | 2019-04-30 | 2021-10-29 | Método para la predicción de eficacia del tratamiento de hemoglobinopatía |
Country Status (18)
Country | Link |
---|---|
US (1) | US20220193142A1 (es) |
EP (1) | EP3964579A4 (es) |
JP (1) | JP2022531269A (es) |
KR (1) | KR20220002565A (es) |
CN (1) | CN113785059A (es) |
AU (1) | AU2020265060A1 (es) |
BR (1) | BR112021021744A8 (es) |
CA (1) | CA3138029A1 (es) |
CL (1) | CL2021002854A1 (es) |
CO (1) | CO2021016058A2 (es) |
CR (1) | CR20210591A (es) |
EA (1) | EA202192931A1 (es) |
IL (1) | IL287672A (es) |
MA (1) | MA55817A (es) |
MX (1) | MX2021013363A (es) |
PE (1) | PE20220003A1 (es) |
SG (1) | SG11202112072QA (es) |
WO (1) | WO2020221291A1 (es) |
Families Citing this family (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20220307020A1 (en) | 2019-04-15 | 2022-09-29 | Edigene Inc. | Methods and compositions for editing rnas |
CN117778385A (zh) | 2019-07-12 | 2024-03-29 | 北京大学 | 采用工程化rna利用内源adar进行靶向的rna编辑 |
CN117120622A (zh) * | 2021-04-02 | 2023-11-24 | 上海科技大学 | 用于治疗β-血红蛋白病的基因疗法 |
Family Cites Families (10)
Publication number | Priority date | Publication date | Assignee | Title |
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KR101833589B1 (ko) * | 2012-02-24 | 2018-03-02 | 프레드 헛친슨 켄서 리서치 센터 | 이상혈색소증 치료를 위한 조성물 및 방법 |
DE202013012242U1 (de) | 2012-05-25 | 2016-02-02 | Emmanuelle Charpentier | Zusammensetzungen für die durch RNA gesteuerte Modifikation einer Ziel-DNA und für die durch RNA gesteuerte Modulation der Transkription |
CN105188767A (zh) | 2012-07-25 | 2015-12-23 | 布罗德研究所有限公司 | 可诱导的dna结合蛋白和基因组干扰工具及其应用 |
DE202013012597U1 (de) | 2012-10-23 | 2017-11-21 | Toolgen, Inc. | Zusammensetzung zum Spalten einer Ziel-DNA, umfassend eine für die Ziel-DNA spezifische guide-RNA und eine Cas-Protein-codierende Nukleinsäure oder ein Cas-Protein, sowie deren Verwendung |
CN109554350B (zh) * | 2012-11-27 | 2022-09-23 | 儿童医疗中心有限公司 | 用于胎儿血红蛋白再诱导的靶向bcl11a远端调控元件 |
US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
WO2016135557A2 (en) * | 2015-02-23 | 2016-09-01 | Crispr Therapeutics Ag | Materials and methods for treatment of hemoglobinopathies |
BR112018013065A2 (pt) * | 2015-12-28 | 2018-12-11 | Intellia Therapeutics Inc | composições e métodos para o tratamento de hemoglobinopatias |
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 |
WO2018218135A1 (en) * | 2017-05-25 | 2018-11-29 | The Children's Medical Center Corporation | Bcl11a guide delivery |
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2020
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- 2020-04-29 WO PCT/CN2020/087766 patent/WO2020221291A1/zh active Application Filing
- 2020-04-29 MA MA055817A patent/MA55817A/fr unknown
- 2020-04-29 KR KR1020217038930A patent/KR20220002565A/ko not_active Application Discontinuation
- 2020-04-29 SG SG11202112072QA patent/SG11202112072QA/en unknown
- 2020-04-29 US US17/607,796 patent/US20220193142A1/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 PE PE2021001804A patent/PE20220003A1/es unknown
- 2020-04-29 EP EP20798490.7A patent/EP3964579A4/en not_active Withdrawn
- 2020-04-29 CA CA3138029A patent/CA3138029A1/en active Pending
- 2020-04-29 CN CN202080028977.8A patent/CN113785059A/zh active Pending
- 2020-04-29 EA EA202192931A patent/EA202192931A1/ru unknown
- 2020-04-29 MX MX2021013363A patent/MX2021013363A/es unknown
- 2020-04-29 BR BR112021021744A patent/BR112021021744A8/pt not_active Application Discontinuation
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EA202192931A1 (ru) | 2022-02-22 |
KR20220002565A (ko) | 2022-01-06 |
WO2020221291A1 (zh) | 2020-11-05 |
BR112021021744A2 (es) | 2021-12-28 |
CN113785059A (zh) | 2021-12-10 |
PE20220003A1 (es) | 2022-01-05 |
MX2021013363A (es) | 2021-11-18 |
AU2020265060A1 (en) | 2021-12-16 |
JP2022531269A (ja) | 2022-07-06 |
CO2021016058A2 (es) | 2021-12-10 |
CR20210591A (es) | 2022-03-11 |
EP3964579A4 (en) | 2023-07-26 |
IL287672A (en) | 2021-12-01 |
CA3138029A1 (en) | 2020-11-05 |
BR112021021744A8 (pt) | 2023-03-21 |
SG11202112072QA (en) | 2021-12-30 |
US20220193142A1 (en) | 2022-06-23 |
MA55817A (fr) | 2022-03-09 |
EP3964579A1 (en) | 2022-03-09 |
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