PT3058959T - Terapia genética para distúrbios neurometabólicos - Google Patents

Terapia genética para distúrbios neurometabólicos

Info

Publication number
PT3058959T
PT3058959T PT16162716T PT16162716T PT3058959T PT 3058959 T PT3058959 T PT 3058959T PT 16162716 T PT16162716 T PT 16162716T PT 16162716 T PT16162716 T PT 16162716T PT 3058959 T PT3058959 T PT 3058959T
Authority
PT
Portugal
Prior art keywords
disorders
disease
nervous system
central nervous
gene therapy
Prior art date
Application number
PT16162716T
Other languages
English (en)
Inventor
A Passini Marco
Dodge James
R Stewart Gregory
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 PT3058959T publication Critical patent/PT3058959T/pt

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • 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
    • C12N2750/14041—Use of virus, viral particle or viral elements as a 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
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
PT16162716T 2005-05-02 2006-05-02 Terapia genética para distúrbios neurometabólicos PT3058959T (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US67705705P 2005-05-02 2005-05-02
US68580805P 2005-05-31 2005-05-31

Publications (1)

Publication Number Publication Date
PT3058959T true PT3058959T (pt) 2019-05-24

Family

ID=37308324

Family Applications (4)

Application Number Title Priority Date Filing Date
PT111696332T PT2420256T (pt) 2005-05-02 2006-05-02 Terapia genética para distúrbios neurometabólicos
PT06759081T PT1879624E (pt) 2005-05-02 2006-05-02 Terapia genética para distúrbios neuro-metabólicos
PT16162716T PT3058959T (pt) 2005-05-02 2006-05-02 Terapia genética para distúrbios neurometabólicos
PT19153520T PT3520823T (pt) 2005-05-02 2006-05-02 Terapia genética para distúrbios neurometabólicos

Family Applications Before (2)

Application Number Title Priority Date Filing Date
PT111696332T PT2420256T (pt) 2005-05-02 2006-05-02 Terapia genética para distúrbios neurometabólicos
PT06759081T PT1879624E (pt) 2005-05-02 2006-05-02 Terapia genética para distúrbios neuro-metabólicos

Family Applications After (1)

Application Number Title Priority Date Filing Date
PT19153520T PT3520823T (pt) 2005-05-02 2006-05-02 Terapia genética para distúrbios neurometabólicos

Country Status (13)

Country Link
US (2) US10632213B2 (pt)
EP (4) EP2420256B1 (pt)
JP (3) JP5829372B2 (pt)
CN (4) CN101212988A (pt)
AT (1) ATE525092T1 (pt)
AU (1) AU2006243776A1 (pt)
BR (1) BRPI0611379A2 (pt)
CA (2) CA2607173C (pt)
ES (1) ES2887076T3 (pt)
IL (2) IL187078A (pt)
PL (4) PL2420256T3 (pt)
PT (4) PT2420256T (pt)
WO (1) WO2006119458A1 (pt)

Families Citing this family (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP5766389B2 (ja) * 2005-05-02 2015-08-19 ジェンザイム・コーポレーション 脊髄疾患の遺伝子療法
WO2006119458A1 (en) 2005-05-02 2006-11-09 Genzyme Corporation Gene therapy for neurometabolic disorders
AR061282A1 (es) 2006-06-07 2008-08-20 Genzyme Corp Terapia genica para esclerosis lateral amiotrofica y otros trastornos del cordon espinal
LT2158322T (lt) 2007-06-06 2017-08-25 Genzyme Corporation Lizosomų kaupimo ligų genų terapija
EP2352847B1 (en) 2008-11-10 2014-01-08 The United States of America, as represented by The Secretary, Department of Health and Human Services Gene signature for predicting prognosis of patients with solid tumors
PL2424991T3 (pl) 2009-05-02 2018-11-30 Genzyme Corporation Terapia genowa dla zaburzeń neurodegeneracyjnych
US9150926B2 (en) 2010-12-06 2015-10-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Diagnosis and treatment of adrenocortical tumors using human microRNA-483
WO2012145646A1 (en) * 2011-04-20 2012-10-26 Miguel Sena-Esteves Methods for the treatment of tay-sachs disease, sandhoff disease, and gmi-gangliosidosis
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
CA2966620A1 (en) 2014-11-05 2016-05-12 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
US10570395B2 (en) 2014-11-14 2020-02-25 Voyager Therapeutics, Inc. Modulatory polynucleotides
EP3218484A4 (en) 2014-11-14 2018-05-30 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US11319555B2 (en) 2014-11-20 2022-05-03 Duke University Compositions, systems and methods for cell therapy
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
CN112472796A (zh) * 2015-03-26 2021-03-12 苏州澳宗生物科技有限公司 使用p75ecd和/或p75诊断或治疗神经障碍的方法
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
WO2016196507A1 (en) * 2015-05-29 2016-12-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
KR102427379B1 (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
SG11201809699XA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Modulatory polynucleotides
CA3035522A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
CN111108198A (zh) 2017-05-05 2020-05-05 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
JP2020518258A (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． 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法
CA3066569A1 (en) 2017-06-07 2018-12-13 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
WO2019018342A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. NETWORK EQUIPMENT TRACK GUIDE SYSTEM
JP7221275B2 (ja) 2017-08-03 2023-02-13 ボイジャー セラピューティクス インコーポレイテッド Ａａｖを送達するための組成物および方法
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
EP3697905A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SE8303626D0 (sv) 1983-06-23 1983-06-23 Kabigen Ab A recombinant plasmid a transformant microorganism, a polydoxyrebonucleotide segment, a process for producing a biologically active protein, and the protein thus produced
US5672344A (en) 1987-12-30 1997-09-30 The Regents Of The University Of Michigan Viral-mediated gene transfer system
US5773278A (en) 1991-05-03 1998-06-30 Mount Sinai Medical Center Acid sphingomyelinase gene
CA2166118C (en) 1993-06-24 2007-04-17 Frank L. Graham Adenovirus vectors for gene therapy
DE69435223D1 (de) 1993-10-25 2009-09-03 Canji Inc Rekombinanter Adenoviren-Vektor und Verfahren zur Verwendung
DE69433592T2 (de) 1993-11-09 2005-02-10 Targeted Genetics Corp., Seattle Die erzielung hoher titer des rekombinanten aav-vektors
US7252989B1 (en) 1994-04-04 2007-08-07 Board Of Regents, The University Of Texas System Adenovirus supervector system
CA2187626C (en) 1994-04-13 2009-11-03 Michael G. Kaplitt Aav-mediated delivery of dna to cells of the nervous system
AU1525797A (en) 1996-04-22 1997-11-12 Medtronic, Inc. Two-stage angled venous cannula
US6544785B1 (en) 1998-09-14 2003-04-08 Mount Sinai School Of Medicine Of New York University Helper-free rescue of recombinant negative strand RNA viruses
US6582692B1 (en) * 1999-11-17 2003-06-24 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
US20030165481A1 (en) * 2000-02-24 2003-09-04 Hersh Louis B. Amyloid peptide inactivating enzyme to treat Alzheimer's disease
US6667174B2 (en) 2000-09-18 2003-12-23 Genzyme Corporation Expression vectors containing hybrid ubiquitin promoters
US20030050273A1 (en) * 2001-08-29 2003-03-13 Keiya Ozawa Compositions and methods for treating neurodegenerative diseases
US7232670B2 (en) * 2001-09-28 2007-06-19 St. Jude Children's Research Hospital Targeting proteins to cells expressing mannose receptors via expression in insect cells
EP2359869B1 (en) 2001-12-17 2018-12-26 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same and uses therefor
WO2003055983A2 (en) 2001-12-21 2003-07-10 The Salk Institute For Biological Studies Targeted retrograde gene delivery to motor neurons
US6998118B2 (en) 2001-12-21 2006-02-14 The Salk Institute For Biological Studies Targeted retrograde gene delivery for neuronal protection
WO2003100031A2 (en) * 2002-05-20 2003-12-04 Board Of Regents, The University Of Texas System Methods and compositions for delivering enzymes and nucleic acid molecules to brain, bone, and other tissues
US20040258666A1 (en) * 2003-05-01 2004-12-23 Passini Marco A. Gene therapy for neurometabolic disorders
SI1620133T1 (sl) * 2003-05-01 2016-03-31 Genzyme Corporation Genska terapija za nevrometabolne motnje
US7740168B2 (en) 2003-08-18 2010-06-22 Visa U.S.A. Inc. Method and system for generating a dynamic verification value
AU2003272868A1 (en) 2003-10-15 2005-04-27 Agtc Gene Technology Company Ltd. Method for large-scale production, isolation, purification and the uses of multi-type recombinant adeno-associated virus vectors
EP1716870A1 (en) 2005-04-29 2006-11-02 Bracco Imaging S.p.A. MRI contrast agents endowed with concentration independent responsiveness
JP5766389B2 (ja) 2005-05-02 2015-08-19 ジェンザイム・コーポレーション 脊髄疾患の遺伝子療法
WO2006119458A1 (en) 2005-05-02 2006-11-09 Genzyme Corporation Gene therapy for neurometabolic disorders
SI1986661T1 (sl) 2006-02-08 2018-12-31 Genzyme Corporation Genska terapija za Niemann-Pickovo bolezen tipa A
US20150030561A1 (en) 2011-05-16 2015-01-29 Genzyme Corp. Use of CXCR4 Antagonists
TWI821227B (zh) 2017-12-22 2023-11-11 美商健臻公司 胺甲喋呤誘發的免疫耐受性之生物標記

Also Published As

Publication number Publication date
ATE525092T1 (de) 2011-10-15
PL1879624T3 (pl) 2012-02-29
CA2607173C (en) 2018-04-24
CA2998603C (en) 2019-07-23
CA2607173A1 (en) 2006-11-09
EP2420256B1 (en) 2016-06-22
EP3520823A1 (en) 2019-08-07
CN101212988A (zh) 2008-07-02
PT2420256T (pt) 2016-09-13
US11957765B2 (en) 2024-04-16
EP3520823B1 (en) 2021-06-30
IL187078A0 (en) 2008-02-09
PT1879624E (pt) 2012-02-02
PL3520823T3 (pl) 2021-11-22
EP1879624A1 (en) 2008-01-23
JP5829372B2 (ja) 2015-12-09
CN107362371A (zh) 2017-11-21
US20200306387A1 (en) 2020-10-01
EP3058959B1 (en) 2019-01-30
BRPI0611379A2 (pt) 2010-08-31
CN107007842A (zh) 2017-08-04
JP2014037418A (ja) 2014-02-27
JP2008540445A (ja) 2008-11-20
IL239167A0 (en) 2015-07-30
CA2998603A1 (en) 2006-11-09
EP1879624A4 (en) 2009-03-25
EP2420256A1 (en) 2012-02-22
ES2887076T3 (es) 2021-12-21
US20080292593A1 (en) 2008-11-27
EP1879624B1 (en) 2011-09-21
EP3058959A1 (en) 2016-08-24
US20140356327A9 (en) 2014-12-04
JP6338560B2 (ja) 2018-06-06
WO2006119458A9 (en) 2007-02-15
WO2006119458A1 (en) 2006-11-09
PL3058959T3 (pl) 2019-07-31
PT3520823T (pt) 2021-09-16
AU2006243776A1 (en) 2006-11-09
JP2016041706A (ja) 2016-03-31
US10632213B2 (en) 2020-04-28
PL2420256T3 (pl) 2016-12-30
IL187078A (en) 2015-06-30
CN104306986A (zh) 2015-01-28

Similar Documents

Publication Publication Date Title
PL1879624T3 (pl) Terapia genowa dla zaburzeń neurometabolicznych
WO2005120581A3 (en) Gene therapy for neurometabolic disorders
Snyder et al. Taking stock and planning for the next decade: realistic prospects for stem cell therapies for the nervous system
UA92472C2 (ru) Фармацевтическая композиция, которая содержит 4-{4-[3-(4-хлор-3-трифторметилфенил)-уреидо]- 3-фторфенокси}-пиридин-2-карбоновой кислоты метиламид (варианты), способ получения твердой дисперсии указанного соединения и способ лечения с применением указанного соединения (варианты)
CN108601766A (zh) Cxcr2抑制剂
HK1103363A1 (en) Treatment of glycogen storage diseases type ii ii
GB0421639D0 (en) Methods and compositions relating to alzheimer's disease
WO2003087368A3 (en) Means and methods for the specific modulation of target genes in the cns and the eye and methods for their identification
JP2002540162A5 (pt)
WO2008103993A3 (en) Compositions and methods for treating glycogen storage diseases
MX2021012184A (es) Terapias genicas para trastornos lisosomales.
Kim et al. Amyotrophic lateral sclerosis-cell based therapy and novel therapeutic development
WO2006057945A3 (en) 2,3,4,6-substituted pyridyl derivative compounds useful as beta-secretase inhibitors for the treatment of alzheimer's disease
WO2006083797A3 (en) Improving and protecting cell therapy for neurological disorders including parkinson's disease
Mendell et al. 480. Gene therapy for spinal muscular atrophy type 1 shows potential to improve survival and motor functional outcomes
EP1786474A4 (en) COMBINED GENE THERAPY FOR THE TREATMENT OF MACROSCOPIC GLIOMES
Gao et al. Enhancement of neural regeneration as a therapeutic strategy for Alzheimer's disease
Palmieri et al. Advanced genetic therapies for the treatment of Rett syndrome: state of the art and future perspectives
WO2002007774A3 (en) Methods for therapy of neurodegenerative disease of the brain
EP1149917A3 (en) Hepatitis B virus vectors for gene therapy
Hernandez-Baltazar et al. Animal model of Parkinson disease: Neuroinflammation and apoptosis in the 6-hydroxydopamine-induced model
WO2022035903A3 (en) Gene therapies for lysosomal disorders
WO2022256499A3 (en) Bcma targeting trispecific proteins and methods of use
WO2001055410A3 (en) Ceramidase compositions and methods based thereon
WO2006044665A3 (en) Neuroprotective spirostenol pharmaceutical compositions