MX2019013151A - Composiciones y metodos para expresar otoferlina. - Google Patents

Composiciones y metodos para expresar otoferlina.

Info

Publication number
MX2019013151A
MX2019013151A MX2019013151A MX2019013151A MX2019013151A MX 2019013151 A MX2019013151 A MX 2019013151A MX 2019013151 A MX2019013151 A MX 2019013151A MX 2019013151 A MX2019013151 A MX 2019013151A MX 2019013151 A MX2019013151 A MX 2019013151A
Authority
MX
Mexico
Prior art keywords
compositions
methods
otoferlin
expressing
dfnb9
Prior art date
Application number
MX2019013151A
Other languages
English (en)
Spanish (es)
Inventor
L Boye Sanford
Dyka Frank
W Hauswirth William
Akil Omar
Original Assignee
Univ Florida
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=64016253&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=MX2019013151(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Univ Florida filed Critical Univ Florida
Publication of MX2019013151A publication Critical patent/MX2019013151A/es

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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/40—Systems of functionally co-operating 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
    • 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)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Mycology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
  • Medicinal Preparation (AREA)
MX2019013151A 2017-05-05 2018-05-04 Composiciones y metodos para expresar otoferlina. MX2019013151A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762502462P 2017-05-05 2017-05-05
PCT/US2018/031009 WO2018204734A1 (en) 2017-05-05 2018-05-04 Compositions and methods for expressing otoferlin

Publications (1)

Publication Number Publication Date
MX2019013151A true MX2019013151A (es) 2020-02-05

Family

ID=64016253

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2019013151A MX2019013151A (es) 2017-05-05 2018-05-04 Composiciones y metodos para expresar otoferlina.
MX2023013435A MX2023013435A (es) 2017-05-05 2019-11-04 Composiciones y metodos para expresar otoferlina.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2023013435A MX2023013435A (es) 2017-05-05 2019-11-04 Composiciones y metodos para expresar otoferlina.

Country Status (9)

Country Link
US (2) US12359221B2 (OSRAM)
EP (1) EP3635100A4 (OSRAM)
JP (3) JP7240675B2 (OSRAM)
KR (2) KR102606810B1 (OSRAM)
CN (1) CN110892062A (OSRAM)
AU (2) AU2018261769B2 (OSRAM)
CA (1) CA3061955A1 (OSRAM)
MX (2) MX2019013151A (OSRAM)
WO (1) WO2018204734A1 (OSRAM)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013075008A1 (en) 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
JP7240675B2 (ja) * 2017-05-05 2023-03-16 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． オトフェルリンを発現させるための組成物および方法
DE102018103924A1 (de) * 2018-02-21 2019-08-22 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Gentherapeutische Behandlung von Schwerhörigkeit
IL276764B2 (en) * 2018-02-22 2026-05-01 Akouos Inc Compositions and methods for treating non-age-related hearing impairment in humans
CN119410636A (zh) 2018-04-27 2025-02-11 分贝治疗公司 肌球蛋白15启动子及其用途
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
WO2020093018A1 (en) * 2018-11-01 2020-05-07 University Of Florida Research Foundation, Incorporated A codon optimized otoferlin aav dual vector gene therapy
BR112021013996A2 (pt) * 2019-01-18 2021-10-26 Institut Pasteur Terapia gênica mediada por aav que recupera o gene otoferlina
EP3921032A4 (en) 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATMENT OF SENSORINEURAL HEARING LOSS USING OTOFERLIN DOUBLE VECTOR SYSTEMS
JP7562544B2 (ja) 2019-02-08 2024-10-07 デシベル セラピューティクス インコーポレイテッド ミオシン１５プロモーター及びその使用
JP7665609B2 (ja) * 2019-10-30 2025-04-21 デシベル セラピューティクス インコーポレイテッド オトフェリン二重ベクター系及びそれを含む感音難聴を治療するための組成物
CN115066432B (zh) * 2019-10-30 2026-04-17 分贝治疗公司 使用耳畸蛋白双载体系统治疗感觉神经性听力损失的组合物和方法
CN116096905A (zh) 2020-02-21 2023-05-09 阿库斯股份有限公司 用于治疗人受试者的非年龄相关性听力损伤的组合物和方法
CA3174500A1 (en) 2020-04-01 2021-10-07 University Of Florida Research Foundation, Incorporated Dual aav-myo7a vectors with improved safety for the treatment of ush1b
CN112125969A (zh) * 2020-09-30 2020-12-25 东南大学 生物因子rimbp2在维持内耳毛细胞特性上的应用
CN116925239B (zh) * 2023-07-17 2024-10-18 苏州星奥拓维生物技术有限公司 双载体系统表达Otof基因的组合物和方法
WO2025201333A1 (zh) * 2024-03-28 2025-10-02 苏州星奥拓维生物技术有限公司 递送Otof基因的双载体系统和其用途

Family Cites Families (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU4445701A (en) * 2000-03-24 2001-10-03 Centre National De La Recherche Scientifique-Cnrs Multiple human and mouse otoferlin isoforms
WO2005071092A1 (ja) 2004-01-22 2005-08-04 Dnavec Research Inc. サイトメガロウイルスエンハンサーおよびニワトリβ-アクチンプロモーターを含むハイブリッドプロモーターを利用したマイナス鎖RNAウイルスベクターの製造方法
WO2007127428A2 (en) 2006-04-28 2007-11-08 University Of Florida Research Foundation, Inc. Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery
JP5244103B2 (ja) 2006-08-09 2013-07-24 ホームステッド クリニカル コーポレイション 器官特異的蛋白質およびその使用方法
US7892824B2 (en) 2007-01-18 2011-02-22 University Of Missouri-Columbia Synthetic mini/micro-dystrophin genes to restore nNOS to the sarcolemma
FR2919305B1 (fr) 2007-07-26 2009-09-18 Genethon Ass Loi De 1901 Vecteurs viraux adeno-associes pour l'expression de la dysferline.
US8236557B2 (en) * 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
WO2010062905A2 (en) * 2008-11-26 2010-06-03 The Regents Of The University Of California Lateral ventricle cell compositions and use for treating neural degenerative diseases
KR20200047642A (ko) * 2010-04-23 2020-05-07 유니버시티 오브 플로리다 리서치 파운데이션, 인크. 레베르 선천성 흑내장-1(lca1)의 치료를 위한 재조합 아데노-관련된 바이러스-구아닐레이트 사이클라제 조성물 및 방법
WO2013075008A1 (en) 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
WO2013151663A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of membrane proteins
CA2904164C (en) 2013-03-11 2022-10-18 Fondazione Telethon Mir-204 and mir-211 and uses thereof
EP2986635B1 (en) 2013-04-18 2018-10-03 Fondazione Telethon Effective delivery of large genes by dual aav vectors
KR102298579B1 (ko) 2013-05-21 2021-09-07 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도
US11000597B2 (en) 2015-01-21 2021-05-11 University Of Florida Research Foundation, Incorporated Engineered receptor/ligand system for delivery of therapeutic agents
WO2016131981A1 (en) * 2015-02-20 2016-08-25 Institut Pasteur Prevention and/or treatment of hearing loss or impairment
US20180327779A1 (en) * 2015-03-03 2018-11-15 Fondazione Telethon (IT/IT) Multiple vector system and uses thereof
JP2018531926A (ja) 2015-09-17 2018-11-01 コーダ バイオセラピューティクス， インコーポレイテッド 神経学的障害を処置するための組成物および方法
CN116236591B (zh) 2015-12-11 2026-02-27 马萨诸塞眼科耳科诊所 用于将核酸递送至耳蜗和前庭细胞的材料和方法
EP3393522B1 (en) 2015-12-22 2021-07-07 INSERM (Institut National de la Santé et de la Recherche Médicale) Improved hybrid dual recombinant aav vector systems for gene therapy
WO2017216560A1 (en) 2016-06-15 2017-12-21 Oxford University Innovation Limited Dual overlapping adeno-associated viral vector system for expressing abc4a
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
WO2018162748A1 (en) 2017-03-10 2018-09-13 Genethon Treatment of glycogen storage disease iii
JP7240675B2 (ja) 2017-05-05 2023-03-16 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． オトフェルリンを発現させるための組成物および方法
IL276764B2 (en) 2018-02-22 2026-05-01 Akouos Inc Compositions and methods for treating non-age-related hearing impairment in humans
AU2019237541B2 (en) 2018-03-23 2025-02-13 Editas Medicine, Inc. CRISPR/Cas9-mediated exon-skipping approach for USH2A-associated Usher syndrome
WO2020093018A1 (en) 2018-11-01 2020-05-07 University Of Florida Research Foundation, Incorporated A codon optimized otoferlin aav dual vector gene therapy
BR112021013996A2 (pt) 2019-01-18 2021-10-26 Institut Pasteur Terapia gênica mediada por aav que recupera o gene otoferlina
JP7665609B2 (ja) 2019-10-30 2025-04-21 デシベル セラピューティクス インコーポレイテッド オトフェリン二重ベクター系及びそれを含む感音難聴を治療するための組成物
CN116096905A (zh) 2020-02-21 2023-05-09 阿库斯股份有限公司 用于治疗人受试者的非年龄相关性听力损伤的组合物和方法
CA3174500A1 (en) 2020-04-01 2021-10-07 University Of Florida Research Foundation, Incorporated Dual aav-myo7a vectors with improved safety for the treatment of ush1b

Also Published As

Publication number Publication date
JP2025063057A (ja) 2025-04-15
EP3635100A1 (en) 2020-04-15
JP7240675B2 (ja) 2023-03-16
AU2024203084A1 (en) 2024-05-30
US20250367325A1 (en) 2025-12-04
AU2024203084B2 (en) 2026-04-02
AU2018261769B2 (en) 2024-03-14
US20200157573A1 (en) 2020-05-21
WO2018204734A8 (en) 2018-12-06
AU2018261769A1 (en) 2019-11-21
US12359221B2 (en) 2025-07-15
CN110892062A (zh) 2020-03-17
CA3061955A1 (en) 2018-11-08
EP3635100A4 (en) 2021-03-24
JP7617600B2 (ja) 2025-01-20
JP2020518268A (ja) 2020-06-25
JP2023070682A (ja) 2023-05-19
KR102606810B1 (ko) 2023-11-27
JP7851563B2 (ja) 2026-04-27
KR20200003881A (ko) 2020-01-10
WO2018204734A1 (en) 2018-11-08
KR20230167138A (ko) 2023-12-07
MX2023013435A (es) 2023-12-12

Similar Documents

Publication Publication Date Title
MX2023013435A (es) Composiciones y metodos para expresar otoferlina.
SA520420518B1 (ar) تبديل الانتحاء النسيجي للفَيروسات المُرتَبِط بالفَيروس الغُدِّيّ
WO2018204803A8 (en) Compositions and methods of treating huntington's disease
JOP20200228A1 (ar) تركيبات وطرق لخفض تعبير البروتين tau
MX2020001187A (es) Composiciones y métodos para la administración de virus adenoasociados.
AU2018338314A1 (en) Protein degraders and uses thereof
WO2019006418A3 (en) ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
AU2018263868A1 (en) Formulations of anti-LAG3 antibodies and co-formulations of anti-LAG3 antibodies and anti-PD-1 antibodies
WO2016172658A3 (en) Microbiome regulators and related uses thereof
EP3735976A3 (en) Therapeutic use of mitochondria and combined mitochondrial agents
AU2017261336A1 (en) Modulators of the integrated stress pathway
AU2017260367A1 (en) Modulators of the integrated stress pathway
MX2018009325A (es) Compuestos y metodos para tratar enfermedades mediadas por el acido ribonucleico (arn).
EP4610279A3 (en) Compositions and methods of inhibiting masp-3 for the treatment of various diseases and disorders
EA201891202A1 (ru) Композиции, содержащие бактериальные штаммы
EA201890725A1 (ru) Агонисты фарнезоидного х-рецептора и их применение
EA201891201A1 (ru) Композиции, содержащие бактериальные штаммы
EA201890050A1 (ru) Композиции, содержащие бактериальные штаммы
WO2015153800A3 (en) Compositions for modulating sod-1 expression
EA201890049A1 (ru) Композиции, содержащие бактериальные штаммы
AU2017260374A1 (en) Modulators of the integrated stress pathway
WO2015168149A3 (en) Sensorineural hair cell differentiation
WO2020093018A8 (en) A codon optimized otoferlin aav dual vector gene therapy
PH12019502025A1 (en) Anti-tigit antigen-binding proteins and methods of use thereof
MX2021006674A (es) Composiciones para estabilizar bacterias y usos de las mismas.