DK1478236T3 - Målrettet retrograd gentilförsel til motorneuroner - Google Patents

Målrettet retrograd gentilförsel til motorneuroner

Info

Publication number
DK1478236T3
DK1478236T3 DK02799296T DK02799296T DK1478236T3 DK 1478236 T3 DK1478236 T3 DK 1478236T3 DK 02799296 T DK02799296 T DK 02799296T DK 02799296 T DK02799296 T DK 02799296T DK 1478236 T3 DK1478236 T3 DK 1478236T3
Authority
DK
Denmark
Prior art keywords
neuron
gene delivery
methods
motor neurons
cell body
Prior art date
Application number
DK02799296T
Other languages
English (en)
Inventor
Brian K Kaspar
Fred H Gage
Daniel Peterson
Original Assignee
Salk Inst For Biological Studi
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 Salk Inst For Biological Studi filed Critical Salk Inst For Biological Studi
Priority claimed from PCT/US2002/041194 external-priority patent/WO2003055983A2/en
Application granted granted Critical
Publication of DK1478236T3 publication Critical patent/DK1478236T3/da

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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4747—Apoptosis related proteins
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575—Hormones
    • C07K14/65—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0618—Cells of the nervous system
    • C12N5/0619—Neurons
    • 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
    • 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
    • C12N2510/00—Genetically modified cells
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Toxicology (AREA)
  • Neurology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicinal Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Diabetes (AREA)
  • Plant Pathology (AREA)
  • Endocrinology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Neurosurgery (AREA)
  • Cell Biology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Jellies, Jams, And Syrups (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
DK02799296T 2001-12-21 2002-12-20 Målrettet retrograd gentilförsel til motorneuroner DK1478236T3 (da)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US10/032,047 US6998118B2 (en) 2001-12-21 2001-12-21 Targeted retrograde gene delivery for neuronal protection
US10/237,567 US7101540B2 (en) 2001-12-21 2002-09-05 Targeted retrograde gene delivery to motor neurons
PCT/US2002/041194 WO2003055983A2 (en) 2001-12-21 2002-12-20 Targeted retrograde gene delivery to motor neurons

Publications (1)

Publication Number Publication Date
DK1478236T3 true DK1478236T3 (da) 2009-06-02

Family

ID=21862809

Family Applications (1)

Application Number Title Priority Date Filing Date
DK02799296T DK1478236T3 (da) 2001-12-21 2002-12-20 Målrettet retrograd gentilförsel til motorneuroner

Country Status (6)

Country Link
US (2) US6998118B2 (da)
AT (1) ATE422549T1 (da)
DE (1) DE60231153D1 (da)
DK (1) DK1478236T3 (da)
IL (1) IL162630A (da)
NZ (1) NZ547243A (da)

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US20060280724A1 (en) * 2000-11-04 2006-12-14 Ferguson Ian A Identification of ligands that enable endocytosis, using in vivo manipulation of neuronal fibers
IL162630A0 (en) * 2001-12-21 2005-11-20 Salk Inst For Biological Studi A composition containing a viral vector containinga heterologous gene to be transduced into a neuron
US6998118B2 (en) * 2001-12-21 2006-02-14 The Salk Institute For Biological Studies Targeted retrograde gene delivery for neuronal protection
US20040258666A1 (en) * 2003-05-01 2004-12-23 Passini Marco A. Gene therapy for neurometabolic disorders
CA2539947A1 (en) * 2003-09-24 2005-04-07 The Board Of Trustees Of The Leland Stanford Junior University Igf-1 instructs multipotent adult cns neural stem cells to an oligodendroglial lineage
EP1680145A4 (en) * 2003-10-14 2007-05-23 Neurologix Res Inc METHODS AND COMPOSITIONS FOR THE TREATMENT OF NEUROLOGICAL DISEASES
US8524499B2 (en) * 2003-11-10 2013-09-03 Cornell Research Foundation, Inc. Regulatory sequences that direct gene expression to spinal motor neurons and uses thereof
US20060171926A1 (en) * 2004-04-30 2006-08-03 Passini Marco A Gene therapy for neurometabolic disorders
JP2008507288A (ja) 2004-07-23 2008-03-13 アクセルロン ファーマ インコーポレーテッド ＡｃｔＲＩＩ受容体ポリペプチド、方法、および組成物
US20060122116A1 (en) * 2004-11-03 2006-06-08 The Johns Hopkins University Treatment for disorders of the peripheral nervous system
US7595295B2 (en) * 2005-02-25 2009-09-29 Rush University Medical Center Use of pleiotrophin to promote neurogeneration
JP5829372B2 (ja) 2005-05-02 2015-12-09 ジェンザイム・コーポレーション 神経代謝性疾患のための遺伝子治療
CA2606490C (en) * 2005-05-02 2018-02-06 Genzyme Corporation Gene therapy for spinal cord disorders
US8128933B2 (en) 2005-11-23 2012-03-06 Acceleron Pharma, Inc. Method of promoting bone growth by an anti-activin B antibody
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WO2008013918A2 (en) * 2006-07-26 2008-01-31 Myelin Repair Foundation, Inc. Cell cycle regulation and differentiation
US8895016B2 (en) 2006-12-18 2014-11-25 Acceleron Pharma, Inc. Antagonists of activin-actriia and uses for increasing red blood cell levels
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TW201803890A (zh) 2007-02-02 2018-02-01 艾瑟勒朗法瑪公司 衍生自ＡｃｔＲＩＩＢ的變體與其用途
EA018221B1 (ru) 2007-02-09 2013-06-28 Акселерон Фарма Инк. АНТАГОНИСТЫ АКТИВИНА-ActRIIa И ПРИМЕНЕНИЕ ДЛЯ СТИМУЛЯЦИИ РОСТА КОСТИ У БОЛЬНЫХ РАКОМ
EP2019143A1 (en) 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
CN101861161B (zh) 2007-09-18 2017-04-19 阿塞勒隆制药公司 活化素‑actriia拮抗剂和减少或抑制fsh分泌的用途
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
BRPI0800957A2 (pt) * 2008-04-04 2009-11-17 Univ Rio De Janeiro vetores biológicos compreendendo o gene max, método de produção dos mesmos, método de expressão do gene max nas células e método de terapia gênica citoprotetora
CN102131822A (zh) * 2008-06-26 2011-07-20 阿塞勒隆制药公司 进行激活素-ActRIIa拮抗剂给药和监测受治疗患者的方法
US8216997B2 (en) 2008-08-14 2012-07-10 Acceleron Pharma, Inc. Methods for increasing red blood cell levels and treating anemia using a combination of GDF traps and erythropoietin receptor activators
TW201803586A (zh) 2008-08-14 2018-02-01 艾瑟勒朗法瑪公司 使用ｇｄｆ阱以增加紅血球水平
AU2010204985A1 (en) 2009-01-13 2011-08-04 Acceleron Pharma Inc. Methods for increasing adiponectin
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KR20180026795A (ko) 2009-06-12 2018-03-13 악셀레론 파마 인코포레이티드 절두된 ＡｃｔＲＩＩＢ-ＦＣ 융합 단백질
EP3202459B1 (en) * 2009-09-09 2021-04-14 Acceleron Pharma Inc. Actriib antagonists and dosing and uses thereof for treating obesity or type 2 diabetes by regulating body fat content
AU2010315245B2 (en) * 2009-11-03 2016-11-03 Acceleron Pharma Inc. Methods for treating fatty liver disease
EP3332796A1 (en) 2009-11-17 2018-06-13 Acceleron Pharma Inc. Actriib proteins and variants and uses therefore relating to utrophin induction for muscular dystrophy therapy
AU2011326586A1 (en) 2010-11-08 2013-05-30 Acceleron Pharma, Inc. ActRIIA binding agents and uses thereof
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
US10195249B2 (en) 2012-11-02 2019-02-05 Celgene Corporation Activin-ActRII antagonists and uses for treating bone and other disorders
WO2014145776A2 (en) * 2013-03-15 2014-09-18 Baylor College Of Medicine Msp and its receptors in the therapy of amyotrophic lateral sclerosis
DK3492593T3 (da) 2013-11-13 2021-11-08 Childrens Medical Center Nukleasemedieret regulering af genekspression
CN114699529A (zh) 2014-06-13 2022-07-05 阿塞勒隆制药公司 用于治疗溃疡的方法和组合物
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US6998118B2 (en) * 2001-12-21 2006-02-14 The Salk Institute For Biological Studies Targeted retrograde gene delivery for neuronal protection

Also Published As

Publication number Publication date
US6998118B2 (en) 2006-02-14
US7101540B2 (en) 2006-09-05
IL162630A (en) 2012-08-30
NZ547243A (en) 2008-09-26
US20030118552A1 (en) 2003-06-26
ATE422549T1 (de) 2009-02-15
US20030118556A1 (en) 2003-06-26
DE60231153D1 (de) 2009-03-26

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