MX380973B - Composiciones y metodos para tratar la atrofia muscular espinal. - Google Patents

Composiciones y metodos para tratar la atrofia muscular espinal.

Info

Publication number
MX380973B
MX380973B MX2015015244A MX2015015244A MX380973B MX 380973 B MX380973 B MX 380973B MX 2015015244 A MX2015015244 A MX 2015015244A MX 2015015244 A MX2015015244 A MX 2015015244A MX 380973 B MX380973 B MX 380973B
Authority
MX
Mexico
Prior art keywords
muscular atrophy
spinal muscular
compositions
methods
treating spinal
Prior art date
Application number
MX2015015244A
Other languages
English (en)
Other versions
MX2015015244A (es
Inventor
Catherine R O'riordan
Lamya S Shihabuddin
Marco A Passini
Seng H Cheng
Original Assignee
Genzyme Corp
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 Genzyme Corp filed Critical Genzyme Corp
Publication of MX2015015244A publication Critical patent/MX2015015244A/es
Publication of MX380973B publication Critical patent/MX380973B/es

Links

Classifications

    • 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
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • A61K35/761Adenovirus
    • 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
    • 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
    • A61K48/0075Medicinal 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 delivery route, e.g. oral, subcutaneous
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0085Brain, e.g. brain implants; Spinal cord
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular 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
    • C12N15/63Introduction 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
    • C12N15/864Parvoviral vectors, e.g. parvovirus, densovirus
    • 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
    • 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
    • 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
    • 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
    • C12N2750/14171Demonstrated in vivo effect

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Organic Chemistry (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Microbiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Neurosurgery (AREA)
  • Dermatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Psychology (AREA)
  • Mycology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)

Abstract

La presente invención se refiere a métodos para tratar la atrofia muscular espinal usando una partícula viral de virus adenoasociado recombinante (rAAV) autocomplementario que comprende un transgene que expresa la SMN. En un aspecto, las partículas virales se administran en la médula espinal o cisterna magna en un sujeto humano; por ejemplo, un sujeto humano pediátrico. Están contempladas partículas virales que comprenden cápsides de AAV9.
MX2015015244A 2013-05-01 2013-05-01 Composiciones y metodos para tratar la atrofia muscular espinal. MX380973B (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
PCT/US2013/039163 WO2014178863A1 (en) 2013-05-01 2013-05-01 Compositions and methods for treating spinal muscular atrophy

Publications (2)

Publication Number Publication Date
MX2015015244A MX2015015244A (es) 2016-07-05
MX380973B true MX380973B (es) 2025-03-12

Family

ID=51843829

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2015015244A MX380973B (es) 2013-05-01 2013-05-01 Composiciones y metodos para tratar la atrofia muscular espinal.

Country Status (7)

Country Link
US (3) US10821154B2 (es)
KR (2) KR20200060536A (es)
AU (1) AU2013388083B2 (es)
BR (1) BR112015027336A2 (es)
CA (1) CA2911105A1 (es)
MX (1) MX380973B (es)
WO (1) WO2014178863A1 (es)

Families Citing this family (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP5879256B2 (ja) 2009-05-02 2016-03-08 ジェンザイム・コーポレーション 神経変性障害のための遺伝子治療
CN105377039A (zh) 2013-05-15 2016-03-02 明尼苏达大学董事会 腺相关病毒介导的基因向中枢神经系统转移
BR112017004056A2 (pt) 2014-09-12 2017-12-05 Biogen Ma Inc composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo
HK1245326A1 (zh) * 2014-12-12 2018-08-24 Voyager Therapeutics, Inc. 用於生产scaav的组合物和方法
LT3350220T (lt) 2015-09-15 2021-09-27 Scholar Rock, Inc. Anti pro/latentinio miostatino antikūnai ir jų panaudojimas
BR112018011975A2 (pt) * 2015-12-14 2018-12-11 Univ Pennsylvania composições úteis no tratamento de atrofia muscular espinhal
PL3411484T3 (pl) * 2016-02-05 2024-02-19 Emory University Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego
IL305449B2 (en) 2016-04-15 2026-01-01 Univ Pennsylvania Gene therapy for the treatment of mucocutaneous type ii diabetes
AU2017283546C1 (en) * 2016-06-13 2020-11-19 Scholar Rock, Inc. Use of myostatin inhibitors and combination therapies
WO2017218884A1 (en) * 2016-06-16 2017-12-21 Ionis Pharmaceuticals, Inc. Combinations for the modulation of smn expression
FI3568483T3 (fi) * 2017-01-10 2025-11-21 Childrens Medical Res Institute Polynukleotideja ja vektoreita siirtogeenien ilmentämiseen
JOP20190200A1 (ar) * 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
JP7455579B2 (ja) 2017-02-28 2024-03-26 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 新規アデノ随伴ウイルス(aav)クレードfベクター及びその用途
US20200370069A1 (en) * 2017-07-08 2020-11-26 Genethon Treatment of spinal muscular atrophy
JP7221275B2 (ja) 2017-08-03 2023-02-13 ボイジャー セラピューティクス インコーポレイテッド Aavを送達するための組成物および方法
MX2020002148A (es) * 2017-08-25 2020-07-20 Ovid Therapeutics Inc Vectores adenoasociados recombinantes.
TWI835747B (zh) 2017-09-22 2024-03-21 賓州大學委員會 用於治療黏多醣病 ii 型之基因治療
MX2020004830A (es) 2017-11-08 2020-11-11 Avexis Inc Medios y metodo para preparar vectores virales y usos de los mismos.
TW202015742A (zh) 2018-05-15 2020-05-01 美商航海家醫療公司 投遞腺相關病毒(aav)之組成物和方法
SG11202011296VA (en) 2018-05-15 2020-12-30 Voyager Therapeutics Inc Compositions and methods for the treatment of parkinson's disease
EP3793615A2 (en) 2018-05-16 2021-03-24 Voyager Therapeutics, Inc. Directed evolution of aav to improve tropism for cns
CN112601557B (zh) * 2018-06-08 2025-06-06 诺华股份有限公司 用于测量药物产品效力的基于细胞的测定
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
WO2020077165A1 (en) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
WO2020113034A1 (en) * 2018-11-30 2020-06-04 Avexis, Inc. Aav viral vectors and uses thereof
JP2022522995A (ja) 2019-01-18 2022-04-21 ボイジャー セラピューティクス インコーポレイテッド Aav粒子を生成するための方法及びシステム
MA55625A (fr) * 2019-04-12 2022-02-16 Encoded Therapeutics Inc Compositions et méthodes d'administration de produits thérapeutiques
US20220280548A1 (en) * 2019-08-15 2022-09-08 Biogen Ma Inc. Combination therapy for spinal muscular atrophy
US20220333133A1 (en) 2019-09-03 2022-10-20 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
KR20210104961A (ko) 2020-02-18 2021-08-26 방윤배 안전난간을 가진 사다리
CR20220485A (es) 2020-02-28 2022-11-10 Ionis Pharmaceuticals Inc Compuestos y métodos para modular smn2
RU2742837C1 (ru) * 2020-06-02 2021-02-11 Общество С Ограниченной Ответственностью "Анабион" Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок SMN1, и ее применение
US20230285596A1 (en) 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
BR112023001456A2 (pt) 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
WO2022028472A1 (en) * 2020-08-05 2022-02-10 Hangzhou Jiayin Biotech Ltd. Nucleic acid constructs and uses thereof for treating spinal muscular atrophy
CN116179605B (zh) * 2021-08-12 2025-11-11 蓝图生物医药(广州)有限公司 一种重组腺相关病毒载体及其应用
EP4405396A2 (en) 2021-09-20 2024-07-31 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
CN118119710A (zh) * 2021-10-15 2024-05-31 上海天泽云泰生物医药有限公司 用于治疗脊髓性肌萎缩的重组腺相关病毒载体
EP4433490A2 (en) 2021-11-17 2024-09-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2023097214A1 (en) * 2021-11-23 2023-06-01 University Of Massachusetts Gene therapy for spinal muscular atrophy
KR102717399B1 (ko) * 2022-05-10 2024-10-16 서울대학교산학협력단 인간 smn1 단백질 변이체 및 이의 용도
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
CN115323002A (zh) * 2022-07-28 2022-11-11 深圳先进技术研究院 一种基因递送系统在从脑部逆行递送基因到脊髓神经元中的应用
IL321628A (en) 2022-12-22 2025-08-01 Scholar Rock Inc Selective and potent inhibitory antibodies for myostatin activation
AU2023427408A1 (en) 2023-02-02 2025-09-04 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5328470A (en) 1989-03-31 1994-07-12 The Regents Of The University Of Michigan Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
PT1916258E (pt) 1999-08-09 2014-07-29 Genzyme Corp Aumento da expressão de uma sequência nucleotídica heteróloga de cadeia simples a partir de vectores virais recombinantes por concepção da sequência de maneira que esta forme pares de bases intracadeia
NZ522840A (en) 2000-06-01 2004-12-24 Univ North Carolina A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together
WO2003004660A1 (en) * 2001-07-03 2003-01-16 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods of administering vectors to synaptically connected neurons
US7765583B2 (en) 2005-02-28 2010-07-27 France Telecom System and method for managing virtual user domains
PL1879623T3 (pl) 2005-05-02 2013-03-29 Genzyme Corp Terapia genowa zaburzeń rdzenia kręgowego
WO2007146046A2 (en) * 2006-06-07 2007-12-21 Genzyme Corporation Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
EP2019143A1 (en) 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
WO2010071832A1 (en) * 2008-12-19 2010-06-24 Nationwide Children's Hospital Delivery of polynucleotides across the blood brain barrier using recombinant aav9
JP5879256B2 (ja) * 2009-05-02 2016-03-08 ジェンザイム・コーポレーション 神経変性障害のための遺伝子治療

Also Published As

Publication number Publication date
BR112015027336A2 (pt) 2017-09-26
KR20160003792A (ko) 2016-01-11
AU2013388083B2 (en) 2019-08-22
US20210121523A1 (en) 2021-04-29
MX2015015244A (es) 2016-07-05
WO2014178863A1 (en) 2014-11-06
KR20200060536A (ko) 2020-05-29
KR102116378B1 (ko) 2020-06-01
US20240000886A1 (en) 2024-01-04
US20160074474A1 (en) 2016-03-17
US10821154B2 (en) 2020-11-03
AU2013388083A1 (en) 2015-12-03
CA2911105A1 (en) 2014-11-06

Similar Documents

Publication Publication Date Title
MX380973B (es) Composiciones y metodos para tratar la atrofia muscular espinal.
ZA201801657B (en) Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same
PE20150163A1 (es) Composicion y metodos para una transferencia de genes altamente eficiente usando variantes capside aav
BR112018071180A2 (pt) métodos de reforço potência biológica de baculovirus system-produzido vírus adeno-associado recombinante
PH12017501436A1 (en) Enhanced delivery of viral particles to the striatum and cortex
MX2015011951A (es) Proteinas de fusion que comprenden partes de union a pdgf y vegf y metodos de uso de las mismas.
MX376190B (es) PARTÍCULAS DE rAAV QUE CODIFICAN miR-708 Y USOS DEL MISMO.
MX2015006234A (es) Uso de akkermansia para tratar trastornos metabolicos.
WO2014145599A3 (en) Recombinant virus and preparations thereof
PH12017501942A1 (en) Oncolytic adenovirus encoding a b7 protein
MX2019001938A (es) Metodos para detectar aav.
BR112016020688A2 (pt) Composições úteis no tratamento da deficiência da ornitina transcarbamilase (otc)
BR112018011975A2 (pt) composições úteis no tratamento de atrofia muscular espinhal
MX384795B (es) Vectores que comprenden secuencias de polinucleotidos rellenadoras/de relleno y metodos de uso.
BR112017021505A2 (pt) produção de vetores adenoassociados superdimensionados
EA201592014A1 (ru) Доставка нацеленных на экзон 2 полинуклеотидных конструкций u7snrna при помощи рекомбинантного аденоассоциированного вируса
PH12021552887A1 (en) Modified viral particles and uses thereof
NZ628385A (en) Batches of recombinant adenovirus with altered terminal ends
BR112016002317A8 (pt) Uso de benralizumab ou de um seu fragmento de ligação ao antígeno para aumentar o volume expiratório forçado em asmáticos e benralizumab ou de um seu fragmento de ligação ao antígeno para uso para aumentar o volume expiratório forçado em asmáticos
WO2015031347A3 (en) Cross-linked platelet material
MX2015009891A (es) Composiciones farmaceuticas que comprenden particulas cristalinas de multicomponentes adecuadas para usar en terapia de inhalacion.
MX2018007330A (es) Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii.
SG11201704919SA (en) Methods of purifying adeno-associated virus (aav) and/or recombinant adeno-associated virus (raav)
EP3500279A4 (en) METHODS AND COMPOSITIONS FOR THE TREATMENT OF CANINE CONDITIONS USING A RECOMBINANT SELF-COMPLEMENTARY ADENO-ASSOCIATED VIRUS.
BR112017004770A2 (pt) partícula tipo flavivírus