MX2022012985A - Metodo y farmaco para tratar el sindrome de hurler. - Google Patents
Metodo y farmaco para tratar el sindrome de hurler.Info
- 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
- Authority
- MX
- Mexico
- Prior art keywords
- hurler syndrome
- drug
- nucleic acid
- acid drug
- rna
- Prior art date
Links
- 239000003814 drug Substances 0.000 title abstract 4
- 238000000034 method Methods 0.000 title abstract 4
- 208000015178 Hurler syndrome Diseases 0.000 title abstract 3
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 title abstract 3
- 229940079593 drug Drugs 0.000 title 1
- 102000039446 nucleic acids Human genes 0.000 abstract 3
- 108020004707 nucleic acids Proteins 0.000 abstract 3
- 150000007523 nucleic acids Chemical class 0.000 abstract 3
- OIRDTQYFTABQOQ-KQYNXXCUSA-N adenosine Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O OIRDTQYFTABQOQ-KQYNXXCUSA-N 0.000 abstract 2
- FDGQSTZJBFJUBT-UHFFFAOYSA-N hypoxanthine Chemical compound O=C1NC=NC2=C1NC=N2 FDGQSTZJBFJUBT-UHFFFAOYSA-N 0.000 abstract 2
- 102100035028 Alpha-L-iduronidase Human genes 0.000 abstract 1
- 239000002126 C01EB10 - Adenosine Substances 0.000 abstract 1
- 101001019502 Homo sapiens Alpha-L-iduronidase Proteins 0.000 abstract 1
- UGQMRVRMYYASKQ-UHFFFAOYSA-N Hypoxanthine nucleoside Natural products OC1C(O)C(CO)OC1N1C(NC=NC2=O)=C2N=C1 UGQMRVRMYYASKQ-UHFFFAOYSA-N 0.000 abstract 1
- 229960005305 adenosine Drugs 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 238000001727 in vivo Methods 0.000 abstract 1
- 230000035772 mutation Effects 0.000 abstract 1
- 230000001717 pathogenic effect Effects 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-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
- C12N15/1137—Non-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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
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- C—CHEMISTRY; METALLURGY
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/52—Genes encoding for enzymes or proenzymes
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
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- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01076—L-Iduronidase (3.2.1.76)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
- C12Y305/04004—Adenosine deaminase (3.5.4.4)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C—CHEMISTRY; METALLURGY
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
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- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
- C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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.
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 |
Family
ID=78084766
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2022012985A MX2022012985A (es) | 2020-04-15 | 2021-04-15 | Metodo y farmaco para tratar el sindrome de hurler. |
Country Status (14)
Country | Link |
---|---|
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)
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)
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 |
-
2021
- 2021-04-15 BR BR112022020795A patent/BR112022020795A2/pt unknown
- 2021-04-15 AU AU2021257508A patent/AU2021257508A1/en active Pending
- 2021-04-15 CA CA3180019A patent/CA3180019A1/en active Pending
- 2021-04-15 PE PE2022002249A patent/PE20230703A1/es unknown
- 2021-04-15 MX MX2022012985A patent/MX2022012985A/es unknown
- 2021-04-15 US US17/918,877 patent/US20230242916A1/en active Pending
- 2021-04-15 JP JP2022562893A patent/JP2023521487A/ja active Pending
- 2021-04-15 WO PCT/CN2021/087534 patent/WO2021209010A1/zh unknown
- 2021-04-15 TW TW110113627A patent/TW202204621A/zh unknown
- 2021-04-15 IL IL297203A patent/IL297203A/en unknown
- 2021-04-15 KR KR1020227038310A patent/KR20220162168A/ko not_active Application Discontinuation
- 2021-04-15 EP EP21787537.6A patent/EP4137161A4/en active Pending
- 2021-04-15 CN CN202180028449.7A patent/CN115697420A/zh active Pending
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2022
- 2022-10-13 CO CONC2022/0014527A patent/CO2022014527A2/es unknown
Also Published As
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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