IL314793A - Antisense-induced exon2 inclusion in acid alpha-glucosidase - Google Patents

Antisense-induced exon2 inclusion in acid alpha-glucosidase

Info

Publication number: IL314793A
Authority: IL; Israel
Prior art keywords: group; alkyl; hydrogen; aralkyl; seq
Prior art date: 2013-09-05

Application number

IL314793A

Other languages

English (en)

Hebrew (he)

Original Assignee

Sarepta Therapeutics Inc

Univ Murdoch

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.)

2013-09-05

Filing date

2014-09-05

Publication date

2024-10-01

2014-09-05 Application filed by Sarepta Therapeutics Inc, Univ Murdoch filed Critical Sarepta Therapeutics Inc

2024-10-01 Publication of IL314793A publication Critical patent/IL314793A/en

Links

230000000692 anti-sense effect Effects 0.000 title claims 8
102100024295 Maltase-glucoamylase Human genes 0.000 title 1
239000002253 acid Substances 0.000 title 1
108010028144 alpha-Glucosidases Proteins 0.000 title 1
229910052739 hydrogen Inorganic materials 0.000 claims 48
239000001257 hydrogen Substances 0.000 claims 46
150000002431 hydrogen Chemical class 0.000 claims 33
125000003710 aryl alkyl group Chemical group 0.000 claims 30
230000008685 targeting Effects 0.000 claims 22
ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical group O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims 20
RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims 20
150000001875 compounds Chemical class 0.000 claims 16
125000002252 acyl group Chemical group 0.000 claims 13
125000003729 nucleotide group Chemical group 0.000 claims 12
229940113082 thymine Drugs 0.000 claims 10
229940035893 uracil Drugs 0.000 claims 10
-1 C 1-C 6 alkyl Chemical group 0.000 claims 9
UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims 9
125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims 9
239000002773 nucleotide Substances 0.000 claims 9
101001018026 Homo sapiens Lysosomal alpha-glucosidase Proteins 0.000 claims 7
125000000217 alkyl group Chemical group 0.000 claims 7
229910052799 carbon Inorganic materials 0.000 claims 7
102000045921 human GAA Human genes 0.000 claims 7
108020004999 messenger RNA Proteins 0.000 claims 7
108090000623 proteins and genes Proteins 0.000 claims 7
108010051109 Cell-Penetrating Peptides Proteins 0.000 claims 6
102000020313 Cell-Penetrating Peptides Human genes 0.000 claims 6
108091093037 Peptide nucleic acid Proteins 0.000 claims 6
QVGXLLKOCUKJST-UHFFFAOYSA-N atomic oxygen Chemical compound [O] QVGXLLKOCUKJST-UHFFFAOYSA-N 0.000 claims 6
125000002915 carbonyl group Chemical group [*:2]C([*:1])=O 0.000 claims 6
230000000295 complement effect Effects 0.000 claims 6
229910052736 halogen Inorganic materials 0.000 claims 6
150000002367 halogens Chemical class 0.000 claims 6
125000002887 hydroxy group Chemical group [H]O* 0.000 claims 6
238000000034 method Methods 0.000 claims 6
229910052757 nitrogen Inorganic materials 0.000 claims 6
125000004433 nitrogen atom Chemical group N* 0.000 claims 6
229910052760 oxygen Inorganic materials 0.000 claims 6
239000001301 oxygen Substances 0.000 claims 6
239000008194 pharmaceutical composition Substances 0.000 claims 6
125000001997 phenyl group Chemical group [H]C1=C([H])C([H])=C(*)C([H])=C1[H] 0.000 claims 6
125000001424 substituent group Chemical group 0.000 claims 6
125000002221 trityl group Chemical group [H]C1=C([H])C([H])=C([H])C([H])=C1C([*])(C1=C(C(=C(C(=C1[H])[H])[H])[H])[H])C1=C([H])C([H])=C([H])C([H])=C1[H] 0.000 claims 6
125000004435 hydrogen atom Chemical group [H]* 0.000 claims 4
150000003839 salts Chemical class 0.000 claims 4
125000003545 alkoxy group Chemical group 0.000 claims 3
125000003282 alkyl amino group Chemical group 0.000 claims 3
125000004429 atom Chemical group 0.000 claims 3
108020004707 nucleic acids Proteins 0.000 claims 3
150000007523 nucleic acids Chemical class 0.000 claims 3
102000039446 nucleic acids Human genes 0.000 claims 3
PTMHPRAIXMAOOB-UHFFFAOYSA-L phosphoramidate Chemical compound NP([O-])([O-])=O PTMHPRAIXMAOOB-UHFFFAOYSA-L 0.000 claims 3
125000004193 piperazinyl group Chemical group 0.000 claims 3
239000000126 substance Substances 0.000 claims 3
RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 3
208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 claims 2
206010053185 Glycogen storage disease type II Diseases 0.000 claims 2
OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims 2
239000003937 drug carrier Substances 0.000 claims 2
201000004502 glycogen storage disease II Diseases 0.000 claims 2
UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims 2
FDGQSTZJBFJUBT-UHFFFAOYSA-N hypoxanthine Chemical compound O=C1NC=NC2=C1NC=N2 FDGQSTZJBFJUBT-UHFFFAOYSA-N 0.000 claims 2
LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical compound CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims 1
MSSXOMSJDRHRMC-UHFFFAOYSA-N 9H-purine-2,6-diamine Chemical compound NC1=NC(N)=C2NC=NC2=N1 MSSXOMSJDRHRMC-UHFFFAOYSA-N 0.000 claims 1
GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Chemical compound NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims 1
229930024421 Adenine Natural products 0.000 claims 1
UGQMRVRMYYASKQ-UHFFFAOYSA-N Hypoxanthine nucleoside Natural products OC1C(O)C(CO)OC1N1C(NC=NC2=O)=C2N=C1 UGQMRVRMYYASKQ-UHFFFAOYSA-N 0.000 claims 1
229960000643 adenine Drugs 0.000 claims 1
150000001413 amino acids Chemical class 0.000 claims 1
229940104302 cytosine Drugs 0.000 claims 1
150000003230 pyrimidines Chemical class 0.000 claims 1

Classifications

- 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
- C12N15/113—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
- 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
- C12N15/113—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
- A61K31/66—Phosphorus compounds
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
- 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
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
- A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
- C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
- C07F9/02—Phosphorus compounds
- C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
- C07F9/6527—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom having nitrogen and oxygen atoms as the only ring hetero atoms
- C07F9/6533—Six-membered rings
- C—CHEMISTRY; METALLURGY
- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
- C—CHEMISTRY; METALLURGY
- 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
- C12N2310/314—Phosphoramidates
- C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
- C—CHEMISTRY; METALLURGY
- 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
- C12N2310/315—Phosphorothioates
- C—CHEMISTRY; METALLURGY
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
- C12N2310/3181—Peptide nucleic acid, PNA
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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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
- C—CHEMISTRY; METALLURGY
- 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
- C12N2310/32—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
- C—CHEMISTRY; METALLURGY
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
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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
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3513—Protein; Peptide
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- 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)
- C12Y302/0102—Alpha-glucosidase (3.2.1.20)

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Health & Medical Sciences (AREA)
Life Sciences & Earth Sciences (AREA)
Chemical & Material Sciences (AREA)
Engineering & Computer Science (AREA)
Genetics & Genomics (AREA)
Organic Chemistry (AREA)
Molecular Biology (AREA)
General Health & Medical Sciences (AREA)
Biomedical Technology (AREA)
Bioinformatics & Cheminformatics (AREA)
Biochemistry (AREA)
Wood Science & Technology (AREA)
Biotechnology (AREA)
General Engineering & Computer Science (AREA)
Zoology (AREA)
Public Health (AREA)
Pharmacology & Pharmacy (AREA)
Veterinary Medicine (AREA)
Animal Behavior & Ethology (AREA)
Medicinal Chemistry (AREA)
Diabetes (AREA)
Biophysics (AREA)
Epidemiology (AREA)
Microbiology (AREA)
Plant Pathology (AREA)
Physics & Mathematics (AREA)
Virology (AREA)
Chemical Kinetics & Catalysis (AREA)
General Chemical & Material Sciences (AREA)
Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
Obesity (AREA)
Hematology (AREA)
Endocrinology (AREA)
Emergency Medicine (AREA)
Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Medicinal Preparation (AREA)
Saccharide Compounds (AREA)

IL314793A 2013-09-05 2014-09-05 Antisense-induced exon2 inclusion in acid alpha-glucosidase IL314793A (en)

Applications Claiming Priority (3)

Application Number	Priority Date	Filing Date	Title
US201361874261P	2013-09-05	2013-09-05
US201461932195P	2014-01-27	2014-01-27
PCT/US2014/054384 WO2015035231A1 (en)	2013-09-05	2014-09-05	Antisense-induced exon2 inclusion in acid alpha-glucosidase

Publications (1)

Publication Number	Publication Date
IL314793A true IL314793A (en)	2024-10-01

Family

ID=51541398

Family Applications (4)

Application Number	Title	Priority Date	Filing Date
IL314793A IL314793A (en)	2013-09-05	2014-09-05	Antisense-induced exon2 inclusion in acid alpha-glucosidase
IL282239A IL282239B2 (en)	2013-09-05	2014-09-05	Antisense-induced exon2 inclusion in acid alpha-glucosidase
IL300444A IL300444A (en)	2013-09-05	2014-09-05	Inclusion of exon 2 is induced by antisense in acid alpha-glucosidase
IL244334A IL244334B (en)	2013-09-05	2016-02-29	Inclusion of exon 2 is induced by antisense in acid alpha-glucosidase

Family Applications After (3)

Application Number	Title	Priority Date	Filing Date
IL282239A IL282239B2 (en)	2013-09-05	2014-09-05	Antisense-induced exon2 inclusion in acid alpha-glucosidase
IL300444A IL300444A (en)	2013-09-05	2014-09-05	Inclusion of exon 2 is induced by antisense in acid alpha-glucosidase
IL244334A IL244334B (en)	2013-09-05	2016-02-29	Inclusion of exon 2 is induced by antisense in acid alpha-glucosidase

Country Status (11)

Country	Link
US (3)	US11236338B2 (2)
EP (1)	EP3041935A1 (2)
JP (3)	JP6618910B2 (2)
KR (2)	KR102275504B1 (2)
CN (2)	CN111235149B (2)
AU (2)	AU2014317961B2 (2)
CA (1)	CA2922838A1 (2)
IL (4)	IL314793A (2)
MX (1)	MX384377B (2)
TW (1)	TWI736514B (2)
WO (1)	WO2015035231A1 (2)

Families Citing this family (26)

* Cited by examiner, † Cited by third party
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US20130085139A1 (en)	2011-10-04	2013-04-04	Royal Holloway And Bedford New College	Oligomers
US11236338B2 (en)	2013-09-05	2022-02-01	Sarepta Therapeutics, Inc.	Antisense-induced exon2 inclusion in acid alpha-glucosidase
EP3044315B1 (en) *	2013-09-11	2019-02-20	Synthena AG	Nucleic acids and methods for the treatment of pompe disease
KR102524543B1 (ko)	2014-06-10	2023-04-20	에라스무스 유니버시티 메디컬 센터 로테르담	폼페병의 치료에 유용한 안티센스 올리고뉴클레오티드
GB201410693D0 (en)	2014-06-16	2014-07-30	Univ Southampton	Splicing modulation
AU2015327836B2 (en)	2014-10-03	2021-07-01	Cold Spring Harbor Laboratory	Targeted augmentation of nuclear gene output
EP3262056A4 (en) *	2015-02-27	2018-09-19	Sarepta Therapeutics, Inc.	Antisense-induced exon2 inclusion in acid alpha-glucosidase
MA41795A (fr) *	2015-03-18	2018-01-23	Sarepta Therapeutics Inc	Exclusion d'un exon induite par des composés antisens dans la myostatine
EP3858993A1 (en) *	2015-10-09	2021-08-04	Sarepta Therapeutics, Inc.	Compositions and methods for treating duchenne muscular dystrophy and related disorders
CN108603230A (zh)	2015-10-09	2018-09-28	南安普敦大学	基因表达的调节与蛋白质表达失调的筛选
WO2017099579A1 (en) *	2015-12-07	2017-06-15	Erasmus University Medical Center Rotterdam	Enzymatic replacement therapy and antisense therapy for pompe disease
US11096956B2 (en)	2015-12-14	2021-08-24	Stoke Therapeutics, Inc.	Antisense oligomers and uses thereof
WO2017106377A1 (en)	2015-12-14	2017-06-22	Cold Spring Harbor Laboratory	Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
WO2017184529A1 (en) *	2016-04-18	2017-10-26	Sarepta Therapeutics, Inc.	Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
NL2017295B1 (en) *	2016-08-05	2018-02-14	Univ Erasmus Med Ct Rotterdam	Antisense oligomeric compound for Pompe disease
NL2017294B1 (en)	2016-08-05	2018-02-14	Univ Erasmus Med Ct Rotterdam	Natural cryptic exon removal by pairs of antisense oligonucleotides.
KR102810425B1 (ko) *	2016-12-19	2025-05-21	사렙타 쎄러퓨틱스 인코퍼레이티드	근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
KR102318434B1 (ko)	2017-08-25	2021-11-01	스톡 테라퓨틱스, 인크.	병태 및 질환 치료용 안티센스 올리고머
NL2019517B1 (en) *	2017-09-08	2019-03-19	Univ Erasmus Med Ct Rotterdam	New therapy for Pompe disease
WO2019060862A1 (en) *	2017-09-25	2019-03-28	Sarepta Therapeutics, Inc.	METHODS FOR PREPARING MORPHOLINO PHOSPHORODIAMIDATE OLIGOMERS VIA FAST FLOW SYNTHESIS
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TWI736514B (zh)	2021-08-21
IL282239B2 (en)	2023-10-01
JP2016533761A (ja)	2016-11-04
JP6671449B2 (ja)	2020-03-25
IL282239B1 (en)	2023-06-01
KR20210088009A (ko)	2021-07-13
CN105793422A (zh)	2016-07-20
AU2020204409A1 (en)	2020-07-23
IL244334A0 (en)	2016-04-21
IL282239A (en)	2021-05-31
WO2015035231A1 (en)	2015-03-12
CA2922838A1 (en)	2015-03-12
IL244334B (en)	2021-04-29
AU2014317961B2 (en)	2020-07-30
CN105793422B (zh)	2020-03-03
JP2020089392A (ja)	2020-06-11
US20220170024A1 (en)	2022-06-02
US20160208264A1 (en)	2016-07-21
JP6618910B2 (ja)	2019-12-11
US20150197534A1 (en)	2015-07-16
JP6865871B2 (ja)	2021-04-28
EP3041935A1 (en)	2016-07-13
CN111235149B (zh)	2024-04-16
IL300444A (en)	2023-04-01
KR102275504B1 (ko)	2021-07-09
JP2019050828A (ja)	2019-04-04
MX2016002934A (es)	2016-12-20
KR20160082972A (ko)	2016-07-11
AU2014317961A1 (en)	2016-03-17
AU2020204409B2 (en)	2023-05-25
TW201605881A (zh)	2016-02-16
MX384377B (es)	2025-03-14
CN111235149A (zh)	2020-06-05
US11236338B2 (en)	2022-02-01

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