MX375360B - Vectores de virus adeno-asociado para el tratamiento de la enfermedad de almacenamiento de glucógeno. - Google Patents

Vectores de virus adeno-asociado para el tratamiento de la enfermedad de almacenamiento de glucógeno.

Info

Publication number
MX375360B
MX375360B MX2016006774A MX2016006774A MX375360B MX 375360 B MX375360 B MX 375360B MX 2016006774 A MX2016006774 A MX 2016006774A MX 2016006774 A MX2016006774 A MX 2016006774A MX 375360 B MX375360 B MX 375360B
Authority
MX
Mexico
Prior art keywords
g6pc
storage disease
glycogen storage
coding sequence
intron
Prior art date
Application number
MX2016006774A
Other languages
English (en)
Other versions
MX2016006774A (es
Inventor
Barry J Byrne
Janice J Chou
Original Assignee
Us Health
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
Application filed by Us Health, Univ Florida filed Critical Us Health
Publication of MX2016006774A publication Critical patent/MX2016006774A/es
Publication of MX375360B publication Critical patent/MX375360B/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
    • C12N15/63—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
    • A61K48/0008—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 '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
    • 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
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/06—Antigout agents, e.g. antihyperuricemic or uricosuric agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
    • C12Y301/03009—Glucose-6-phosphatase (3.1.3.9)
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Endocrinology (AREA)
  • Emergency Medicine (AREA)
  • Immunology (AREA)
  • Rheumatology (AREA)
  • Urology & Nephrology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pain & Pain Management (AREA)
  • Physical Education & Sports Medicine (AREA)

Abstract

La presente descripción describe vectores de virus adeno-asociados (AAV) mejorados para aplicaciones de terapia génica en el tratamiento de la enfermedad de almacenamiento de glucógeno, en especial glucogenosis tipo Ia (GSD-Ia). Se describen moléculas recombinantes de ácido nucleico, vectores y AAV recombinante que incluyen un promotor/potenciador G6PC, un intrón sintético, una secuencia codificante G6PC (tal como una secuencia codificante codón-optimizada de G6PC o de tipo salvaje) y la secuencia stuffer o de relleno de ácido nucleico situada entre el promotor/potenciador G6PC y el intrón, así como entre el intrón y la secuencia codificante de G6PC. Los AAV recombinantes descritos en el presente documento muestran transducción altamente eficiente del hígado y son capaces de corregir anormalidades metabólicas en un modelo animal de GSD-Ia.
MX2016006774A 2013-11-26 2014-11-25 Vectores de virus adeno-asociado para el tratamiento de la enfermedad de almacenamiento de glucógeno. MX375360B (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361908861P 2013-11-26 2013-11-26
PCT/US2014/067415 WO2015081101A1 (en) 2013-11-26 2014-11-25 Adeno-associated virus vectors for treatment of glycogen storage disease

Publications (2)

Publication Number Publication Date
MX2016006774A MX2016006774A (es) 2016-10-13
MX375360B true MX375360B (es) 2025-03-06

Family

ID=52101617

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2016006774A MX375360B (es) 2013-11-26 2014-11-25 Vectores de virus adeno-asociado para el tratamiento de la enfermedad de almacenamiento de glucógeno.

Country Status (10)

Country Link
US (3) US9644216B2 (es)
EP (2) EP3074510B1 (es)
JP (1) JP6649265B2 (es)
CN (1) CN105934515B (es)
AU (1) AU2014354839B2 (es)
CA (1) CA2930872C (es)
ES (1) ES2690643T3 (es)
IL (1) IL245659B (es)
MX (1) MX375360B (es)
WO (1) WO2015081101A1 (es)

Families Citing this family (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
CN107106689A (zh) 2014-11-05 2017-08-29 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
CN112410338A (zh) 2014-11-14 2021-02-26 沃雅戈治疗公司 调节性多核苷酸
IL292999A (en) 2014-11-14 2022-07-01 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
ES2824829T3 (es) 2014-12-23 2021-05-13 Us Health Vectores de virus adenoasociado que codifican G6PC modificada y usos de estos
EP3371305A1 (en) * 2015-11-06 2018-09-12 Crispr Therapeutics AG Materials and methods for treatment of glycogen storage disease type 1a
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
SG11201809699XA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Modulatory polynucleotides
KR20220108216A (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
CN110650673B (zh) 2016-08-30 2024-04-09 加利福尼亚大学董事会 用于生物医学靶向和递送的方法以及用于实践该方法的装置和系统
JP2020506695A (ja) * 2017-01-30 2020-03-05 ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ 糖原病を処置するための組換えウイルスベクター
CN110536964A (zh) * 2017-03-10 2019-12-03 国立研究开发法人国立成育医疗研究中心 反义寡核苷酸和糖原贮积病Ia型预防或治疗用组合物
SG11201909870SA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ハンチントン病治療組成物および方法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
JP7229989B2 (ja) 2017-07-17 2023-02-28 ボイジャー セラピューティクス インコーポレイテッド 軌道アレイガイドシステム
MX2020003042A (es) 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.
US11434502B2 (en) 2017-10-16 2022-09-06 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
EP4454654A3 (en) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
EP3763815A4 (en) 2018-03-09 2021-12-15 Daiichi Sankyo Company, Limited THERAPEUTIC FOR GLYCOGEN STORAGE DISEASE OF TYPE IA
EP3794126A1 (en) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
CN112334489A (zh) * 2018-05-16 2021-02-05 星火治疗有限公司 密码子优化的酸性α-葡萄糖苷酶表达盒及其使用方法
SG11202012499RA (en) 2018-06-28 2021-01-28 Crispr Therapeutics Ag Compositions and methods for genomic editing by insertion of donor polynucleotides
EP3849595B1 (en) * 2018-09-13 2025-11-19 ModernaTX, Inc. Polynucleotides encoding glucose-6-phosphatase for the treatment of glycogen storage disease
CN113383010B (zh) 2018-09-28 2025-09-09 沃雅戈治疗公司 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法
US20220026414A1 (en) * 2018-12-12 2022-01-27 Merck Sharp & Dohme Corp. Cell-based bioidentity test for insulin
DK3898981T5 (da) 2018-12-18 2024-08-05 Ultragenyx Pharmaceutical Inc Fremgangsmåder og sammensætninger til behandling af glykogenlagringssygdomme
EP3913060A1 (en) * 2020-05-22 2021-11-24 Genethon Vectors encoding a glucose-6-phosphatase (g6pase-a) for gene therapy
CN111972355A (zh) * 2020-08-31 2020-11-24 中国医学科学院北京协和医院 GSDIa型糖原累积症小鼠模型及其构建方法
KR102398032B1 (ko) * 2020-09-22 2022-05-19 비피진 주식회사 암 치료를 위한 5''-뉴클레오티다아제 변이체
WO2023069891A1 (en) * 2021-10-19 2023-04-27 University Of Connecticut Compositions and methods for the treatment of glycogen storage disorders

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6759050B1 (en) 1998-12-03 2004-07-06 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
US7261544B2 (en) 2003-05-21 2007-08-28 Genzyme Corporation Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
CA2713338C (en) 2008-01-29 2021-10-26 Applied Genetic Technologies Corporation Recombinant virus production using mammalian cells in suspension
JP2012521750A (ja) * 2009-03-27 2012-09-20 プロイェクト、デ、ビオメディシナ、シーマ、ソシエダッド、リミターダ 肝硬変及び肝線維症の治療のための方法及び組成物
US8679837B2 (en) 2009-04-02 2014-03-25 University Of Florida Research Foundation, Inc. Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors
ES2680915T3 (es) 2010-01-28 2018-09-11 The Children's Hospital Of Philadelphia Plataforma de fabricación escalable para la purificación de vectores virales y vectores virales purificados de este modo para su utilización en terapia génica

Also Published As

Publication number Publication date
MX2016006774A (es) 2016-10-13
US20170233763A1 (en) 2017-08-17
EP3415620B1 (en) 2021-03-31
CN105934515A (zh) 2016-09-07
BR112016011997A2 (pt) 2017-09-26
WO2015081101A8 (en) 2016-06-16
CN105934515B (zh) 2020-08-04
WO2015081101A1 (en) 2015-06-04
JP2016538885A (ja) 2016-12-15
US9644216B2 (en) 2017-05-09
ES2690643T3 (es) 2018-11-21
US11060110B2 (en) 2021-07-13
JP6649265B2 (ja) 2020-02-19
IL245659A0 (en) 2016-06-30
EP3074510B1 (en) 2018-08-01
EP3415620A1 (en) 2018-12-19
EP3074510A1 (en) 2016-10-05
IL245659B (en) 2020-01-30
CA2930872A1 (en) 2015-06-04
AU2014354839A1 (en) 2016-06-09
US20190017069A1 (en) 2019-01-17
CA2930872C (en) 2022-05-31
US10113183B2 (en) 2018-10-30
US20160376608A1 (en) 2016-12-29
AU2014354839B2 (en) 2020-06-04

Similar Documents

Publication Publication Date Title
WO2015081101A8 (en) Adeno-associated virus vectors for treatment of glycogen storage disease
MX2020005282A (es) Vector para la produccion de particulas aav.
EP3597760A3 (en) Adeno-associated virus vector
PH12018501055A1 (en) Cpg reduced factor viii variants, compositions and methods and uses for treatment of hemostasis disorders
EA201591992A1 (ru) Эффективная доставка больших генов посредством двойных aav векторов
EP4223772A3 (en) Optimized factor viii gene
MX2023001394A (es) Genes del factor viii optimizados.
MX2022010959A (es) Terapia genica para trastornos oculares.
MY187559A (en) Modified hepatitis post-transcriptional regulatory elements
WO2016075473A3 (en) Factor ix gene therapy
EA201791971A1 (ru) Онколитический аденовирус, кодирующий белок b7
MX2016001204A (es) Vectores virales adeno-asociados (aav) utiles para transducir tejido adiposo.
MX2016012558A (es) Suministro sistemico de vectores de virus que codifican urocortina-2 y genes relacionados para tratar disfunciones cardiacas relacionadas con diabetes y falla de corazon congestivo.
JP2013509168A5 (es)
MX2018007234A (es) Vectores virales adenoasociados utiles en el tratamiento de atrofia muscular espinal.
NZ628385A (en) Batches of recombinant adenovirus with altered terminal ends
MX2018015629A (es) Sistema de vector viral adeno-asociado traslapado doble para la expresion de abc4a.
MX2021006253A (es) Terapias génicas para enfermedad neurodegenerativa.
EP3978614A3 (en) Single-vector gene construct comprising insulin and glucokinase genes
JOP20220043A1 (ar) بروتين كابسيد vp1 معدل معزول من aav5
WO2014143884A3 (en) Synthetic methylmalonyl-coa mutase transgene for the treatment of mut class methylmalonic acidemia (mma)
AU2015247777A8 (en) Codon optimized nucleic acid encoding a retinitis pigmentosa GTPase regulator (RPGR)
NZ739286A (en) Recombinant orf virus vector
JP2016538885A5 (es)
EP3950939A3 (en) Lipase variants and polynucleotides encoding same

Legal Events

Date Code Title Description
FG Grant or registration