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 modificadas

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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
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BR
Brazil
Prior art keywords
modified
hemoglobinopathy
population
positive
inappropriate
Prior art date
Application number
BR112021021744A
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English (en)
Portuguese (pt)
Other versions
BR112021021744A2 (enExample
Inventor
Fang Riguo
Yu Lingling
Yang Huihui
Original Assignee
Edigene Inc
Edigene Guangzhou 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.)
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Publication date
Application filed by Edigene Inc, Edigene Guangzhou Inc filed Critical Edigene Inc
Publication of BR112021021744A2 publication Critical patent/BR112021021744A2/pt
Publication of BR112021021744A8 publication Critical patent/BR112021021744A8/pt

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BR112021021744A 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 BR112021021744A8 (pt)

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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BR112021021744A2 BR112021021744A2 (enExample) 2021-12-28
BR112021021744A8 true BR112021021744A8 (pt) 2023-03-21

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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)
CR (1) CR20210591A (enExample)
EA (1) EA202192931A1 (enExample)
IL (1) IL287672A (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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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
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BR112021021744A2 (enExample) 2021-12-28
AU2020265060A1 (en) 2021-12-16
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