JP2021522836A - スプライスオパシーの軽減、及びｒｎａドミナンス障害の治療のための組成物及び方法 - Google Patents

スプライスオパシーの軽減、及びｒｎａドミナンス障害の治療のための組成物及び方法 Download PDF

Info

Publication number
JP2021522836A
JP2021522836A JP2020563947A JP2020563947A JP2021522836A JP 2021522836 A JP2021522836 A JP 2021522836A JP 2020563947 A JP2020563947 A JP 2020563947A JP 2020563947 A JP2020563947 A JP 2020563947A JP 2021522836 A JP2021522836 A JP 2021522836A
Authority
JP
Japan
Prior art keywords
nucleic acid
acid sequence
interfering rna
rna
viral vector
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
JP2020563947A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021522836A5 (enExample
Inventor
ジョエル シャンバーレイン，
ジョエル シャンバーレイン，
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Washington
Original Assignee
University of Washington
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.)
Filing date
Publication date
Application filed by University of Washington filed Critical University of Washington
Publication of JP2021522836A publication Critical patent/JP2021522836A/ja
Publication of JP2021522836A5 publication Critical patent/JP2021522836A5/ja
Pending legal-status Critical Current

Links

Images

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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11—Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11001—Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/122—Hairpin
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Veterinary Medicine (AREA)
  • Biophysics (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2020563947A 2018-05-15 2019-05-15 スプライスオパシーの軽減、及びｒｎａドミナンス障害の治療のための組成物及び方法 Pending JP2021522836A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862671769P 2018-05-15 2018-05-15
US62/671,769 2018-05-15
PCT/US2019/032423 WO2019222354A1 (en) 2018-05-15 2019-05-15 Compositions and methods for reducing spliceopathy and treating rna dominance disorders

Publications (2)

Publication Number Publication Date
JP2021522836A true JP2021522836A (ja) 2021-09-02
JP2021522836A5 JP2021522836A5 (enExample) 2022-05-24

Family

ID=68540673

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2020563947A Pending JP2021522836A (ja) 2018-05-15 2019-05-15 スプライスオパシーの軽減、及びｒｎａドミナンス障害の治療のための組成物及び方法

Country Status (15)

Country Link
US (1) US20210269825A1 (enExample)
EP (1) EP3793566A4 (enExample)
JP (1) JP2021522836A (enExample)
KR (1) KR20210010549A (enExample)
CN (1) CN112469421A (enExample)
AU (1) AU2019268346A1 (enExample)
BR (1) BR112020023298A2 (enExample)
CA (1) CA3098249A1 (enExample)
CL (1) CL2020002955A1 (enExample)
CO (1) CO2020015239A2 (enExample)
MA (1) MA51938B1 (enExample)
MX (1) MX2020012269A (enExample)
PH (1) PH12020551913A1 (enExample)
SG (1) SG11202011151VA (enExample)
WO (1) WO2019222354A1 (enExample)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113166208B (zh) 2018-10-02 2024-11-22 沃雅戈治疗公司 重新定向aav衣壳的嗜性
US12630842B2 (en) 2020-05-13 2026-05-19 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
US11833221B2 (en) 2021-09-01 2023-12-05 Ionis Pharmaceuticals, Inc. Oligomeric compounds for reducing DMPK expression
EP4426716A1 (en) 2021-11-02 2024-09-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
KR20240169111A (ko) * 2022-04-06 2024-12-02 젠자임 코포레이션 Dm-1 근긴장성 이영양증에 대한 표적화된 유전자 요법
US20250352672A1 (en) * 2022-05-13 2025-11-20 University Of Washington Method for treatment of myotonic dystrophy combining protein expression and rna interference vector delivery with tissue detargeting
TW202409289A (zh) * 2022-06-27 2024-03-01 美商安斯泰來基因治療股份有限公司 用於治療肌強直性營養不良之組合物及方法
WO2024211480A2 (en) * 2023-04-05 2024-10-10 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
WO2024229164A2 (en) * 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025038795A1 (en) * 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025122536A1 (en) * 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
TW202615058A (zh) * 2024-06-12 2026-04-16 美商艾拉倫製藥股份有限公司 失養強直性肌肉蛋白質激酶（ｄｍｐｋ）ｉｒｎａ組成物及其使用方法

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110111491A1 (en) * 2002-08-05 2011-05-12 University Of Iowa Research Foundation Rna interference suppresion of neurodegenerative diseases and methods of use thereof
JP2018502601A (ja) * 2014-12-24 2018-02-01 ユニクア・アイピー・ベーフェー ＲＮＡｉで誘発されるハンチンチン遺伝子抑制

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5977333A (en) * 1992-02-06 1999-11-02 Massachusetts Institute Of Technology DNA sequence encoding the myotonic dystrophy gene and uses thereof
US20120322861A1 (en) * 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
ES2756326T3 (es) * 2010-07-19 2020-04-27 Ionis Pharmaceuticals Inc Modulación de la expresión de distrofia miotónica-proteína quinasa (DMPK)
WO2017079291A1 (en) * 2015-11-02 2017-05-11 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating c90rf72
IL302455A (en) * 2016-03-01 2023-06-01 Univ Florida Adeno-associated virus vectors for the treatment of dominant retinitis pigmentosa
MX381216B (es) * 2016-09-30 2025-03-12 Regeneron Pharma Animales no humanos que tienen una expansion de repeticiones de hexanucleotidos en un locus c9orf72.
WO2020029303A1 (zh) * 2018-08-10 2020-02-13 富士通株式会社 一种通信方法、装置和通信系统

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110111491A1 (en) * 2002-08-05 2011-05-12 University Of Iowa Research Foundation Rna interference suppresion of neurodegenerative diseases and methods of use thereof
JP2018502601A (ja) * 2014-12-24 2018-02-01 ユニクア・アイピー・ベーフェー ＲＮＡｉで誘発されるハンチンチン遺伝子抑制

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
HU J. ET AL.: "Engineering Duplex RNAs for Challenging Targets: Recognition of GGGGCC/CCCCGG Repeats at the ALS/FTD", CHEMISTRY & BIOLOGY, 2015, VOL. 22, P. 1505-1511, JPN6023019416, ISSN: 0005210881 *
HU J. ET AL.: "Recognition of c9orf72 Mutant RNA by Single-Stranded Silencing RNAs", NUCLEIC ACID THERAPEUTICS, 2017, VOL. 28, NO. 2, P. 87-94, JPN6023019417, ISSN: 0005210882 *
LANGLOIS, MA. ET AL.: "Cytoplasmic and Nuclear Retained DMPK mRNAs Are Targets for RNA Interference in Myotonic Dystrophy C", JOURNAL OF BIOLOGICAL CHEMISTRY, 2005, VOL. 280, P. 16949-16954, JPN6023019415, ISSN: 0005210880 *

Also Published As

Publication number Publication date
MA51938A1 (fr) 2021-11-30
SG11202011151VA (en) 2020-12-30
PH12020551913A1 (en) 2021-06-14
MA51938B1 (fr) 2022-10-31
AU2019268346A1 (en) 2020-12-24
KR20210010549A (ko) 2021-01-27
MX2020012269A (es) 2021-04-28
EP3793566A4 (en) 2022-03-16
CL2020002955A1 (es) 2021-04-23
WO2019222354A1 (en) 2019-11-21
BR112020023298A2 (pt) 2021-03-09
US20210269825A1 (en) 2021-09-02
EP3793566A1 (en) 2021-03-24
CO2020015239A2 (es) 2021-03-08
CN112469421A (zh) 2021-03-09
CA3098249A1 (en) 2019-11-21

Similar Documents

Publication Publication Date Title
US20210269825A1 (en) Compositions and methods for reducing spliceopathy and treating rna dominance disorders
CN105960413A (zh) 人工dna-结合蛋白及其用途
JP2025175283A (ja) Ａｍｄの処置のためのｈｔｒａ１調節
JP7500429B2 (ja) 優性遺伝性カテコラミン誘発多形性心室頻拍の治療のための組成物および方法
EP3901262A1 (en) Compositions for use in treating autosomal dominant best1-related retinopathies
US20240376497A1 (en) Frataxin gene therapy
CA3210480A1 (en) Treatment of inflammation with glucocorticoids and angiopoietin-like 7 (angptl7) inhibitors
WO2024161022A2 (en) Compositions and methods for treating neurological diseases
AU2023342734A1 (en) Compositions and methods for treating neurological diseases
WO2025126153A2 (en) Compositions and methods for treating sod1-mediated neurological diseases
JP2026508803A (ja) 神経学的疾患を治療するための組成物及び方法
WO2024006770A1 (en) Compositions and methods for the treatment of myotonic dystrophies
CN112779252B (zh) 靶向SMN2启动子区MeCP2结合的关键甲基化区域的反义寡核苷酸
US20260125686A1 (en) Compositions and methods for modulation of cftr
WO2025056018A1 (en) Mutation-independent allele-specific crispr targeting strategy for treating genetic diseases
WO2024206672A1 (en) Nucleic acid off-switches and methods and uses thereof
WO2024006775A2 (en) Compositions and methods for the treatment of myotonic dystrophies
JP2025530647A (ja) ダイナミン２関連疾患に対するアレル特異的ｓｉＲＮＡ療法
WO2024154102A1 (en) Compositions and methods for administration of therapeutic and diagnostic agents to the central nervous system
JP2026504808A (ja) 神経変性疾患の処置のためのポリヌクレオチド組成物および方法
CN120882870A (zh) 用于外科手术相关纤维化治疗的基因编辑
WO2025072604A1 (en) Rna-editing gene therapy approaches for treating myotonic dystrophy type 1 (dm1)

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220516

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20220516

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230516

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20230814

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20231205