BR112020021228A2 - moléculas de união trans - Google Patents

moléculas de união trans Download PDF

Info

Publication number
BR112020021228A2
BR112020021228A2 BR112020021228-8A BR112020021228A BR112020021228A2 BR 112020021228 A2 BR112020021228 A2 BR 112020021228A2 BR 112020021228 A BR112020021228 A BR 112020021228A BR 112020021228 A2 BR112020021228 A2 BR 112020021228A2
Authority
BR
Brazil
Prior art keywords
trans
abca4
nucleic acid
nucleotides
intron
Prior art date
Application number
BR112020021228-8A
Other languages
English (en)
Portuguese (pt)
Inventor
Philip R. Johnson
Bruce C. SCHNEPP
Jean Bennett
Scott J. Dooley
Krishna Jawaharlal Fisher
Junwei Sun
Original Assignee
The Trustees Of The University Of Pennsylvania
Ascidian Therapeutics, Inc.
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 The Trustees Of The University Of Pennsylvania, Ascidian Therapeutics, Inc. filed Critical The Trustees Of The University Of Pennsylvania
Publication of BR112020021228A2 publication Critical patent/BR112020021228A2/pt

Links

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
    • 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
    • C12N15/1138—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 receptors or cell surface proteins
    • 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
    • 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
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/71—Fusion polypeptide containing domain for protein-protein interaction containing domain for transcriptional activaation, e.g. VP16
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/85—Fusion polypeptide containing an RNA binding domain
    • 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
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • C12N2320/33—Alteration of splicing
    • 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
    • C12N2320/34—Allele or polymorphism specific uses
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (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)
  • Medicinal Preparation (AREA)
BR112020021228-8A 2018-04-17 2019-04-17 moléculas de união trans BR112020021228A2 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862658667P 2018-04-17 2018-04-17
US201862658658P 2018-04-17 2018-04-17
US62/658,658 2018-04-17
US62/658,667 2018-04-17
PCT/US2019/027981 WO2019204514A1 (en) 2018-04-17 2019-04-17 Trans-splicing molecules

Publications (1)

Publication Number Publication Date
BR112020021228A2 true BR112020021228A2 (pt) 2021-03-02

Family

ID=68239871

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112020021228-8A BR112020021228A2 (pt) 2018-04-17 2019-04-17 moléculas de união trans

Country Status (11)

Country Link
US (3) US11993776B2 (https=)
EP (1) EP3781213A4 (https=)
JP (2) JP7561624B2 (https=)
KR (2) KR102866133B1 (https=)
CN (1) CN112449605A (https=)
AU (1) AU2019255708B2 (https=)
BR (1) BR112020021228A2 (https=)
CA (1) CA3097004A1 (https=)
IL (1) IL278019A (https=)
MX (2) MX2020010959A (https=)
WO (1) WO2019204514A1 (https=)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3097004A1 (en) 2018-04-17 2019-10-24 The Trustees Of The University Of Pennsylvania Trans-splicing molecules
WO2020214973A1 (en) * 2019-04-17 2020-10-22 The Trustees Of The University Of Pennsylvania Triple helix terminator for efficient rna trans-splicing
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
AU2022337146A1 (en) 2021-09-03 2024-03-14 Tacit Therapeutics, Inc. Rna editing via recruitment of spliceosome components
AU2023269865A1 (en) * 2022-05-13 2024-11-21 Ascidian Therapeutics, Inc. Abca4 trans-splicing molecules
WO2024011203A2 (en) * 2022-07-07 2024-01-11 Intergalactic Therapeutics, Inc. Ocular vectors and uses thereof
EP4594505A1 (en) * 2022-09-30 2025-08-06 Centre National de la Recherche Scientifique Therapy by trans-splicing of opa1 pre-messenger rnas for the treatment of diseases associated with opa1 gene mutations
CN121099993A (zh) * 2023-02-15 2025-12-09 海鞘治疗公司 Htt反式剪接分子
WO2025166360A1 (en) * 2024-02-03 2025-08-07 Duke University Compositions for and methods of engineering the transcriptome
WO2026012439A1 (en) * 2024-07-11 2026-01-15 Sichuan Real & Best Biotech Co., Ltd. Rna trans-splicing molecules and systems

Family Cites Families (42)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6280978B1 (en) 1995-12-15 2001-08-28 Intronn Holdings, Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
DE69636937T3 (de) 1995-12-15 2011-01-05 Virxsys Corp. Durch trans-spaltung erhaltene therapeutische molekule
JP2001510557A (ja) 1996-10-04 2001-07-31 イントロン エルエルシー サンプル収集デバイス及びマーカーを用いたサンプル収集方法、及びサンプル確認、ラボの評価及び／又は認定の際の比較試料としてのマーカーの使用
US6929907B2 (en) 1999-12-31 2005-08-16 North Carolina State University Methods and compositions for determining the purity of chemically synthesized nucleic acids
JP4500540B2 (ja) 2001-06-29 2010-07-14 ヴェリ‐キュー，インコーポレイテッド 化学的に合成された核酸の純度を決定し、かつ化学的に合成された核酸を精製するための方法および組成物
JP2006505242A (ja) 2002-02-12 2006-02-16 イントロン，インコーポレーテッド スプライセオソームにより媒介されるｒｎａトランス−スプライシングにおいて使用するための方法および組成物
AU2003213270A1 (en) 2002-02-25 2003-09-09 Intronn, Inc. Trans-splicing mediated imaging of gene expression
US7399753B2 (en) 2002-02-25 2008-07-15 Virxsys Corporation Trans-splicing mediated photodynamic therapy
AU2003247505A1 (en) 2002-06-05 2003-12-22 University Of Iowa Research Foundation Spliceosome mediated rna trans-splicing in stem cells
CA2488246A1 (en) 2002-06-05 2003-12-18 Intronn, Inc. Spliceosome mediated rna trans-splicing and correction of factor viii genetic defects using spliceosome mediated rna trans spling
US20040018622A1 (en) 2002-07-17 2004-01-29 Mitchell Lloyd G. Spliceosome mediated RNA trans-splicing for correction of skin disorders
EP1579004B1 (en) 2002-10-23 2010-06-16 VIRxSYS Corporation Screening methods for identification of efficient pre-trans-splicing molecules
WO2005070948A1 (en) 2004-01-23 2005-08-04 Intronn, Inc. Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated rna trans splicing
JP2007518423A (ja) 2004-01-23 2007-07-12 イントロン、インコーポレイテッド スプライセオソーム仲介型ｒｎａトランススプライシングを使用するアポａ−１及びその変異体の発現
WO2006026611A2 (en) 2004-08-31 2006-03-09 Mitchell Lloyd G Reverse transcriptase mediated rna gene expression
US7943374B2 (en) 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
US20090214478A1 (en) 2008-02-21 2009-08-27 Alberto Auricchio Method of treating ocular diseases by gene therapy
US8236557B2 (en) 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
EP2151248A1 (en) 2008-07-30 2010-02-10 Johann Bauer Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses
AU2013234376B2 (en) * 2008-10-22 2016-07-28 Acucela, Inc. Compounds for treating ophthalmic diseases and disorders
US8648053B2 (en) 2010-10-20 2014-02-11 Rosalind Franklin University Of Medicine And Science Antisense oligonucleotides that target a cryptic splice site in Ush1c as a therapeutic for Usher syndrome
US9249425B2 (en) 2011-05-16 2016-02-02 The Trustees Of The University Of Pennslyvania Proviral plasmids and production of recombinant adeno-associated virus
BR112014004895B1 (pt) * 2011-09-05 2022-07-05 Stichting Radboud Universitair Medisch Centrum Oligonucleotídeos antissentido para o tratamento de amaurose congênita de leber
EP2872183B1 (en) 2012-07-11 2018-09-26 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for rpgr x-linked retinal degeneration
CN104955951A (zh) 2012-10-16 2015-09-30 麻省理工学院 稳定的非多腺苷酸化rna的产生
GB201219762D0 (en) 2012-11-02 2012-12-19 Bauer Johann A RNA trans-splicing molecule (RTM) for use in the treatment of cancer
US10266845B2 (en) 2013-02-08 2019-04-23 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
US9567376B2 (en) 2013-02-08 2017-02-14 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
CA2909733C (en) 2013-04-18 2023-10-17 Fondazione Telethon Effective delivery of large genes by dual aav vectors
WO2015009575A1 (en) 2013-07-16 2015-01-22 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of disorders related to cep290
WO2015138510A1 (en) 2014-03-10 2015-09-17 Editas Medicine., Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
WO2016005524A1 (en) 2014-07-09 2016-01-14 Lexogen Gmbh Methods and products for quantifying rna transcript variants
RS63416B1 (sr) * 2015-03-03 2022-08-31 Fond Telethon Sistem višestrukih vektora i njegove primene
EP3377116A4 (en) 2015-11-19 2019-07-10 The Trustees of The University of Pennsylvania COMPOSITIONS AND METHODS FOR CORRECTING ECG-RELATED EYE DISEASE
EP3390634A4 (en) * 2015-12-14 2019-08-14 Cold Spring Harbor Laboratory COMPOSITIONS AND METHODS FOR THE TREATMENT OF EYE DISEASES
KR20200036912A (ko) * 2017-07-31 2020-04-07 리플렉션 바이오테크놀러지스 리미티드 안과 질환을 위한 세포 모델 및 치료요법
US11795455B2 (en) 2017-07-31 2023-10-24 Massachusetts Institute Of Technology RNA cleavage-induced transcript stabilizer and uses thereof
CA3097004A1 (en) 2018-04-17 2019-10-24 The Trustees Of The University Of Pennsylvania Trans-splicing molecules
WO2020214990A1 (en) 2019-04-17 2020-10-22 Ascidian Therapeutics, Inc. Abca4 cell line and methods of use
WO2020214973A1 (en) 2019-04-17 2020-10-22 The Trustees Of The University Of Pennsylvania Triple helix terminator for efficient rna trans-splicing
CA3138520C (en) 2019-05-01 2023-10-10 The Procter & Gamble Company Probiotic bacterial strains that produce short chain fatty acids and compositions comprising same
AU2023269865A1 (en) 2022-05-13 2024-11-21 Ascidian Therapeutics, Inc. Abca4 trans-splicing molecules

Also Published As

Publication number Publication date
CN112449605A (zh) 2021-03-05
US11993776B2 (en) 2024-05-28
US20240318186A1 (en) 2024-09-26
CA3097004A1 (en) 2019-10-24
JP2021520837A (ja) 2021-08-26
MX2020010959A (es) 2021-01-15
EP3781213A4 (en) 2022-10-19
EP3781213A1 (en) 2021-02-24
JP2024099770A (ja) 2024-07-25
MX2025007209A (es) 2025-07-01
KR102866133B1 (ko) 2025-09-30
US20260078380A1 (en) 2026-03-19
JP7561624B2 (ja) 2024-10-04
US20210155938A1 (en) 2021-05-27
KR20250150157A (ko) 2025-10-17
AU2019255708B2 (en) 2026-02-12
IL278019A (en) 2020-11-30
WO2019204514A1 (en) 2019-10-24
AU2019255708A1 (en) 2020-11-26
KR20210018222A (ko) 2021-02-17
US12442003B2 (en) 2025-10-14

Similar Documents

Publication Publication Date Title
US12442003B2 (en) Trans-splicing molecules
US12403204B2 (en) Gene therapy for ocular disorders
BR112020001940A2 (pt) modelos celulares de e terapias para doenças oculares
JP7572990B2 (ja) Ｒｄｈ１２が関与する疾患及び疾病を治療する方法及び組成物
US20190343920A1 (en) Aav-mediated gene therapy for nphp5 lca-ciliopathy
US12377169B2 (en) Dual AAV-MYO7A vectors with improved safety for the treatment of USH1B
JP2018522529A (ja) 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用
AU2016355343A1 (en) Compositions and methods for correction of heritable ocular disease
CN114040974B (zh) 用于高效rna反式剪接的三螺旋终止子
JP2023116709A (ja) 眼疾患のための遺伝子療法
US20230233709A1 (en) Gene therapy for ocular disorders
WO2020038473A1 (zh) 重组人ⅱ型线粒体动力蛋白样gtp酶基因序列及其应用
KR20250011939A (ko) Abca4 트랜스-스플라이싱 분자
JP2025156349A (ja) Ｃｌｒｎ１に関連する聴力喪失及び／または視力喪失を治療する方法
WO2024069144A1 (en) Rna editing vector
JP2021520231A (ja) シュタルガルト病の処置のための組成物及び方法
BR122024014087A2 (pt) Vetores e células

Legal Events

Date Code Title Description
B350 Update of information on the portal [chapter 15.35 patent gazette]
B06W Patent application suspended after preliminary examination (for patents with searches from other patent authorities) chapter 6.23 patent gazette]