JOP20210160A1 - تركيبات لخفض التعبير عن الجين الناقل بشكل انتقائي لـ drg - Google Patents

تركيبات لخفض التعبير عن الجين الناقل بشكل انتقائي لـ drg

Info

Publication number
JOP20210160A1
JOP20210160A1 JOP/2021/0160A JOP20210160A JOP20210160A1 JO P20210160 A1 JOP20210160 A1 JO P20210160A1 JO P20210160 A JOP20210160 A JO P20210160A JO P20210160 A1 JOP20210160 A1 JO P20210160A1
Authority
JO
Jordan
Prior art keywords
drg
expression
cells
combinations
aav
Prior art date
Application number
JOP/2021/0160A
Other languages
Arabic (ar)
English (en)
Inventor
James M Wilson
Juliette Hordeaux
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of JOP20210160A1 publication Critical patent/JOP20210160A1/ar

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • A61K35/761Adenovirus
    • 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
    • A61K48/005Medicinal 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
    • A61P25/00Drugs for disorders of the nervous system
    • 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
    • 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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]
    • C12N2310/141MicroRNAs, miRNAs
    • 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)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Mycology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JOP/2021/0160A 2018-12-21 2019-12-20 تركيبات لخفض التعبير عن الجين الناقل بشكل انتقائي لـ drg JOP20210160A1 (ar)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862783956P 2018-12-21 2018-12-21
US201962924970P 2019-10-23 2019-10-23
US201962934915P 2019-11-13 2019-11-13
PCT/US2019/067872 WO2020132455A1 (en) 2018-12-21 2019-12-20 Compositions for drg-specific reduction of transgene expression

Publications (1)

Publication Number Publication Date
JOP20210160A1 true JOP20210160A1 (ar) 2023-01-30

Family

ID=71100588

Family Applications (1)

Application Number Title Priority Date Filing Date
JOP/2021/0160A JOP20210160A1 (ar) 2018-12-21 2019-12-20 تركيبات لخفض التعبير عن الجين الناقل بشكل انتقائي لـ drg

Country Status (17)

Country Link
US (1) US20210077553A1 (enExample)
EP (1) EP3908326A4 (enExample)
JP (1) JP7660062B2 (enExample)
KR (1) KR20210107037A (enExample)
CN (1) CN113646005A (enExample)
AU (1) AU2019401314A1 (enExample)
BR (1) BR112021011143A2 (enExample)
CA (1) CA3123600A1 (enExample)
CL (1) CL2021001624A1 (enExample)
CO (1) CO2021008538A2 (enExample)
IL (1) IL284185B2 (enExample)
JO (1) JOP20210160A1 (enExample)
MX (1) MX2021007600A (enExample)
PE (1) PE20211581A1 (enExample)
PH (1) PH12021551341A1 (enExample)
SG (1) SG11202105907QA (enExample)
WO (1) WO2020132455A1 (enExample)

Families Citing this family (62)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112021021908A2 (pt) 2019-05-03 2022-02-01 Univ Pennsylvania Composições úteis no tratamento da leucodistrofia metacromática
WO2021211753A1 (en) 2020-04-15 2021-10-21 Voyager Therapeutics, Inc. Tau binding compounds
CN115803064A (zh) * 2020-05-12 2023-03-14 宾夕法尼亚州大学信托人 用于drg特异性降低转基因表达的组合物
PE20230767A1 (es) 2020-05-13 2023-05-09 Voyager Therapeutics Inc Redireccion del tropismo de las capsides de aav
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
US20230220069A1 (en) 2020-06-17 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of gene therapy patients
EP4179097A1 (en) 2020-07-13 2023-05-17 The Trustees of The University of Pennsylvania Compositions useful for treatment of charcot-marie-tooth disease
EP4189095A1 (en) 2020-07-27 2023-06-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
US20230285596A1 (en) 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
KR20230118075A (ko) * 2020-10-09 2023-08-10 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 파브리병 치료를 위한 조성물 및 방법
US20230383313A1 (en) 2020-10-18 2023-11-30 The Trustees Of The University Of Pennsylvania Improved adeno-associated virus (aav) vector and uses therefor
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
IL303239A (en) 2020-12-01 2023-07-01 Univ Pennsylvania Compositions and their uses for the treatment of Engelmann syndrome
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022198079A1 (en) * 2021-03-19 2022-09-22 Mayo Foundation For Medical Education And Research Methods and materials for treating propionic acidemia
US20230034817A1 (en) 2021-04-12 2023-02-02 Affinia Therapeutics Inc. Recombinant aav for treatment of neural disease
WO2023113806A1 (en) 2021-12-16 2023-06-22 Affinia Therapeutics, Inc. Recombinant aav for treatment of neural disease
AR125406A1 (es) 2021-04-23 2023-07-12 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen
WO2022232267A1 (en) 2021-04-27 2022-11-03 The Trustees Of The University Of Pennsylvania Porcine-derived adeno-associated virus capsids and uses thereof
US20250223376A1 (en) 2021-09-20 2025-07-10 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
WO2023056399A1 (en) 2021-10-02 2023-04-06 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
JP2024542950A (ja) * 2021-10-18 2024-11-19 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Cdkl5欠損症(cdd)の治療において有用な組成物
US20250295807A1 (en) * 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
EP4433169A1 (en) 2021-11-17 2024-09-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
US20250034559A1 (en) 2021-11-17 2025-01-30 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
WO2023091949A2 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
TW202340467A (zh) 2022-01-10 2023-10-16 賓州大學委員會 有用於治療c9orf72介導之病症之組成物及方法
EP4469585A2 (en) 2022-01-25 2024-12-04 Voyager Therapeutics, Inc. Baculovirus expression system
WO2023154693A1 (en) 2022-02-08 2023-08-17 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
KR20250007064A (ko) 2022-04-06 2025-01-13 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Her2 양성 전이성 유방암 및 기타 암을 치료하기 위한 조성물 및 방법
WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
WO2023220695A2 (en) 2022-05-13 2023-11-16 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
CA3257081A1 (en) 2022-06-02 2023-12-07 Voyager Therapeutics, Inc. VAA CAPSIDE VARIANTS AND THEIR USES
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
EP4543473A1 (en) 2022-06-22 2025-04-30 Voyager Therapeutics, Inc. Tau binding compounds
CA3259902A1 (en) 2022-06-28 2024-01-04 Voyager Therapeutics, Inc. AAV Capsid Variants and Their Uses
TW202424201A (zh) 2022-07-06 2024-06-16 美商航海家醫療公司 Aav殼體變異體及其用途
AU2023304708A1 (en) * 2022-07-08 2025-01-23 Consiglio Nazionale Delle Ricerche Transgene cassettes
AU2023320453A1 (en) 2022-08-03 2025-02-06 Voyager Therapeutics, Inc. Compositions and methods for crossing the blood brain barrier
CN120019071A (zh) 2022-09-15 2025-05-16 沃雅戈治疗公司 Tau结合化合物
EP4594494A2 (en) * 2022-09-26 2025-08-06 Encoded Therapeutics, Inc. Elements for de-targeting gene expression in dorsal root ganglion and/or liver
EP4634394A2 (en) 2022-12-17 2025-10-22 The Trustees of The University of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
KR20250129743A (ko) 2022-12-29 2025-08-29 보이저 테라퓨틱스, 인크. Mapt를 조절하기 위한 조성물 및 방법
WO2024163012A1 (en) 2023-02-02 2024-08-08 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2024226761A2 (en) 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
WO2024228943A1 (en) 2023-05-02 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
CN121127597A (zh) 2023-05-03 2025-12-12 沃雅戈治疗公司 用于治疗与葡糖苷酰鞘氨醇酶β1缺陷相关的病症的组合物及方法
WO2024229389A1 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
WO2024229425A1 (en) 2023-05-04 2024-11-07 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
CN121175424A (zh) * 2023-05-12 2025-12-19 上海金珂博生物技术有限公司 一种携带smn基因表达框的病毒载体及其用途
WO2025007046A1 (en) 2023-06-29 2025-01-02 The Trustees Of The University Of Pennsylvania Mutant aav with central nervous system targeting motifs and compositions containing same
WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
WO2025038805A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
WO2025106661A1 (en) 2023-11-14 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof
WO2025122634A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
WO2025122530A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
WO2025122644A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease
WO2025147436A1 (en) 2024-01-03 2025-07-10 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2025217023A1 (en) * 2024-04-08 2025-10-16 The Trustees Of The University Of Pennsylvania Gene therapy for gangliosidosis type 2 (gm2)
WO2025235734A2 (en) 2024-05-09 2025-11-13 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1959012A3 (en) * 2004-12-29 2009-12-30 Exiqon A/S Novel oligonucleotide compositions and probe sequences useful for detection and analysis of microRNAs and their target mRNAs
SI2002003T1 (sl) * 2005-05-27 2016-05-31 Ospedale San Raffaele S.R.L. Genski vektor, ki vsebuje mi-RNA
EP1966390A1 (en) * 2005-12-29 2008-09-10 Exiqon A/S Detection of tissue origin of cancer
KR101026502B1 (ko) * 2007-11-23 2011-04-01 주식회사 파나진 마이크로rna 안티센스 pna, 그를 포함하는 조성물, 및 그의 사용 및 평가 방법
US9217155B2 (en) * 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
EP3540055A1 (en) * 2010-04-23 2019-09-18 University of Massachusetts Cns targeting aav vectors and methods of use thereof
FR3004463A1 (fr) 2013-04-11 2014-10-17 Genethon Systeme d'expression pour une therapie genique selective
EP3411488A1 (en) * 2016-02-03 2018-12-12 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
JOP20190200A1 (ar) * 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي

Also Published As

Publication number Publication date
SG11202105907QA (en) 2021-07-29
JP7660062B2 (ja) 2025-04-10
CN113646005A (zh) 2021-11-12
IL284185B2 (en) 2025-04-01
BR112021011143A2 (pt) 2022-01-25
IL284185A (en) 2021-08-31
EP3908326A4 (en) 2022-10-26
JP2022517174A (ja) 2022-03-07
KR20210107037A (ko) 2021-08-31
CO2021008538A2 (es) 2021-07-19
MX2021007600A (es) 2021-08-11
CA3123600A1 (en) 2020-06-25
US20210077553A1 (en) 2021-03-18
PH12021551341A1 (en) 2021-12-13
EP3908326A1 (en) 2021-11-17
CL2021001624A1 (es) 2021-11-26
AU2019401314A1 (en) 2021-06-24
PE20211581A1 (es) 2021-08-17
IL284185B1 (en) 2024-12-01
WO2020132455A1 (en) 2020-06-25

Similar Documents

Publication Publication Date Title
JOP20210160A1 (ar) تركيبات لخفض التعبير عن الجين الناقل بشكل انتقائي لـ drg
MX2020005673A (es) Terapia génica para mucopolisacaridosis iiib.
WO2019108857A8 (en) Gene therapy for mucopolysaccharidosis iiia
PH12020550094A1 (en) Adeno-associated virus variant capsids and methods of use thereof
MX2020008932A (es) Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos.
PH12018501055A1 (en) Cpg reduced factor viii variants, compositions and methods and uses for treatment of hemostasis disorders
AR118850A1 (es) Composiciones útiles en el tratamiento de leucodistrofia metacromática
EA201991911A1 (ru) Способы и композиции для переноса генов по сосудистой сети
PE20160188A1 (es) Variantes aav y composiciones, metodos y usos para transferencia de gen a celulas, organos y tejidos
PH12021552638A1 (en) Compositions useful for treatment of pompe disease
RU2020108189A (ru) Композиции и способы лечения бокового амиотрофического склероза (als)
RU2016149206A (ru) Аденоассоциированные вирусные векторы для лечения лизосомных болезней накопления
MX2022014258A (es) Composiciones para la reducción específica de un fármaco de la expresión de transgén.
RU2017142006A (ru) Векторы на основе аденоассоциированных вирусов для лечения мукополисахаридозов
RU2019100525A (ru) Векторная система на основе аденоассоциированного вируса
CL2020000295A1 (es) Métodos de terapia génica del factor viii (fviii)
AR105822A1 (es) Análogos de insulina
MX2022015048A (es) Viriones de virus adenoasociados con variantes de capsides y metodos de uso de estos.
WO2022229703A3 (en) New aav8 based immune escaping variants
MX2023006694A (es) Tratamiento de la enfermedad de danon.
MX2023012509A (es) Vectores aavrh74 para la terapia génica de distrofias musculares.
AU2018261420A1 (en) Method for treating ischemic tissue
EA202191744A1 (ru) Композиции для drg-специфического снижения экспрессии трансгена
WO2022169861A3 (en) Gene therapy for angelman syndrome
CN111100847A (zh) 安全性更高的端粒基因治疗产品使人体健康逆转衰老