CN107083400A - 神经退行性疾病的基因治疗 - Google Patents

神经退行性疾病的基因治疗 Download PDF

Info

Publication number
CN107083400A
CN107083400A CN201610906309.6A CN201610906309A CN107083400A CN 107083400 A CN107083400 A CN 107083400A CN 201610906309 A CN201610906309 A CN 201610906309A CN 107083400 A CN107083400 A CN 107083400A
Authority
CN
China
Prior art keywords
aav
cell
sma
mouse
disease
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CN201610906309.6A
Other languages
English (en)
Chinese (zh)
Inventor
M·A·帕西尼
L·谢哈布丁
S·H·程
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Genzyme Corp
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 CN107083400A publication Critical patent/CN107083400A/zh
Pending legal-status Critical Current

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
    • C12N15/63Introduction 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
    • C12N15/861Adenoviral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • 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
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61K48/0008Medicinal 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
    • 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
    • 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • 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
    • A61P21/02Muscle relaxants, e.g. for tetanus or cramps
    • 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
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702Regulators; Modulating activity
    • 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
    • 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
    • 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
    • C12N15/8645Adeno-associated virus
    • 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
    • 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
    • C12N2750/14121Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • C12N2750/14133Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
    • 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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Marine Sciences & Fisheries (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Toxicology (AREA)
  • Neurosurgery (AREA)
  • Pain & Pain Management (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN201610906309.6A 2009-05-02 2010-04-27 神经退行性疾病的基因治疗 Pending CN107083400A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US17498209P 2009-05-02 2009-05-02
US61/174,982 2009-05-02
US26805909P 2009-06-08 2009-06-08
US61/268,059 2009-06-08
CN201080027833.7A CN102459611B (zh) 2009-05-02 2010-04-27 神经退行性疾病的基因治疗

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
CN201080027833.7A Division CN102459611B (zh) 2009-05-02 2010-04-27 神经退行性疾病的基因治疗

Publications (1)

Publication Number Publication Date
CN107083400A true CN107083400A (zh) 2017-08-22

Family

ID=43050324

Family Applications (2)

Application Number Title Priority Date Filing Date
CN201080027833.7A Active CN102459611B (zh) 2009-05-02 2010-04-27 神经退行性疾病的基因治疗
CN201610906309.6A Pending CN107083400A (zh) 2009-05-02 2010-04-27 神经退行性疾病的基因治疗

Family Applications Before (1)

Application Number Title Priority Date Filing Date
CN201080027833.7A Active CN102459611B (zh) 2009-05-02 2010-04-27 神经退行性疾病的基因治疗

Country Status (23)

Country Link
US (9) US20130287736A1 (enExample)
EP (5) EP2424991B1 (enExample)
JP (3) JP5879256B2 (enExample)
KR (2) KR20180027619A (enExample)
CN (2) CN102459611B (enExample)
BR (1) BRPI1010868A2 (enExample)
CA (1) CA2759801C (enExample)
CY (1) CY1120982T1 (enExample)
DK (4) DK3421603T3 (enExample)
ES (4) ES3038109T3 (enExample)
FI (2) FI3988660T3 (enExample)
HR (4) HRP20212024T1 (enExample)
HU (3) HUE072183T2 (enExample)
IL (2) IL216094A0 (enExample)
LT (4) LT3988660T (enExample)
MX (4) MX356669B (enExample)
PL (4) PL3988660T3 (enExample)
PT (4) PT3988660T (enExample)
RS (3) RS67078B1 (enExample)
RU (2) RU2603740C2 (enExample)
SG (3) SG10202109219SA (enExample)
SI (4) SI3988660T1 (enExample)
WO (1) WO2010129021A1 (enExample)

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109266682A (zh) * 2018-09-29 2019-01-25 中国科学院武汉物理与数学研究所 一种神经细胞快速逆行跨突触标记的方法及应用
CN108096243B (zh) * 2017-11-24 2020-05-29 江苏康缘药业股份有限公司 银杏内酯组合物的医药用途
CN112567246A (zh) * 2018-01-25 2021-03-26 比奥根Ma公司 治疗脊髓性肌萎缩症的方法
CN114903010A (zh) * 2022-05-19 2022-08-16 暨南大学 神经退行性疾病模型的构建方法
CN118045206A (zh) * 2024-04-12 2024-05-17 四川至善唯新生物科技有限公司 一种治疗脊髓型肌肉萎缩的药物组合物及其用途

Families Citing this family (93)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US11219696B2 (en) 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
DK3421603T3 (da) 2009-05-02 2022-01-10 Genzyme Corp Genterapi for neurodegenerative forstyrrelser
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
US9102949B2 (en) 2010-04-23 2015-08-11 University Of Massachusetts CNS targeting AAV vectors and methods of use thereof
EP2561075B1 (en) 2010-04-23 2018-06-27 University of Massachusetts Aav-based treatment of cholesterol-related disorders
EP2640407A4 (en) * 2010-11-16 2014-07-09 Denis G Kay PROCESS FOR INCREASING THE EXPRESSION AND ACTIVITY OF NEPRILYSIN
GB201103062D0 (en) * 2011-02-22 2011-04-06 Isis Innovation Method
CN103476399A (zh) 2011-04-18 2013-12-25 独立行政法人国立精神·神经医疗研究中心 药剂递送粒子及其制造方法
EP3318634A1 (en) 2011-04-21 2018-05-09 University of Massachusetts Raav-based compositions and methods for treating diseases involving dominant-negative or gain of function mutations
US20130039888A1 (en) 2011-06-08 2013-02-14 Nationwide Children's Hospital Inc. Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
EP3536795A3 (en) * 2012-06-21 2019-12-11 Association Institut de Myologie Widespread gene expression
PT2879719T (pt) * 2012-08-01 2018-10-08 Ohio State Innovation Foundation Administração intratecal do vírus adeno-associado 9 recombinante
CN103638604B (zh) * 2012-12-31 2016-05-04 深圳先进技术研究院 一种助行系统
WO2014169087A2 (en) * 2013-04-10 2014-10-16 The Trustees Of Columbia University In The City Of New York Treatment of proximal spinal muscular atrophy
KR102116378B1 (ko) * 2013-05-01 2020-06-01 젠자임 코포레이션 척수성 근위축증을 치료하기 위한 조성물 및 방법
EP3019620A4 (en) 2013-07-12 2017-01-18 The Children's Hospital of Philadelphia Aav vector and assay for anti-aav (adeno-associated virus) neutralizing antibodies
WO2015127128A2 (en) 2014-02-19 2015-08-27 University Of Massachusetts Recombinant aavs having useful transcytosis properties
WO2015140799A1 (en) 2014-03-18 2015-09-24 Carmel-Haifa University Economic Corp. Ltd Methods for improving cognitive function via modulation of quinone reductase 2
AU2015231294B2 (en) 2014-03-18 2020-10-29 University Of Massachusetts rAAV-based compositions and methods for treating amyotrophic lateral sclerosis
EP2933335A1 (en) * 2014-04-18 2015-10-21 Genethon A method of treating peripheral neuropathies and motor neuron diseases
JP6983511B2 (ja) 2014-04-25 2021-12-17 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 脳中の転移乳癌および他の癌を処置するための方法および組成物
US10975391B2 (en) 2014-04-25 2021-04-13 University Of Massachusetts Recombinant AAV vectors useful for reducing immunity against transgene products
US10689653B2 (en) 2014-06-03 2020-06-23 University Of Massachusetts Compositions and methods for modulating dysferlin expression
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
WO2016054557A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Novel high efficiency library-identified aav vectors
CA2964272A1 (en) 2014-10-21 2016-04-28 Guangping Gao Recombinant aav variants and uses thereof
RU2716991C2 (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
CA2967468A1 (en) 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
SMT201900347T1 (it) * 2014-12-24 2019-07-11 Uniqure Ip Bv Soppressione del gene huntingtina indotta da rnai
US10584321B2 (en) 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases
CA3021949C (en) 2015-04-24 2023-10-17 University Of Massachusetts Modified aav constructs and uses thereof
HK1257024A1 (zh) * 2015-08-31 2019-10-11 宾夕法尼亚大学 用於治疗伴侣动物的aav-epo
CN108137655B (zh) 2015-09-28 2022-04-22 北卡罗来纳-查佩尔山大学 逃避抗体的病毒载体的方法和组合物
CA3002980A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
DK3364997T5 (da) 2015-10-22 2024-09-30 Univ Massachusetts Aspartoacylase genterapi til behandling af canavans sygdom
CA3011939A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
WO2017139643A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
WO2017151884A1 (en) * 2016-03-02 2017-09-08 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia
US11207426B2 (en) 2016-04-05 2021-12-28 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
EP3467497B1 (en) * 2016-05-24 2021-09-29 Tokyo Women's Medical University Method for analyzing expression of smn protein nuclear body
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
WO2018071831A1 (en) 2016-10-13 2018-04-19 University Of Massachusetts Aav capsid designs
JP7309196B2 (ja) * 2016-11-07 2023-07-18 マックォーリー・ユニバーシティ タンパク質蓄積の調節及びその使用
JOP20190200A1 (ar) * 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
CA3059213A1 (en) 2017-05-09 2018-11-15 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (als)
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
ES2970198T3 (es) * 2017-08-16 2024-05-27 Lgv1 S R L Isoforma VTFT de una proteína BPIFB4 para uso en enfermedades y lesiones neuronales
WO2019060686A1 (en) 2017-09-22 2019-03-28 University Of Massachusetts NEW DUAL EXPRESSION VECTORS OF SOD1 AND USES THEREOF
CN111465691A (zh) 2017-10-03 2020-07-28 普利维尔治疗公司 用于溶酶体障碍的基因疗法
KR102709597B1 (ko) 2017-10-03 2024-09-26 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
EP3707264A1 (en) 2017-11-08 2020-09-16 Avexis Inc. Means and method for preparing viral vectors and uses of same
KR102112476B1 (ko) * 2017-12-29 2020-05-19 주식회사 헬릭스미스 하이브리드 HGF 유전자가 도입된 AAV(Adeno-Associated Virus) 벡터
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
PE20212076A1 (es) 2018-02-01 2021-10-26 Homology Medicines Inc Composiciones de virus adeno-asociado para restaurar la funcion del gen pah y metodos de uso de las mismas
EP3749766A1 (en) 2018-02-09 2020-12-16 F.Hoffmann-La Roche Ag Oligonucleotides for modulating tmem106b expression
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
SG11202009451VA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス インコーポレイテッド 抗体を回避するウイルスベクター
JP2021521889A (ja) * 2018-04-17 2021-08-30 アプライド ステムセル,インコーポレイテッド 脊髄性筋萎縮症を処置するための組成物および方法
BR112020023082A2 (pt) 2018-05-15 2021-02-09 Voyager Therapeutics, Inc. composições e métodos para o tratamento de doença de parkinson
US12163129B2 (en) 2018-06-08 2024-12-10 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
US12228571B2 (en) 2018-06-08 2025-02-18 Novartis Ag Cell-based assay for measuring drug product potency
CN108795946B (zh) * 2018-06-28 2022-03-04 北京锦篮基因科技有限公司 携带设计smn1基因表达框的重组腺相关病毒及应用
CA3114621A1 (en) 2018-09-28 2020-04-02 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
CN113365640A (zh) 2018-12-06 2021-09-07 比奥根Ma公司 引导肌萎缩侧索硬化症的治疗性干预的神经丝蛋白
RU2731514C2 (ru) * 2018-12-21 2020-09-03 Селл энд Джин Терапи Лтд Генотерапевтический ДНК-вектор на основе генотерапевтического ДНК-вектора VTvaf17, несущий целевой ген, выбранный из группы генов SHH, CTNNB1, NOG, WNT7A для повышения уровня экспрессии этих целевых генов, способ его получения и применения, штамм Escherichia coli SCS110-AF/VTvaf17-SHH, или Escherichia coli SCS110-AF/VTvaf17-CTNNB1, или Escherichia coli SCS110-AF/VTvaf17-NOG, или Escherichia coli SCS110-AF/VTvaf17-WNT7A, несущий генотерапевтический ДНК-вектор, способ его получения, способ производства в промышленных масштабах генотерапевтического ДНК-вектора
CA3128003A1 (en) * 2019-02-01 2020-08-06 Avrobio, Inc. Compositions and methods for treating neurocognitive disorders
KR20210131370A (ko) 2019-02-22 2021-11-02 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Grn-연관 성인-발병 신경퇴화의 치료를 위한 재조합 아데노-연관 바이러스
CA3133453A1 (en) 2019-03-21 2020-09-24 Daniel Mccoy Recombinant adeno-associated virus vectors
US20220154217A1 (en) 2019-04-01 2022-05-19 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
AU2020273182B2 (en) 2019-04-10 2025-11-20 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
JP2022526425A (ja) * 2019-04-12 2022-05-24 エンコーデッド セラピューティクス, インコーポレイテッド 治療剤の投与のための組成物および方法
TW202128736A (zh) 2019-10-17 2021-08-01 美商史崔德生物公司 用於治療c型尼曼—匹克病之腺相關病毒載體
KR20230019402A (ko) * 2019-10-22 2023-02-08 어플라이드 제네틱스 테크놀로지스 코퍼레이션 프로그래뉼린 연관 신경변성 질환 또는 장애의 치료를 위한 아데노-연관 바이러스 (aav) 시스템
WO2021119622A1 (en) * 2019-12-12 2021-06-17 Northwestern University Modulation of ubiquitin carboxy-terminal hydrolase ligase 1 expression
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
WO2021216503A1 (en) 2020-04-20 2021-10-28 Christiana Care Health Services, Inc. Aav delivery system for lung cancer treatment
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復pah基因功能的腺相關病毒組成物及其使用方法
CN116745409A (zh) 2020-08-19 2023-09-12 萨雷普塔治疗公司 用于治疗雷特综合征的腺相关病毒载体
CN112121179A (zh) * 2020-09-15 2020-12-25 山东兴瑞生物科技有限公司 一种组合物及其在治疗脊髓性肌萎缩症中的应用
TW202246501A (zh) * 2021-01-29 2022-12-01 俄羅斯聯邦商亞那拜恩有限公司 SMN1和miR-23a在治療脊髓性肌萎縮中的協同作用
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
EP4472701A1 (en) * 2022-02-01 2024-12-11 Seattle Children's Hospital d/b/a Seattle Children's Research Institute Simplified method of preparing cells for patient administration
EP4504951A2 (en) * 2022-04-05 2025-02-12 The Johns Hopkins University Methods and materials for treating syngap1-associated neurodevelopmental disorders
JP2025512333A (ja) 2022-04-11 2025-04-17 テナヤ セラピューティクス, インコーポレイテッド 操作されたカプシドを伴うアデノ随伴ウイルス
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
CN116042719B (zh) * 2022-12-28 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
WO2024215653A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1575340A (zh) * 2001-08-29 2005-02-02 田边制药株式会社 用于治疗神经退行性疾病的组合物和方法
US20080187512A1 (en) * 2007-02-07 2008-08-07 Academia Sinica Treatment for spinal muscular atrophy
US20090069261A1 (en) * 2005-05-02 2009-03-12 Genzyme Corporation Gene therapy for spinal cord disorders

Family Cites Families (88)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ210501A (en) 1983-12-13 1991-08-27 Kirin Amgen Inc Erythropoietin produced by procaryotic or eucaryotic expression of an exogenous dna sequence
US4703008A (en) 1983-12-13 1987-10-27 Kiren-Amgen, Inc. DNA sequences encoding erythropoietin
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
JPS62171696A (ja) 1986-01-23 1987-07-28 Sumitomo Chem Co Ltd ヒトエリスロポエチンの製造方法
US4954437A (en) 1986-09-15 1990-09-04 Integrated Genetics, Inc. Cell encoding recombinant human erythropoietin
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
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
US5605690A (en) 1989-09-05 1997-02-25 Immunex Corporation Methods of lowering active TNF-α levels in mammals using tumor necrosis factor receptor
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
WO1992001070A1 (en) 1990-07-09 1992-01-23 The United States Of America, As Represented By The Secretary, U.S. Department Of Commerce High efficiency packaging of mutant adeno-associated virus using amber suppressions
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5747469A (en) 1991-03-06 1998-05-05 Board Of Regents, The University Of Texas System Methods and compositions comprising DNA damaging agents and p53
CA2115742A1 (en) 1991-08-20 1993-03-04 Ronald G. Crystal Adenovirus mediated transfer of genes to the gastrointestinal tract
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US6686200B1 (en) 1993-08-31 2004-02-03 Uab Research Foundation Methods and compositions for the large scale production of recombinant adeno-associated virus
ES2216005T3 (es) 1993-11-09 2004-10-16 Targeted Genetics Corporation Produccion de titulos elevados de vectores de aav recombinantes.
EP0728214B1 (en) 1993-11-09 2004-07-28 Medical College Of Ohio Stable cell lines capable of expressing the adeno-associated virus replication gene
EP0755454B1 (en) 1994-04-13 2008-02-13 The Rockefeller University Aav-mediated delivery of dna to cells of the nervous system
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
EP0708178A1 (en) * 1994-10-19 1996-04-24 Institut National De La Sante Et De La Recherche Medicale (Inserm) Survival motor neuron (SMN) gene: a gene for spinal muscular atrophy
AU707866B2 (en) 1994-12-06 1999-07-22 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
US5756283A (en) 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US5882914A (en) 1995-06-06 1999-03-16 The Johns Hopkins University School Of Medicine Nucleic acids encoding the hypoxia inducible factor-1
US5677158A (en) 1995-06-07 1997-10-14 Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US5622856A (en) 1995-08-03 1997-04-22 Avigen High efficiency helper system for AAV vector production
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US6004797A (en) 1995-11-09 1999-12-21 Avigen, Inc. Adenovirus helper-free recombinant AAV Virion production
JP2000506010A (ja) 1996-02-29 2000-05-23 イミューソル インコーポレイテッド C型肝炎ウイルスリボザイム
CA2247099A1 (en) 1996-03-04 1997-09-12 Targeted Genetics Corporation Methods for transducing cells in blood vessels using recombinant aav vectors
US20040076613A1 (en) * 2000-11-03 2004-04-22 Nicholas Mazarakis Vector system
US6261823B1 (en) 1996-12-13 2001-07-17 Schering Corporation Methods for purifying viruses
US6541258B2 (en) 1996-12-18 2003-04-01 Targeted Genetics Corporation AAV split-packaging genes and cell lines comprising such genes for use in the production of recombinant AAV vectors
CA2269661A1 (en) 1996-12-18 1998-06-25 Targeted Genetics Corporation Recombinase-activatable aav packaging cassettes for use in the production of aav vectors
EP0996741A4 (en) 1997-01-23 2004-06-09 Immusol Inc FUNCTIONAL ANALYSIS AND GENE DISCOVERY USING TARGET SPECIFIC RIBOZY VECTOR BANKS OR WELL MADE RANDOM
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
WO2000014205A2 (en) 1998-09-04 2000-03-16 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant aav vectors
ES2399232T3 (es) 1997-09-05 2013-03-26 Genzyme Corporation Procedimientos para la generación de preparaciones de vectores de AAV recombinantes de títulos altos exentas de auxiliares
JP2001520051A (ja) 1997-10-21 2001-10-30 ターゲテッド ジェネティックス コーポレイション 組換えaavベクターの産生のための増幅可能アデノ随伴ウイルス(aav)パッケージングカセット
DE69840948D1 (de) 1997-10-21 2009-08-13 Targeted Genetics Corp Invertierte terminale repeats von aav, die für die transkription aktiviert sind, zur verwendung in rekombinanten aav-vektoren
IL136454A0 (en) 1997-12-04 2001-06-14 Genzyme Corp Compositions and methods for inducing gene expression
BR9908015A (pt) 1998-02-17 2001-04-24 Schering Corp Composições que compreendem vìrus e métodos para concentração de preparações de vìrus
AUPP249298A0 (en) 1998-03-20 1998-04-23 Ag-Gene Australia Limited Synthetic genes and genetic constructs comprising same I
WO1999047691A1 (en) 1998-03-20 1999-09-23 Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
JP2002516295A (ja) 1998-05-27 2002-06-04 アビジェン, インコーポレイテッド Aavベクターの対流増加送達
EP1080218A1 (en) 1998-05-27 2001-03-07 University of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient
US6646113B1 (en) * 1998-09-17 2003-11-11 The Trustees Of The University Of Pennsylvania Nucleic acid molecule encoding human survival of motor neuron-interacting protein 1 (SIP1) deletion mutants
US6759050B1 (en) 1998-12-03 2004-07-06 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
US6893865B1 (en) 1999-04-28 2005-05-17 Targeted Genetics Corporation Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
PT1180159E (pt) 1999-05-28 2008-12-05 Targeted Genetics Corp Métodos e composições para abaixamento do nível de factor de necrose tumoral (tnf) nos distúrbios associados a tnf
ES2308989T3 (es) 1999-08-09 2008-12-16 Targeted Genetics Corporation Aumento de la expresion de una secuencia nucleotidica heterologa a partir de vectores viricos recombinantes que contienen una secuencia que forman pares de bases intracatenarios.
US20020099025A1 (en) * 1999-12-30 2002-07-25 Heywood James A. Treatment of neurological disorders
CA2399321C (en) 2000-03-07 2013-04-30 Robert K. Evans Adenovirus formulations
AU2001257611A1 (en) 2000-04-28 2001-11-12 Avigen, Inc. Polynucleotides for use in recombinant adeno-associated virus virion production
AU6972301A (en) 2000-06-01 2001-12-11 Univ North Carolina Duplexed parvovirus vectors
US6593123B1 (en) 2000-08-07 2003-07-15 Avigen, Inc. Large-scale recombinant adeno-associated virus (rAAV) production and purification
AU2001290984A1 (en) 2000-09-18 2002-04-02 Genzyme Corporation Expression vectors containing hybrid ubiquitin promoters
US20050032219A1 (en) 2001-07-03 2005-02-10 Patrick Aubourg Methods of administering vectors to synaptically connected neurons
WO2003039459A2 (en) 2001-11-05 2003-05-15 Genvec, Inc. Viral vector production methods and compositions
AU2002360429A1 (en) 2001-11-26 2003-06-10 Avigen, Inc. Methods for producing stocks of recombinant aav virions
DK2573170T3 (en) 2001-12-17 2018-04-09 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 9, vectors containing them, and their use
US20060292117A1 (en) * 2002-04-17 2006-12-28 Loiler Scott A Improved rAAv vectors
PL1620133T3 (pl) 2003-05-01 2016-05-31 Genzyme Corp Terapia genowa dla zaburzeń neurometabolicznych
US7765583B2 (en) 2005-02-28 2010-07-27 France Telecom System and method for managing virtual user domains
CN101212988A (zh) 2005-05-02 2008-07-02 建新公司 神经代谢疾病的基因治疗
AR059089A1 (es) 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
EP1979485A2 (en) 2006-01-31 2008-10-15 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
US8129510B2 (en) 2006-03-30 2012-03-06 The Board Of Trustees Of The Leland Stanford Junior University Minigene expression cassette
PL2489733T3 (pl) 2006-06-07 2019-08-30 Genzyme Corporation Terapia genowa w stwardnieniu zanikowym bocznym i innych zaburzeniach rdzenia kręgowego
ES2391789T3 (es) 2006-10-03 2012-11-29 Genzyme Corporation Terapia génica para la esclerosis lateral amiotrófica y otros trastornos de la médula espinal
ITMI20070127A1 (it) 2007-01-29 2008-07-30 Fond I R C C S Proteine e-o peptidi per la prevenzione e-o cura di malattie neurodegenerative
CA3002933A1 (en) 2007-07-14 2009-01-22 Beverly L. Davidson Methods and compositions for treating brain diseases
EP2019143A1 (en) * 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
WO2009151546A2 (en) 2008-05-27 2009-12-17 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
WO2010071832A1 (en) 2008-12-19 2010-06-24 Nationwide Children's Hospital Delivery of polynucleotides across the blood brain barrier using recombinant aav9
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
US11219696B2 (en) 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
DK3421603T3 (da) 2009-05-02 2022-01-10 Genzyme Corp Genterapi for neurodegenerative forstyrrelser
PT2879719T (pt) 2012-08-01 2018-10-08 Ohio State Innovation Foundation Administração intratecal do vírus adeno-associado 9 recombinante
FR3002237B1 (fr) 2013-02-15 2017-12-15 Genethon Methodes pour la production de particules virales aav double brin
KR102116378B1 (ko) 2013-05-01 2020-06-01 젠자임 코포레이션 척수성 근위축증을 치료하기 위한 조성물 및 방법
PE20170260A1 (es) 2014-05-02 2017-04-12 Genzyme Corp Vectores de aav para la terapia genica de la retina y el snc
FI3411484T3 (fi) 2016-02-05 2023-11-15 Univ Emory Yksisäikeisen tai itsekomplementaarisen adenoassosioidun viruksen 9 injektio serebrospinaaliseen fluidiin

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1575340A (zh) * 2001-08-29 2005-02-02 田边制药株式会社 用于治疗神经退行性疾病的组合物和方法
US20090069261A1 (en) * 2005-05-02 2009-03-12 Genzyme Corporation Gene therapy for spinal cord disorders
US20080187512A1 (en) * 2007-02-07 2008-08-07 Academia Sinica Treatment for spinal muscular atrophy

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
EDMUND R HOLLIS II ET AL.: "Efficient retrograde neuronal transduction utilizing self complementary AAV1", 《MOLECULAR THERAPY》 *

Cited By (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108096243B (zh) * 2017-11-24 2020-05-29 江苏康缘药业股份有限公司 银杏内酯组合物的医药用途
CN112567246A (zh) * 2018-01-25 2021-03-26 比奥根Ma公司 治疗脊髓性肌萎缩症的方法
CN112567246B (zh) * 2018-01-25 2024-12-03 比奥根Ma公司 治疗脊髓性肌萎缩症的方法
CN109266682A (zh) * 2018-09-29 2019-01-25 中国科学院武汉物理与数学研究所 一种神经细胞快速逆行跨突触标记的方法及应用
CN109266682B (zh) * 2018-09-29 2022-04-01 中国科学院武汉物理与数学研究所 一种神经细胞快速逆行跨突触标记的方法及应用
CN114903010A (zh) * 2022-05-19 2022-08-16 暨南大学 神经退行性疾病模型的构建方法
CN114903010B (zh) * 2022-05-19 2024-06-07 暨南大学 神经退行性疾病模型的构建方法
CN118045206A (zh) * 2024-04-12 2024-05-17 四川至善唯新生物科技有限公司 一种治疗脊髓型肌肉萎缩的药物组合物及其用途
CN118045206B (zh) * 2024-04-12 2024-07-05 四川至善唯新生物科技有限公司 一种治疗脊髓型肌肉萎缩的药物组合物及其用途

Also Published As

Publication number Publication date
CN102459611B (zh) 2016-11-09
SG175409A1 (en) 2011-12-29
MX356669B (es) 2018-06-08
CA2759801C (en) 2019-04-02
RS67078B1 (sr) 2025-08-29
MX2011011594A (es) 2011-11-18
PT2424991T (pt) 2018-10-19
SG10201404528SA (en) 2014-10-30
PT3421603T (pt) 2022-01-10
EP3988660B1 (en) 2025-05-14
LT2424991T (lt) 2018-09-25
SG10202109219SA (en) 2021-10-28
HUE039345T2 (hu) 2018-12-28
KR101835490B1 (ko) 2018-03-08
US20130287736A1 (en) 2013-10-31
FI3988660T3 (fi) 2025-08-14
US20230135379A1 (en) 2023-05-04
EP3988660A1 (en) 2022-04-27
HRP20250897T1 (hr) 2025-09-26
IL259898A (en) 2018-07-31
HRP20212024T1 (hr) 2022-04-01
US9415119B2 (en) 2016-08-16
LT3421603T (lt) 2022-01-10
DK3988660T3 (da) 2025-08-18
EP2424991A1 (en) 2012-03-07
ES3041882T3 (en) 2025-11-17
DK3421603T3 (da) 2022-01-10
EP4342992B1 (en) 2025-05-14
CY1120982T1 (el) 2019-12-11
US20200384076A1 (en) 2020-12-10
ES2686504T3 (es) 2018-10-18
HUE072183T2 (hu) 2025-10-28
RS67077B1 (sr) 2025-08-29
PL3988660T3 (pl) 2025-09-22
HRP20181423T1 (hr) 2018-11-30
SI4342992T1 (sl) 2025-09-30
MX341073B (es) 2016-08-05
US20250288644A1 (en) 2025-09-18
MX2018006955A (es) 2020-11-12
US20170087212A1 (en) 2017-03-30
EP4342992A2 (en) 2024-03-27
EP4342992A3 (en) 2024-04-24
ES3038109T3 (en) 2025-10-09
EP3421603A1 (en) 2019-01-02
JP2018148927A (ja) 2018-09-27
FI4342992T3 (fi) 2025-08-12
KR20180027619A (ko) 2018-03-14
PT4342992T (pt) 2025-07-31
MX2023006169A (es) 2023-06-09
PL3421603T3 (pl) 2022-02-14
EP4585263A2 (en) 2025-07-16
US11975043B2 (en) 2024-05-07
WO2010129021A1 (en) 2010-11-11
RU2743398C2 (ru) 2021-02-18
BRPI1010868A2 (pt) 2018-06-12
HUE057606T2 (hu) 2022-05-28
PT3988660T (pt) 2025-07-31
HRP20250892T1 (hr) 2025-09-26
US12350311B2 (en) 2025-07-08
SI3421603T1 (sl) 2022-02-28
US11911440B2 (en) 2024-02-27
US20240033323A1 (en) 2024-02-01
JP2012526046A (ja) 2012-10-25
PL2424991T3 (pl) 2018-11-30
DK2424991T3 (en) 2018-09-17
EP3421603B1 (en) 2021-10-06
RU2011149094A (ru) 2013-06-10
RU2603740C2 (ru) 2016-11-27
PL4342992T3 (pl) 2025-09-22
KR20120006073A (ko) 2012-01-17
LT3988660T (lt) 2025-08-11
US20240350584A1 (en) 2024-10-24
EP2424991B1 (en) 2018-06-06
RU2016140850A (ru) 2018-12-14
US10369193B2 (en) 2019-08-06
IL216094A0 (en) 2012-01-31
EP4585263A3 (en) 2025-10-22
LT4342992T (lt) 2025-08-25
CA2759801A1 (en) 2010-11-11
DK4342992T3 (da) 2025-08-18
RS62779B1 (sr) 2022-01-31
JP2016052309A (ja) 2016-04-14
JP5879256B2 (ja) 2016-03-08
RU2016140850A3 (enExample) 2019-11-25
US20160051699A1 (en) 2016-02-25
CN102459611A (zh) 2012-05-16
US20230084580A1 (en) 2023-03-16
SI2424991T1 (sl) 2018-11-30
SI3988660T1 (sl) 2025-09-30
JP6397391B2 (ja) 2018-09-26
ES2903127T3 (es) 2022-03-31
EP2424991A4 (en) 2012-11-14

Similar Documents

Publication Publication Date Title
US12350311B2 (en) Gene therapy for neurodegenerative disorders
US20200297872A1 (en) Delivery of polynucleotides using recombinant aav9
RU2836835C2 (ru) Генная терапия нейродегенеративных нарушений
HK40106863B (en) Gene therapy for neurodegenerative disorders
HK40106863A (en) Gene therapy for neurodegenerative disorders
HK40073109B (en) Gene therapy for neurodegenerative disorders
HK40073109A (en) Gene therapy for neurodegenerative disorders

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination