CN108884022B - 用于治疗神经元的过度兴奋的方法和组合物 - Google Patents

用于治疗神经元的过度兴奋的方法和组合物 Download PDF

Info

Publication number
CN108884022B
CN108884022B CN201780020853.3A CN201780020853A CN108884022B CN 108884022 B CN108884022 B CN 108884022B CN 201780020853 A CN201780020853 A CN 201780020853A CN 108884022 B CN108884022 B CN 108884022B
Authority
CN
China
Prior art keywords
gad65
vgat
subject
vector
gene
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.)
Active
Application number
CN201780020853.3A
Other languages
English (en)
Chinese (zh)
Other versions
CN108884022A (zh
Inventor
马丁·马尔萨拉
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.)
University of California San Diego UCSD
Original Assignee
University of California San Diego UCSD
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 University of California San Diego UCSD filed Critical University of California San Diego UCSD
Publication of CN108884022A publication Critical patent/CN108884022A/zh
Application granted granted Critical
Publication of CN108884022B publication Critical patent/CN108884022B/zh
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70571—Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
    • 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
    • A61K48/0075—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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C233/00—Carboxylic acid amides
    • C07C233/64—Carboxylic acid amides having carbon atoms of carboxamide groups bound to carbon atoms of six-membered aromatic rings
    • C07C233/67—Carboxylic acid amides having carbon atoms of carboxamide groups bound to carbon atoms of six-membered aromatic rings having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms
    • C07C233/68—Carboxylic acid amides having carbon atoms of carboxamide groups bound to carbon atoms of six-membered aromatic rings having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom
    • C07C233/69—Carboxylic acid amides having carbon atoms of carboxamide groups bound to carbon atoms of six-membered aromatic rings having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom of an acyclic saturated carbon skeleton
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C235/00—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms
    • C07C235/02—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to acyclic carbon atoms and singly-bound oxygen atoms bound to the same carbon skeleton
    • C07C235/32—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to acyclic carbon atoms and singly-bound oxygen atoms bound to the same carbon skeleton the carbon skeleton containing six-membered aromatic rings
    • C07C235/34—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to acyclic carbon atoms and singly-bound oxygen atoms bound to the same carbon skeleton the carbon skeleton containing six-membered aromatic rings having the nitrogen atoms of the carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C235/00—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms
    • C07C235/42—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to carbon atoms of six-membered aromatic rings and singly-bound oxygen atoms bound to the same carbon skeleton
    • 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
    • C12N9/88—Lyases (4.)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y401/00—Carbon-carbon lyases (4.1)
    • C12Y401/01—Carboxy-lyases (4.1.1)
    • C12Y401/01015—Glutamate decarboxylase (4.1.1.15)
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • 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)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Neurology (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Neurosurgery (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN201780020853.3A 2016-03-28 2017-03-27 用于治疗神经元的过度兴奋的方法和组合物 Active CN108884022B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662314128P 2016-03-28 2016-03-28
US62/314,128 2016-03-28
PCT/US2017/024285 WO2017172606A1 (en) 2016-03-28 2017-03-27 Method and composition for treating neuronal hyper-excitability

Publications (2)

Publication Number Publication Date
CN108884022A CN108884022A (zh) 2018-11-23
CN108884022B true CN108884022B (zh) 2022-01-28

Family

ID=59966409

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201780020853.3A Active CN108884022B (zh) 2016-03-28 2017-03-27 用于治疗神经元的过度兴奋的方法和组合物

Country Status (8)

Country Link
US (1) US11472859B2 (https=)
EP (1) EP3436427B1 (https=)
JP (1) JP6949867B2 (https=)
KR (1) KR102489437B1 (https=)
CN (1) CN108884022B (https=)
AU (1) AU2017241534A1 (https=)
CA (1) CA3018960C (https=)
WO (1) WO2017172606A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10688285B2 (en) 2015-01-30 2020-06-23 The Regents Of The University Of California Spinal subpial gene delivery system
AU2017241534A1 (en) 2016-03-28 2018-10-04 The Regents Of The University Of California Method and composition for treating neuronal hyper-excitability
CA3074587A1 (en) * 2017-09-08 2019-03-14 The Regents Of The University Of California Method and composition for treating neuropathic pain
GB201810301D0 (en) * 2018-06-22 2018-08-08 Evox Therapeutics Ltd Combinatorial gene therapy
CN113260317B (zh) 2019-01-10 2024-05-07 加利福尼亚大学董事会 软膜下递送系统和使用方法
USD963164S1 (en) 2020-01-09 2022-09-06 The Regents Of The University Of California Surgical needle
WO2025070749A1 (ja) * 2023-09-29 2025-04-03 田辺三菱製薬株式会社 脊髄又は脳の障害により引き起こされる疾患に伴う随伴症状の予防又は治療剤

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014116652A2 (en) * 2013-01-23 2014-07-31 The Regents Of The University Of California Method and composition for treating spasticity

Family Cites Families (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5292722A (en) 1992-11-06 1994-03-08 Brigham And Women's Hospital Intravenous solution that diminishes body protein loss
FR2705361B1 (fr) 1993-05-18 1995-08-04 Centre Nat Rech Scient Vecteurs viraux et utilisation en thérapie génique.
EP0705344B8 (en) 1993-06-24 2006-05-10 Advec Inc. Adenovirus vectors for gene therapy
SK282843B6 (sk) 1993-07-13 2002-12-03 Rhone-Poulenc Rorer S. A. Defektný rekombinantný adenovírus a farmaceutický prostriedok s jeho obsahom
WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
WO1995016772A1 (en) 1993-12-14 1995-06-22 Cornell Research Foundation, Inc. Adenovirus gene expression system
FR2716893B1 (fr) 1994-03-03 1996-04-12 Rhone Poulenc Rorer Sa Virus recombinants, leur préparation et leur utilisation thérapeutique.
AUPM452094A0 (en) 1994-03-17 1994-04-14 University Of Queensland, The Waste treatment plant and process
US5643286A (en) 1994-06-24 1997-07-01 Cytotherapeutics, Inc. Microdrive for use in stereotactic surgery
CA2207927A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
US20020007145A1 (en) 1998-10-23 2002-01-17 Timothy Stivland Catheter having improved bonding region
CA2363254C (en) 1999-03-07 2009-05-05 Discure Ltd. Method and apparatus for computerized surgery
WO2002020753A2 (en) 2000-09-01 2002-03-14 Lexicon Genetics Incorporated Human gaba transporter protein and polynucleotides encoding the same
US7011647B2 (en) 2001-07-13 2006-03-14 Scimed Life Systems, Inc. Introducer sheath
ATE524121T1 (de) 2004-11-24 2011-09-15 Abdou Samy Vorrichtungen zur platzierung eines orthopädischen intervertebralen implantats
US20070213292A1 (en) 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
KR20080007968A (ko) 2006-07-19 2008-01-23 연세대학교 산학협력단 Ｇａｄ65를 발현하는 재조합 벡터를 포함하는 신경병증성통증 완화용 조성물
EP2066791B1 (en) 2006-10-03 2012-09-12 Genzyme Corporation Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
CA2710969C (en) 2008-01-14 2016-02-09 I-V Access Technology, Inc. An apparatus for peripheral vascular access
EP2274041B1 (en) 2008-04-04 2016-06-08 The Cleveland Clinic Foundation Spinal platform for delivering a therapeutic agent to a spinal cord target
WO2010071832A1 (en) 2008-12-19 2010-06-24 Nationwide Children's Hospital Delivery of polynucleotides across the blood brain barrier using recombinant aav9
WO2011057171A1 (en) 2009-11-08 2011-05-12 Quark Pharmaceuticals, Inc. METHODS FOR DELIVERY OF siRNA TO THE SPINAL CORD AND THERAPIES ARISING THEREFROM
DK2621282T3 (da) 2010-09-28 2020-05-04 Univ California Gaba-agonister i behandlingen af forstyrrelser forbundet med metabolisk syndrom og gaba-kombinationer i behandling eller profylakse af type i diabetes
US20120310140A1 (en) 2010-12-01 2012-12-06 Spinal Modulation, Inc. Directed delivery of agents to neural anatomy
JP6188728B2 (ja) 2012-02-07 2017-08-30 グローバル・バイオ・セラピューティクス・インコーポレイテッドＧｌｏｂａｌ Ｂｉｏ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 核酸送達の区画化方法ならびにその組成物および使用
EP2897670A4 (en) 2012-09-24 2016-05-25 Univ California INJECTION SYSTEM FOR BACKMARK WITH PULSATION SUPPRESSION
GB201308917D0 (en) 2013-05-17 2013-07-03 Renishaw Plc Delivery
US10688285B2 (en) 2015-01-30 2020-06-23 The Regents Of The University Of California Spinal subpial gene delivery system
US20180008727A1 (en) 2015-01-30 2018-01-11 The Regents Of The University Of California Spinal subpial gene delivery system
US20170151416A1 (en) * 2015-12-01 2017-06-01 Invivo Therapeutics Corporation Methods and Systems for Delivery of a Trail of a Therapeutic Substance into an Anatomical Space
AU2017241534A1 (en) 2016-03-28 2018-10-04 The Regents Of The University Of California Method and composition for treating neuronal hyper-excitability

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014116652A2 (en) * 2013-01-23 2014-07-31 The Regents Of The University Of California Method and composition for treating spasticity

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Changes in vesicular glutamate transporter 2, vesicular GABA transporter and vesicular acetylcholine transporter labeling of sacrocaudal motoneurons in the spastic rat;Patrick Kitzman;《Experimental Neurology》;20060201;第197卷(第2期);第407-419页 *

Also Published As

Publication number Publication date
JP6949867B2 (ja) 2021-10-13
CA3018960A1 (en) 2017-10-05
WO2017172606A1 (en) 2017-10-05
CA3018960C (en) 2023-09-26
EP3436427A1 (en) 2019-02-06
CN108884022A (zh) 2018-11-23
JP2019510047A (ja) 2019-04-11
US11472859B2 (en) 2022-10-18
US20190071486A1 (en) 2019-03-07
NZ746414A (en) 2024-11-29
AU2017241534A1 (en) 2018-10-04
EP3436427B1 (en) 2021-12-08
KR20180124980A (ko) 2018-11-21
EP3436427A4 (en) 2019-08-28
KR102489437B1 (ko) 2023-01-16

Similar Documents

Publication Publication Date Title
CN108884022B (zh) 用于治疗神经元的过度兴奋的方法和组合物
WO2015158924A1 (en) A method of treating peripheral neuropathies and motor neuron diseases
CN120324641A (zh) 用于治疗达农病的基因治疗载体
JP7430917B2 (ja) 眼圧及び頭蓋内圧を調節するための材料及び方法
EP4093754A1 (en) Zinc finger protein transcription factors for repressing tau expression
US8642341B2 (en) Materials and methods for gene mediated therapy of psychiatric disorders
TW202426033A (zh) Bag3用於治療心臟澱粉樣變性之方法及用途
JP2023089148A (ja) 神経障害性疼痛を処置するための方法および組成物
HK1262485A1 (en) Method and composition for treating neuronal hyper-excitability
HK1262485B (zh) 用於治疗神经元的过度兴奋的方法和组合物
WO2024079292A1 (en) Gene therapy treatment

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
REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 1262485

Country of ref document: HK

GR01 Patent grant
GR01 Patent grant