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PE20220003A1 - Metodo para la prediccion de eficacia del tratamiento de hemoglobinopatia - Google Patents

Metodo para la prediccion de eficacia del tratamiento de hemoglobinopatia

Info

Publication number
PE20220003A1
PE20220003A1 PE2021001804A PE2021001804A PE20220003A1 PE 20220003 A1 PE20220003 A1 PE 20220003A1 PE 2021001804 A PE2021001804 A PE 2021001804A PE 2021001804 A PE2021001804 A PE 2021001804A PE 20220003 A1 PE20220003 A1 PE 20220003A1
Authority
PE
Peru
Prior art keywords
modified
individual
treatment
hemoglobinopathy
population
Prior art date
Application number
PE2021001804A
Other languages
English (en)
Inventor
Riguo Fang
Lingling Yu
Huihui Yang
Original Assignee
Edigene Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Edigene Inc filed Critical Edigene Inc
Publication of PE20220003A1 publication Critical patent/PE20220003A1/es

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Abstract

SE REFIERE A UN METODO PARA EL TRATAMIENTO DE HEMOGLOBINOPATIA EN UN INDIVIDUO, QUE COMPRENDE: A) EVALUAR LA CAPACIDAD DE UNA PRIMERA POBLACION DE LAS CELULAS MADRE HEMATOPOYETICAS POSITIVAS A CD34/CELULAS PROGENITORAS MODIFICADAS (HSPCS POSITIVAS A CD34) PARA PRODUCIR UN NIVEL DESEADO DE GAMMA-GLOBINA O HEMOGLOBINA FETAL (HBF) DESPUES DE LA DIFERENCIACION, EN DONDE LAS HSPCs POSITIVAS A CD34 MODIFICADAS DE LA PRIMERA POBLACION SON DERIVADAS DEL INDIVIDUO Y SON MODIFICADAS PARA REDUCIR LA FUNCION DE BCL11A (CELULAS EV MODIFICADAS); Y B) UN PASO DE TRATAMIENTO, QUE COMPRENDE: ADMINISTRAR UNA SEGUNDA POBLACION DE HSPCs POSITIVAS A CD34 MODIFICADAS AL INDIVIDUO, QUE SON DERIVADAS DEL INDIVIDUO Y SON MODIFICADAS PARA REDUCIR LA FUNCION DE BCL11A.
PE2021001804A 2019-04-30 2020-04-29 Metodo para la prediccion de eficacia del tratamiento de hemoglobinopatia PE20220003A1 (es)

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

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PE20220003A1 true PE20220003A1 (es) 2022-01-05

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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)
PH (1) PH12021552732A1 (es)
SG (1) SG11202112072QA (es)
WO (1) WO2020221291A1 (es)

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JP7698828B2 (ja) 2019-04-15 2025-06-26 北京大学 Rnaを編集する方法および組成物
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.
IL306119A (en) * 2021-04-02 2023-11-01 Univ Shanghai Technology Gene therapy for the treatment of beta-hemoglobinopathies

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EP3272356A1 (en) * 2012-02-24 2018-01-24 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
US20160046961A1 (en) 2012-05-25 2016-02-18 Emmanuelle Charpentier Methods and Compositions for RNA-Directed Target DNA Modification and For RNA-Directed Modulation of Transcription
DK3494997T3 (da) 2012-07-25 2019-12-02 Broad Inst Inc Inducerbare dna-bindende proteiner og værktøjer til genomperturbation samt anvendelser deraf
EP2912175B1 (en) 2012-10-23 2018-08-22 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
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CA2977455A1 (en) * 2015-02-23 2019-09-01 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
NZ783531A (en) * 2015-12-28 2025-09-26 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
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PH12021552732A1 (en) 2023-01-16
EP3964579A1 (en) 2022-03-09
BR112021021744A8 (pt) 2023-03-21
US20220193142A1 (en) 2022-06-23
MX2021013363A (es) 2021-11-18
WO2020221291A1 (zh) 2020-11-05
KR20220002565A (ko) 2022-01-06
CA3138029A1 (en) 2020-11-05
JP2022531269A (ja) 2022-07-06
CL2021002854A1 (es) 2022-08-12
AU2020265060A1 (en) 2021-12-16
CR20210591A (es) 2022-03-11
EA202192931A1 (ru) 2022-02-22
MA55817A (fr) 2022-03-09
EP3964579A4 (en) 2023-07-26
SG11202112072QA (en) 2021-12-30
CN113785059A (zh) 2021-12-10
BR112021021744A2 (es) 2021-12-28
CO2021016058A2 (es) 2021-12-10
IL287672A (en) 2021-12-01

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