MX384479B - Oligonucleótidos antisentido para saltar un exón aberrante en cep290 asociado con la amaurosis congénita de leber. - Google Patents

Oligonucleótidos antisentido para saltar un exón aberrante en cep290 asociado con la amaurosis congénita de leber.

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Publication number
MX384479B
MX384479B MX2017011013A MX2017011013A MX384479B MX 384479 B MX384479 B MX 384479B MX 2017011013 A MX2017011013 A MX 2017011013A MX 2017011013 A MX2017011013 A MX 2017011013A MX 384479 B MX384479 B MX 384479B
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MX
Mexico
Prior art keywords
antisense oligonucleotides
skip
cep290
congenital amaurosis
leber congenital
Prior art date
Application number
MX2017011013A
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English (en)
Other versions
MX2017011013A (es
Inventor
Hee Lam Chan
Patricia Coromoto Biasutto
Original Assignee
Proqr Therapeutics Ii Bv
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Publication date
Application filed by Proqr Therapeutics Ii Bv filed Critical Proqr Therapeutics Ii Bv
Publication of MX2017011013A publication Critical patent/MX2017011013A/es
Publication of MX384479B publication Critical patent/MX384479B/es

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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
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  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

La presente invención se refiere a oligonucleótidos antisentído enfocan la mutación en intrón 26 del gene CEP290 y reducen la inclusión del exón aberrante en el mRNA de CEP290. Los oligonucleótidos incluyen no más de 3 guanosinas consecutivas, no tienen más de 60% de nucleobases de guanosina, incluyen cuando mucho una secuencia de CpG y/o no tienen el potencial de formar una horquilla que comprende 3 o más pares de bases complementarios.
MX2017011013A 2015-02-27 2016-02-26 Oligonucleótidos antisentido para saltar un exón aberrante en cep290 asociado con la amaurosis congénita de leber. MX384479B (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GB201503408A GB201503408D0 (en) 2015-02-27 2015-02-27 Oligonucleotides
PCT/EP2016/054164 WO2016135334A1 (en) 2015-02-27 2016-02-26 Oligonucleotide therapy for leber congenital amaurosis

Publications (2)

Publication Number Publication Date
MX2017011013A MX2017011013A (es) 2018-03-06
MX384479B true MX384479B (es) 2025-03-14

Family

ID=52876291

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MX2017011013A MX384479B (es) 2015-02-27 2016-02-26 Oligonucleótidos antisentido para saltar un exón aberrante en cep290 asociado con la amaurosis congénita de leber.

Country Status (14)

Country Link
US (3) US10421963B2 (es)
EP (1) EP3262170A1 (es)
JP (1) JP6813506B2 (es)
KR (1) KR102368918B1 (es)
CN (1) CN107406852B (es)
AU (1) AU2016223336B2 (es)
CA (1) CA2976601C (es)
EA (1) EA036791B1 (es)
GB (1) GB201503408D0 (es)
IL (1) IL253882B (es)
MX (1) MX384479B (es)
NZ (1) NZ735342A (es)
WO (1) WO2016135334A1 (es)
ZA (1) ZA201705331B (es)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
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GB201503408D0 (en) 2015-02-27 2015-04-15 Proqr Therapeutics N V Oligonucleotides
CN109477103A (zh) 2016-06-22 2019-03-15 ProQR治疗上市公司Ⅱ 单链rna-编辑寡核苷酸
EP3507366B1 (en) 2016-09-01 2020-10-07 ProQR Therapeutics II B.V. Chemically modified single-stranded rna-editing oligonucleotides
GB201706009D0 (en) 2017-04-13 2017-05-31 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of stargardt disease
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
EP3840758A1 (en) * 2018-08-21 2021-06-30 Deep Genomics Incorporated Therapeutic splice-switching oligonucleotides
US12398393B2 (en) 2019-01-28 2025-08-26 Proqr Therapeutics Ii B.V. RNA-editing oligonucleotides for the treatment of usher syndrome
WO2020157014A1 (en) 2019-01-28 2020-08-06 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of leber's congenital amaurosis
US20220177894A1 (en) 2019-04-02 2022-06-09 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for immunotherapy
WO2020212567A1 (en) 2019-04-18 2020-10-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of usher syndrome
EP3959315A1 (en) * 2019-04-26 2022-03-02 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment of retinal dystrophies by exon-skipping strategy
WO2020254249A1 (en) 2019-06-21 2020-12-24 Proqr Therapeutics Ii B.V. Delivery of nucleic acids for the treatment of auditory disorders
CN115038789A (zh) 2019-12-02 2022-09-09 塑造治疗公司 治疗性编辑
EP4081638A1 (en) 2019-12-23 2022-11-02 ProQR Therapeutics II B.V. Antisense oligonucleotides for nucleotide deamination in the treatment of stargardt disease
US20230134677A1 (en) 2020-03-04 2023-05-04 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for use in the treatment of usher syndrome
WO2022090256A1 (en) 2020-10-26 2022-05-05 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of stargardt disease
EP4301856A2 (en) 2021-03-05 2024-01-10 ProQR Therapeutics II B.V. Antisense oligonucleotides for use in the treatment of corneal dystrophies
CN113736812A (zh) * 2021-09-06 2021-12-03 武汉翼康基因科技有限公司 一种pcMINI载体及其构建方法与应用
US20250154504A1 (en) 2022-02-14 2025-05-15 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia

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Also Published As

Publication number Publication date
JP6813506B2 (ja) 2021-01-13
KR102368918B1 (ko) 2022-02-28
CA2976601C (en) 2023-03-21
WO2016135334A1 (en) 2016-09-01
JP2018507711A (ja) 2018-03-22
CN107406852B (zh) 2021-06-11
US20200063134A1 (en) 2020-02-27
KR20170118755A (ko) 2017-10-25
AU2016223336A1 (en) 2017-09-28
US20180030439A1 (en) 2018-02-01
AU2016223336B2 (en) 2021-12-09
NZ735342A (en) 2022-07-01
BR112017018335A2 (pt) 2018-04-17
MX2017011013A (es) 2018-03-06
EA036791B1 (ru) 2020-12-22
US10421963B2 (en) 2019-09-24
CA2976601A1 (en) 2016-09-01
US10889817B2 (en) 2021-01-12
IL253882A0 (en) 2017-10-31
IL253882B (en) 2022-03-01
EP3262170A1 (en) 2018-01-03
US11920132B2 (en) 2024-03-05
US20210139905A1 (en) 2021-05-13
ZA201705331B (en) 2019-07-31
EA201791820A1 (ru) 2017-12-29
GB201503408D0 (en) 2015-04-15
CN107406852A (zh) 2017-11-28

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