KR20240031224A - 전정 지지 세포 프로모터 및 이의 용도 - Google Patents

전정 지지 세포 프로모터 및 이의 용도 Download PDF

Info

Publication number
KR20240031224A
KR20240031224A KR1020237041741A KR20237041741A KR20240031224A KR 20240031224 A KR20240031224 A KR 20240031224A KR 1020237041741 A KR1020237041741 A KR 1020237041741A KR 20237041741 A KR20237041741 A KR 20237041741A KR 20240031224 A KR20240031224 A KR 20240031224A
Authority
KR
South Korea
Prior art keywords
nucleic acid
vestibular
acid vector
protein
seq
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
KR1020237041741A
Other languages
English (en)
Korean (ko)
Inventor
조셉 번즈
타일러 깁슨
가브리엘라 프레게니그
캐시 쏘
Original Assignee
데시벨 테라퓨틱스, 인크.
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 데시벨 테라퓨틱스, 인크. filed Critical 데시벨 테라퓨틱스, 인크.
Publication of KR20240031224A publication Critical patent/KR20240031224A/ko
Pending legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/16Otologicals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702Regulators; Modulating activity
    • C07K14/4705Regulators; Modulating activity stimulating, promoting or activating activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Display Devices Of Pinball Game Machines (AREA)
  • Absorbent Articles And Supports Therefor (AREA)
  • Orthopedics, Nursing, And Contraception (AREA)
KR1020237041741A 2021-05-04 2022-05-04 전정 지지 세포 프로모터 및 이의 용도 Pending KR20240031224A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163184015P 2021-05-04 2021-05-04
US63/184,015 2021-05-04
PCT/US2022/027679 WO2022235805A1 (en) 2021-05-04 2022-05-04 Vestibular supporting cell promoters and uses thereof

Publications (1)

Publication Number Publication Date
KR20240031224A true KR20240031224A (ko) 2024-03-07

Family

ID=83932529

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020237041741A Pending KR20240031224A (ko) 2021-05-04 2022-05-04 전정 지지 세포 프로모터 및 이의 용도

Country Status (13)

Country Link
US (1) US20240218391A1 (https=)
EP (1) EP4334460A4 (https=)
JP (1) JP2024517250A (https=)
KR (1) KR20240031224A (https=)
CN (1) CN117616127A (https=)
AU (1) AU2022271238A1 (https=)
BR (1) BR112023023092A2 (https=)
CA (1) CA3219057A1 (https=)
CL (2) CL2023003257A1 (https=)
CO (1) CO2023016746A2 (https=)
IL (1) IL308143A (https=)
MX (1) MX2023013058A (https=)
WO (1) WO2022235805A1 (https=)

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20140024663A1 (en) * 2012-07-17 2014-01-23 Georgia Health Sciences University Research Institute, Inc. Atb(0,+) amino acid transporter as a drug target for treatment of estrogen receptor-positive breast cancer
US20160298080A1 (en) * 2013-12-03 2016-10-13 The Johns Hopkins University Method for highly efficient conversion of human stem cells to lineage-specific neurons
SG10201913266UA (en) * 2015-12-11 2020-02-27 Massachusetts Eye & Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells

Also Published As

Publication number Publication date
AU2022271238A9 (en) 2024-01-04
CN117616127A (zh) 2024-02-27
BR112023023092A2 (pt) 2024-01-30
WO2022235805A1 (en) 2022-11-10
IL308143A (en) 2023-12-01
CO2023016746A2 (es) 2024-02-26
MX2023013058A (es) 2023-12-14
CL2024002594A1 (es) 2025-01-03
EP4334460A1 (en) 2024-03-13
AU2022271238A1 (en) 2023-12-14
EP4334460A4 (en) 2025-11-05
US20240218391A1 (en) 2024-07-04
JP2024517250A (ja) 2024-04-19
CA3219057A1 (en) 2022-11-10
CL2023003257A1 (es) 2024-05-31

Similar Documents

Publication Publication Date Title
AU2020289750B2 (en) Engineered meganucleases with recognition sequences found in the human T cell receptor alpha constant region gene
US20230364266A1 (en) Methods and compositions for genomic integration
AU2024205047A1 (en) Genetically-modified cells comprising a modified human T cell receptor alpha constant region gene
AU2021204620A1 (en) Central nervous system targeting polynucleotides
US11779657B2 (en) Compositions and methods for mitochondrial genome editing
KR20230019843A (ko) 표적 이중 가닥 뉴클레오티드 서열의 두 가닥의 동시 편집을 위한 방법 및 조성물
JP7275043B2 (ja) 増大したhATファミリートランスポゾン媒介遺伝子導入ならびに関連する組成物、システムおよび方法
US20190269762A1 (en) Dystrophin gene exon deletion using engineered nucleases
KR20210143230A (ko) 뉴클레오티드 서열을 편집하기 위한 방법 및 조성물
JP5051738B2 (ja) 人工遍在的クロマチン開放エレメント(ucoe)
AU2016343979A1 (en) Delivery of central nervous system targeting polynucleotides
KR20210151785A (ko) 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도
AU2019365100B2 (en) Genome editing by directed non-homologous DNA insertion using a retroviral integrase-Cas9 fusion protein
CN112725282A (zh) 携带正交tRNA/氨酰tRNA合成酶的稳定细胞系的构建
AU2016228914B2 (en) Tools and methods for using cell division loci to control proliferation of cells
CN115362000A (zh) 使用多核苷酸沉默和替换的神经退行性病症的基因疗法
KR20200085812A (ko) 바이러스 벡터-유도된 염증 반응을 억제하기 위한 조성물 및 방법
KR20240037192A (ko) 게놈 통합을 위한 방법 및 조성물
CN110637090A (zh) 用于表达大型核酸转基因的质粒载体
PT2380979E (pt) Substância proteica com estrutura em tripla hélice e seu método de fabrico
CN113939595A (zh) 包括人源化白蛋白基因座的非人动物
CN110831614A (zh) 用于递送Na/K ATP酶/Src受体复合物拮抗剂的表达载体和相关方法
CN109295100A (zh) 携带正交tRNA/氨酰tRNA合成酶的稳定细胞系的构建
CN112522321B (zh) 一种基因治疗载体及其用途
CN113302202A (zh) 利用表达胰岛素样生长因子1异构体的脱氧核糖核酸构建体的神经病变的治疗

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

PG1501 Laying open of application

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

A201 Request for examination
P11-X000 Amendment of application requested

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

R18 Changes to party contact information recorded

Free format text: ST27 STATUS EVENT CODE: A-3-3-R10-R18-OTH-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

R18-X000 Changes to party contact information recorded

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