MX2022012985A - Metodo y farmaco para tratar el sindrome de hurler. - Google Patents

Metodo y farmaco para tratar el sindrome de hurler.

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Publication number
MX2022012985A
MX2022012985A MX2022012985A MX2022012985A MX2022012985A MX 2022012985 A MX2022012985 A MX 2022012985A MX 2022012985 A MX2022012985 A MX 2022012985A MX 2022012985 A MX2022012985 A MX 2022012985A MX 2022012985 A MX2022012985 A MX 2022012985A
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Mexico
Prior art keywords
hurler syndrome
drug
nucleic acid
acid drug
rna
Prior art date
Application number
MX2022012985A
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English (en)
Inventor
Pengfei Yuan
Yanxia Zhao
Nengyin Liu
Zexuan Yi
Gangbin Tang
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Edigene Therapeutics Beijing Inc
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Publication date
Application filed by Edigene Therapeutics Beijing Inc filed Critical Edigene Therapeutics Beijing Inc
Publication of MX2022012985A publication Critical patent/MX2022012985A/es

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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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Abstract

Se divulgan un fármaco de ácido nucleico basado en la técnica LEAPER y un método para tratar enfermedades tales como el síndrome de Hurler mediante el uso del fármaco de ácido nucleico para dirigir y editar ARN; el método comprende: realizar una edición de la base de adenosina a la base de hipoxantina en el ARN usando el fármaco de ácido nucleico para reparar con precisión el sitio de mutación patógena de G > A de, por ejemplo, el síndrome de Hurler; recuperando así la expresión normal in vivo de una proteína codificada por ARN, tal como IDUA.
MX2022012985A 2020-04-15 2021-04-15 Metodo y farmaco para tratar el sindrome de hurler. MX2022012985A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
CN2020084925 2020-04-15
PCT/CN2021/087534 WO2021209010A1 (zh) 2020-04-15 2021-04-15 一种治疗赫勒氏综合征的方法和药物

Publications (1)

Publication Number Publication Date
MX2022012985A true MX2022012985A (es) 2022-11-09

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MX2022012985A MX2022012985A (es) 2020-04-15 2021-04-15 Metodo y farmaco para tratar el sindrome de hurler.

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US (1) US20230242916A1 (es)
EP (1) EP4137161A4 (es)
JP (1) JP2023521487A (es)
KR (1) KR20220162168A (es)
CN (1) CN115697420A (es)
AU (1) AU2021257508A1 (es)
BR (1) BR112022020795A2 (es)
CA (1) CA3180019A1 (es)
CO (1) CO2022014527A2 (es)
IL (1) IL297203A (es)
MX (1) MX2022012985A (es)
PE (1) PE20230703A1 (es)
TW (1) TW202204621A (es)
WO (1) WO2021209010A1 (es)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4069842A1 (en) 2019-12-02 2022-10-12 Shape Therapeutics Inc. Therapeutic editing
WO2023143538A1 (zh) * 2022-01-28 2023-08-03 北京辑因医疗科技有限公司 基于leaper技术治疗mpsi的方法和组合物
WO2023152371A1 (en) 2022-02-14 2023-08-17 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
WO2024013361A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Oligonucleotides for adar-mediated rna editing and use thereof
WO2024013360A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Chemically modified oligonucleotides for adar-mediated rna editing

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6049623B2 (ja) * 2010-10-22 2016-12-21 カッパーアールエヌエー,インコーポレイテッド α‐L‐イズロニダーゼ(IDUA)への天然アンチセンス転写物の阻害によるIDUA関連疾患の治療
CN107532162A (zh) * 2014-12-12 2018-01-02 托德·M·伍尔夫 用于利用寡核苷酸编辑细胞中核酸的组合物和方法
WO2017220751A1 (en) * 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
CN110352244B (zh) 2016-09-01 2023-03-21 ProQR治疗上市公司Ⅱ 化学修饰的编辑rna的单链寡核苷酸
WO2019060746A1 (en) * 2017-09-21 2019-03-28 The Broad Institute, Inc. SYSTEMS, METHODS, AND COMPOSITIONS FOR THE TARGETED EDITING OF NUCLEIC ACIDS
EP3752611A1 (en) * 2018-02-14 2020-12-23 ProQR Therapeutics II B.V. Antisense oligonucleotides for rna editing
KR20210076082A (ko) * 2018-10-12 2021-06-23 페킹 유니버시티 Rna를 편집하기 위한 방법 및 조성물
CR20210572A (es) * 2019-04-15 2022-04-07 Univ Beijing Métodos y composiciones para editar ácido ribonucleicos (arn)
AU2020313143B2 (en) * 2019-07-12 2024-04-04 Peking University Targeted RNA editing by leveraging endogenous adar using engineered RNAs
CN114846139A (zh) * 2019-12-30 2022-08-02 北京辑因医疗科技有限公司 一种基于leaper技术治疗mps ih的方法和组合物

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Publication number Publication date
CN115697420A (zh) 2023-02-03
PE20230703A1 (es) 2023-04-24
TW202204621A (zh) 2022-02-01
AU2021257508A1 (en) 2022-11-17
WO2021209010A1 (zh) 2021-10-21
CA3180019A1 (en) 2021-10-21
BR112022020795A2 (pt) 2022-11-29
IL297203A (en) 2022-12-01
JP2023521487A (ja) 2023-05-24
KR20220162168A (ko) 2022-12-07
EP4137161A1 (en) 2023-02-22
EP4137161A4 (en) 2023-10-11
US20230242916A1 (en) 2023-08-03
CO2022014527A2 (es) 2022-11-08

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