MX2021013363A - Metodo para la prediccion de eficacia del tratamiento de hemoglobinopatia. - Google Patents

Metodo para la prediccion de eficacia del tratamiento de hemoglobinopatia.

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Publication number
MX2021013363A
MX2021013363A MX2021013363A MX2021013363A MX2021013363A MX 2021013363 A MX2021013363 A MX 2021013363A MX 2021013363 A MX2021013363 A MX 2021013363A MX 2021013363 A MX2021013363 A MX 2021013363A MX 2021013363 A MX2021013363 A MX 2021013363A
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Mexico
Prior art keywords
modified
individual
hemoglobinopathy
population
treatment
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MX2021013363A
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English (en)
Inventor
Huihui Yang
Riguo Fang
Lingling Yu
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Edigene Guangzhou Inc
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Application filed by Edigene Guangzhou Inc filed Critical Edigene Guangzhou Inc
Publication of MX2021013363A publication Critical patent/MX2021013363A/es

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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.
MX2021013363A 2019-04-30 2020-04-29 Metodo para la prediccion de eficacia del tratamiento de hemoglobinopatia. MX2021013363A (es)

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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

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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)

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PE20220300A1 (es) 2019-07-12 2022-03-09 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
IL306119A (en) * 2021-04-02 2023-11-01 Univ Shanghai Tech Gene therapy for the treatment of beta-hemoglobinopathies

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