KR20220113940A - Rna 분자의 고-효율 재조합을 위한 조성물 및 방법 - Google Patents

Rna 분자의 고-효율 재조합을 위한 조성물 및 방법 Download PDF

Info

Publication number
KR20220113940A
KR20220113940A KR1020227019191A KR20227019191A KR20220113940A KR 20220113940 A KR20220113940 A KR 20220113940A KR 1020227019191 A KR1020227019191 A KR 1020227019191A KR 20227019191 A KR20227019191 A KR 20227019191A KR 20220113940 A KR20220113940 A KR 20220113940A
Authority
KR
South Korea
Prior art keywords
rna
sequence
molecule
protein
dimerization domain
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
KR1020227019191A
Other languages
English (en)
Korean (ko)
Inventor
루카스 크리스토프 바흐만
새뮤얼 로렌스 파프
Original Assignee
솔크 인스티튜트 포 바이올로지칼 스터디즈
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
Priority claimed from PCT/US2020/025430 external-priority patent/WO2020205604A1/en
Application filed by 솔크 인스티튜트 포 바이올로지칼 스터디즈 filed Critical 솔크 인스티튜트 포 바이올로지칼 스터디즈
Publication of KR20220113940A publication Critical patent/KR20220113940A/ko
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
    • 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
    • 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
    • C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
    • 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
    • C12N15/64—General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
    • 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
    • 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
    • C12N2310/16—Aptamers
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
    • C12N2840/445—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor for trans-splicing, e.g. polypyrimidine tract, branch point splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Saccharide Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
KR1020227019191A 2019-11-11 2020-09-30 Rna 분자의 고-효율 재조합을 위한 조성물 및 방법 Pending KR20220113940A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962933714P 2019-11-11 2019-11-11
US62/933,714 2019-11-11
USPCT/US2020/025430 2020-03-27
PCT/US2020/025430 WO2020205604A1 (en) 2019-03-29 2020-03-27 High-efficiency reconstitution of rna molecules
PCT/US2020/053643 WO2021096605A1 (en) 2019-11-11 2020-09-30 Compositions and methods for high-efficiency recombination of rna molecules

Publications (1)

Publication Number Publication Date
KR20220113940A true KR20220113940A (ko) 2022-08-17

Family

ID=75911442

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020227019191A Pending KR20220113940A (ko) 2019-11-11 2020-09-30 Rna 분자의 고-효율 재조합을 위한 조성물 및 방법

Country Status (11)

Country Link
US (1) US20220265855A1 (https=)
EP (1) EP4058571A1 (https=)
JP (2) JP7759106B2 (https=)
KR (1) KR20220113940A (https=)
CN (1) CN114945666A (https=)
AU (1) AU2020384996A1 (https=)
BR (1) BR112022009006A2 (https=)
CA (1) CA3157799A1 (https=)
IL (1) IL292904A (https=)
MX (1) MX2022005670A (https=)
WO (1) WO2021096605A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020205604A1 (en) 2019-03-29 2020-10-08 Salk Institute For Biological Studies High-efficiency reconstitution of rna molecules
KR20220157944A (ko) 2020-02-21 2022-11-29 아카우오스, 인크. 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법
AU2022337146A1 (en) * 2021-09-03 2024-03-14 Tacit Therapeutics, Inc. Rna editing via recruitment of spliceosome components
CA3240197A1 (en) * 2021-12-10 2025-02-26 Aavantibio, Inc. Compositions comprising kozak sequences selected for enhanced expression
WO2023250324A2 (en) * 2022-06-21 2023-12-28 The Regents Of The University Of California Compositions and methods for reducing rna levels
US20260055400A1 (en) * 2022-06-21 2026-02-26 The Regents Of The University Of California Compositions and methods for treating huntington's disease

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6110743A (en) 1995-02-10 2000-08-29 The Regents Of The University Of California Development and use of human pancreatic cell lines
US20060134658A1 (en) * 2004-08-09 2006-06-22 Garcia-Blanco Mariano A Use of RNA trans-splicing for generation of interfering RNA molecules
ES2763526T3 (es) * 2014-07-03 2020-05-29 Hoffmann La Roche Sistemas de expresión de polipéptidos

Also Published As

Publication number Publication date
JP2025118824A (ja) 2025-08-13
JP2023500957A (ja) 2023-01-11
CN114945666A (zh) 2022-08-26
MX2022005670A (es) 2022-07-19
WO2021096605A1 (en) 2021-05-20
US20220265855A1 (en) 2022-08-25
IL292904A (en) 2022-07-01
EP4058571A1 (en) 2022-09-21
JP7759106B2 (ja) 2025-10-23
AU2020384996A1 (en) 2022-06-23
BR112022009006A2 (pt) 2022-10-11
CA3157799A1 (en) 2021-05-20

Similar Documents

Publication Publication Date Title
KR102666695B1 (ko) Rna를 편집하기 위한 방법 및 조성물
KR102935609B1 (ko) 유전자 편집을 위한 cpf1-관련 방법 및 조성물
JP7399866B2 (ja) ＣＡＲＴｙｒｉｎ組成物とその利用方法
KR20220113940A (ko) Rna 분자의 고-효율 재조합을 위한 조성물 및 방법
US12448636B2 (en) High-efficiency reconstitution of RNA molecules
KR102808368B1 (ko) 선택적 유전자 조절을 위한 조성물 및 방법
CN110730821B (zh) 增强的hAT家族转座子介导的基因转移及相关组合物、系统和方法
CN114269919A (zh) 采用工程化rna利用内源adar进行靶向的rna编辑
KR20230057487A (ko) 게놈 조정을 위한 방법 및 조성물
KR20210125560A (ko) 유전성 질환의 치료를 위한 것을 포함하는, 아데노신 데아미나제 염기 편집기를 사용한 질환-관련 유전자의 스플라이스 수용체 부위 파괴
KR20220038362A (ko) 재조합 ad35 벡터 및 관련 유전자 요법 개선
KR20200107949A (ko) 조작된 dna 결합 단백질
KR102249982B1 (ko) 트랜스포존 시스템, 이를 포함한 키트, 및 이들의 용도
KR20220044811A (ko) Crispr/cas13을 사용하는 표적화된 트랜스-이어맞추기
KR20210082205A (ko) 레트로바이러스 인테그라제-Cas9 융합 단백질을 이용한 유도된 비상동 DNA 삽입에 의한 게놈 편집
CN117015605A (zh) 使用工程化rna通过利用内源性adar进行靶向rna编辑
CN117337326A (zh) 工程化的Cas12i核酸酶、效应蛋白及其用途
JP2024518413A (ja) 修飾ヌクレアーゼ
KR20230125806A (ko) 선천성 근이영양증의 치료를 위한 치료용 lama2 페이로드
KR20230066453A (ko) 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도
US20240216544A1 (en) Methods and compositions for expression of editing proteins
RU2792187C2 (ru) Композиции cart-иринов и способы их применения
CN117043324A (zh) 用于治疗先天性肌营养不良的治疗性lama2载荷
CN117836420A (zh) 重组tert编码病毒基因组和运载体
HK40109651A (zh) 重组tert编码病毒基因组和运载体

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

R18-X000 Changes to party contact information recorded

St.27 status event code: A-3-3-R10-R18-oth-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

D21 Rejection of application intended

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D21-EXM-PE0902 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902