RU2021134920A - Композиции и способы деградации неправильно упакованных белков - Google Patents

Композиции и способы деградации неправильно упакованных белков Download PDF

Info

Publication number
RU2021134920A
RU2021134920A RU2021134920A RU2021134920A RU2021134920A RU 2021134920 A RU2021134920 A RU 2021134920A RU 2021134920 A RU2021134920 A RU 2021134920A RU 2021134920 A RU2021134920 A RU 2021134920A RU 2021134920 A RU2021134920 A RU 2021134920A
Authority
RU
Russia
Prior art keywords
disease
composition
disorder
trim11
protein
Prior art date
Application number
RU2021134920A
Other languages
English (en)
Russian (ru)
Inventor
Сяолу ЯН
Лили ГО
Original Assignee
Дзе Трастиз Оф Дзе Юнивёрсити Оф Пенсильвания
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 Дзе Трастиз Оф Дзе Юнивёрсити Оф Пенсильвания filed Critical Дзе Трастиз Оф Дзе Юнивёрсити Оф Пенсильвания
Publication of RU2021134920A publication Critical patent/RU2021134920A/ru

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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19Cytokines; Lymphokines; Interferons
    • A61K38/21Interferons [IFN]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/395Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • A61P11/12Mucolytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5008Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
    • G01N33/5044Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics involving specific cell types
    • G01N33/5058Neurological cells
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/53Immunoassay; Biospecific binding assay; Materials therefor
    • G01N33/566Immunoassay; Biospecific binding assay; Materials therefor using specific carrier or receptor proteins as ligand binding reagents where possible specific carrier or receptor proteins are classified with their target compounds
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • G01N33/6896Neurological disorders, e.g. Alzheimer's disease
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/505Medicinal preparations containing antigens or antibodies comprising antibodies
    • 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
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10041Use of virus, viral particle or viral elements as a vector
    • C12N2710/10043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/28Neurological disorders
    • G01N2800/2814Dementia; Cognitive disorders

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Organic Chemistry (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Cell Biology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Analytical Chemistry (AREA)
  • Food Science & Technology (AREA)
  • Pathology (AREA)
  • General Physics & Mathematics (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
RU2021134920A 2015-05-29 2016-05-27 Композиции и способы деградации неправильно упакованных белков RU2021134920A (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562168309P 2015-05-29 2015-05-29
US62/168,309 2015-05-29

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
RU2017144862A Division RU2761564C9 (ru) 2015-05-29 2016-05-27 Композиции и способы деградации неправильно упакованных белков

Publications (1)

Publication Number Publication Date
RU2021134920A true RU2021134920A (ru) 2022-04-15

Family

ID=57441753

Family Applications (2)

Application Number Title Priority Date Filing Date
RU2021134920A RU2021134920A (ru) 2015-05-29 2016-05-27 Композиции и способы деградации неправильно упакованных белков
RU2017144862A RU2761564C9 (ru) 2015-05-29 2016-05-27 Композиции и способы деградации неправильно упакованных белков

Family Applications After (1)

Application Number Title Priority Date Filing Date
RU2017144862A RU2761564C9 (ru) 2015-05-29 2016-05-27 Композиции и способы деградации неправильно упакованных белков

Country Status (15)

Country Link
US (2) US11542500B2 (https=)
EP (1) EP3302706A4 (https=)
JP (3) JP7011467B2 (https=)
KR (2) KR20240108531A (https=)
CN (1) CN108271364A (https=)
AU (3) AU2016271635B2 (https=)
BR (1) BR112017025574A2 (https=)
CA (1) CA2987057A1 (https=)
HK (1) HK1252757A1 (https=)
IL (3) IL255912B2 (https=)
MX (3) MX2017015373A (https=)
RU (2) RU2021134920A (https=)
SG (1) SG10202011833TA (https=)
WO (1) WO2016196328A1 (https=)
ZA (1) ZA201708067B (https=)

Families Citing this family (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
CA3193811A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
CN119876138A (zh) 2014-11-14 2025-04-25 沃雅戈治疗公司 调节性多核苷酸
CA3024448C (en) 2016-05-18 2025-09-09 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
CN110753557B (zh) * 2017-03-22 2024-03-22 神济昌华(北京)生物科技有限公司 Trim72通过泛素化fus蛋白突变体作为als的潜在治疗性靶标
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CN109609614B (zh) * 2017-09-30 2022-07-15 首都医科大学附属北京胸科医院 检测trim2、trim4、trim32和/或trim46基因或蛋白产品的用途
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
CN108018303A (zh) * 2017-12-20 2018-05-11 江苏浩欧博生物医药股份有限公司 一种人Ro52重组蛋白的制备方法
WO2019175881A1 (en) 2018-03-14 2019-09-19 Technion Research & Development Foundation Limited A prognostic biomarker in cancer
SG11202011296VA (en) 2018-05-15 2020-12-30 Voyager Therapeutics Inc Compositions and methods for the treatment of parkinson's disease
US11147815B2 (en) 2018-08-21 2021-10-19 Synkrino Biotherapeutics, Inc. Compositions and methods for treating diseases associated with uncontrolled inflammatory responses
EP3863642A4 (en) * 2018-10-09 2022-06-29 The Regents of the University of California Covalent targeting of e3 ligases
WO2020227515A1 (en) * 2019-05-07 2020-11-12 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
CN110302383A (zh) * 2019-07-29 2019-10-08 深圳先进技术研究院 Trim11基因和蛋白靶点以及抑制剂在乳腺癌中的用途
CN110669791B (zh) * 2019-10-29 2020-08-04 成都益安博生物技术有限公司 一种提高细胞抗体表达量的方法
KR102114880B1 (ko) * 2020-01-15 2020-05-26 서울대학교병원 코돈 최적화된 TIF1γ 폴리뉴클레오티드를 포함하는 재조합 벡터 및 이의 용도
GB202005876D0 (en) * 2020-04-22 2020-06-03 Univ Dundee Protein degradation
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
CN113292658B (zh) * 2021-04-17 2023-02-28 复旦大学 一种融合蛋白及其在靶向降解细胞内蛋白中的应用
CN113651879A (zh) * 2021-08-18 2021-11-16 武汉华美生物工程有限公司 一种trim21全长蛋白的制备方法及应用
CN117159747B (zh) * 2023-08-30 2024-12-20 暨南大学 一种亨廷顿舞蹈症的药物及其制备方法
CN118272444A (zh) * 2024-02-29 2024-07-02 宜昌市中心人民医院(三峡大学第一临床医学院、三峡大学附属中心人民医院) 一种慢病毒载体LV-Trim69及其在制备改善心脏衰老药物上的应用
CN120209082B (zh) * 2025-05-28 2025-08-05 中南大学 一种靶向阻断trim25与brd7蛋白结合的多肽及其应用

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20010027182A1 (en) * 1998-05-29 2001-10-04 Huda Y. Zoghbi Chaperone suppression of ataxin-1 aggregation and altered subcellular proteasome localization imply protein misfilind in sca1
WO2001090342A2 (en) 2000-05-22 2001-11-29 Johns Hopkins University Genetic engineering of vascular grafts to resist disease
US20040235733A1 (en) * 2003-02-27 2004-11-25 Steffan Joan S. Methods and reagents for reducing polyglutamine toxicity
US20050215562A1 (en) * 2003-06-23 2005-09-29 Patrick Tremblay Methods for treating protein aggregation disorders
WO2005081911A2 (en) * 2004-02-25 2005-09-09 Dana-Farber Cancer Institute, Inc. METHODS AND COMPOSITIONS FOR THE TREATMENT AND PREVENTION OF HIV INFECTION USING TRIM5α
US20140056873A1 (en) 2004-06-10 2014-02-27 University Of Leicester Methods for Treating Conditions Associated with MASP-2 Dependent Complement Activation
WO2006116716A2 (en) * 2005-04-27 2006-11-02 University Of Florida Materials and methods for enhanced degradation of mutant proteins associated with human disease
EP1870107A1 (en) * 2006-06-23 2007-12-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Use of inducer of promyelocytic leukemia protein (PML) for treating polyglutamine expansion neurodegenerative diseases
US7785857B2 (en) 2006-08-31 2010-08-31 Saint Louis University Protein C variant
CA2749163A1 (en) 2009-01-14 2010-07-22 The Salk Institute For Biological Studies Methods for screening and compounds that protect against amyloid diseases
EP2826860B1 (en) * 2010-04-23 2018-08-22 University of Massachusetts CNS targeting AAV vectors and methods of use thereof
GB201012410D0 (en) * 2010-07-23 2010-09-08 Medical Res Council Intracellular immunity
AU2011295629B2 (en) 2010-09-01 2014-05-01 Organ Transport Pty Ltd Perfusion composition
CN103127492A (zh) * 2011-11-23 2013-06-05 中国科学院上海巴斯德研究所 一种抗丙型肝炎病毒的蛋白及其用途
TR201905612T4 (tr) * 2012-12-05 2019-05-21 Univ Heidelberg Ruprecht Karls Proteinlerin ve çok değerlikli, hücreye nüfuz eden peptitlerin konjugatları ve bunların kullanımları.
US20160136123A1 (en) * 2013-06-14 2016-05-19 Vojo P. Deretic Treatment of autophagy-related disorders
WO2016054554A1 (en) * 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs

Also Published As

Publication number Publication date
JP2024161501A (ja) 2024-11-19
RU2017144862A (ru) 2019-07-02
RU2761564C2 (ru) 2021-12-10
US20180163202A1 (en) 2018-06-14
US11542500B2 (en) 2023-01-03
RU2017144862A3 (https=) 2019-11-20
IL302965B1 (en) 2025-08-01
MX2024001616A (es) 2024-02-28
RU2761564C9 (ru) 2022-03-17
KR20240108531A (ko) 2024-07-09
IL322085A (en) 2025-09-01
BR112017025574A2 (en) 2018-08-14
AU2022200673A1 (en) 2022-02-24
CN108271364A (zh) 2018-07-10
JP2018521014A (ja) 2018-08-02
IL302965B2 (en) 2025-12-01
IL302965A (en) 2023-07-01
ZA201708067B (en) 2018-11-28
IL255912B1 (en) 2023-06-01
KR20180011276A (ko) 2018-01-31
AU2025230665A1 (en) 2025-10-02
MX2021014954A (es) 2022-01-31
CA2987057A1 (en) 2016-12-08
JP2022000459A (ja) 2022-01-04
JP7011467B2 (ja) 2022-02-10
EP3302706A1 (en) 2018-04-11
US20230313190A1 (en) 2023-10-05
WO2016196328A1 (en) 2016-12-08
EP3302706A4 (en) 2019-05-08
AU2016271635A1 (en) 2018-01-25
MX2017015373A (es) 2018-07-06
SG10202011833TA (en) 2021-01-28
AU2022200673B2 (en) 2025-06-05
IL255912B2 (en) 2023-10-01
HK1252757A1 (zh) 2019-05-31
IL255912A (en) 2018-01-31
AU2016271635B2 (en) 2021-11-04

Similar Documents

Publication Publication Date Title
RU2021134920A (ru) Композиции и способы деградации неправильно упакованных белков
JP2018521014A5 (https=)
JP7374119B2 (ja) 減少した肝臓向性を有するAAV9とAAVrh74とのハイブリッド組換えアデノ随伴ウイルス血清型
Ramaswamy et al. Gene therapy for Huntington's disease
HRP20250897T1 (hr) Genska terapija za neurodegenerativne poremećaje
Dantuma et al. Stem cells for the treatment of neurodegenerative diseases
CO2020004738A2 (es) Variantes de cápsides de virus adenoasociados y métodos de uso de estas
JP2024540221A (ja) Aavカプシドバリアント及びその使用
BR112022004027A2 (pt) Métodos para tratar doenças neovasculares oculares usando variantes de aav2 que codificam aflibercepte
MX2020008932A (es) Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos.
CL2018003196A1 (es) Cápsides variantes de virus adenoasociados y métodos de uso de estas.
WO2018204803A8 (en) Compositions and methods of treating huntington's disease
BR112015027336A2 (pt) composições e processos para tratamento de atrofia muscular espinhal
JP2018502112A5 (https=)
PH12021550254A1 (en) Variant rnai against alpha-synuclein
MX2021006253A (es) Terapias génicas para enfermedad neurodegenerativa.
UY38881A (es) Métodos de tratamiento de enfermedades neovasculares oculares utilizando variantes de aav2 que codifican aflibercept
WO2023039503A3 (en) App irna compositions and methods of use thereof for treating or preventing diseases characterized by enlarged endosomes
MX2024006612A (es) Oligonucleotidos antisentido de cambio de empalme con estructuras quimicas modificadas.
Zhang et al. Celastrol enhances AAV1-mediated gene expression in mice adipose tissues
Chan et al. Astrocytes go awry in Huntington's disease
FI3873511T3 (fi) Ekspressiovektori kolesteroli-24-hydrolaasille amyotroofisen lateraaliskleroosin hoitoon
FI3574091T3 (fi) Ekspressiovektori kolesterolin 24-hydrolaasia varten polyglutamiinin toiston spinoserebellaariataksioiden hoidossa
Passineau Salivary gland gene therapy in experimental and clinical trials
Ma et al. Intranasal delivery of recombinant NT4-NAP/AAV exerts potential antidepressant effect