PT3520823T - Terapia genética para distúrbios neurometabólicos - Google Patents

Terapia genética para distúrbios neurometabólicos

Info

Publication number
PT3520823T
PT3520823T PT19153520T PT19153520T PT3520823T PT 3520823 T PT3520823 T PT 3520823T PT 19153520 T PT19153520 T PT 19153520T PT 19153520 T PT19153520 T PT 19153520T PT 3520823 T PT3520823 T PT 3520823T
Authority
PT
Portugal
Prior art keywords
disorders
disease
nervous system
central nervous
gene therapy
Prior art date
Application number
PT19153520T
Other languages
English (en)
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 PT3520823T publication Critical patent/PT3520823T/pt

Links

Classifications

    • 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
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/06Antihyperlipidemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14041Use of virus, viral particle or viral elements as a 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
    • C12N2750/14141Use 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
    • C12N2799/00Uses of viruses
    • C12N2799/02Uses of viruses as vector
    • C12N2799/021Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
PT19153520T 2005-05-02 2006-05-02 Terapia genética para distúrbios neurometabólicos PT3520823T (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
PT3520823T true PT3520823T (pt) 2021-09-16

Family

ID=37308324

Family Applications (4)

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

Family Applications Before (1)

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

Family Applications After (2)

Application Number Title Priority Date Filing Date
PT111696332T PT2420256T (pt) 2005-05-02 2006-05-02 Terapia genética para distúrbios neurometabólicos
PT16162716T PT3058959T (pt) 2005-05-02 2006-05-02 Terapia genética para distúrbios neurometabólicos

Country Status (13)

Country Link
US (2) US10632213B2 (pt)
EP (4) EP1879624B1 (pt)
JP (3) JP5829372B2 (pt)
CN (4) CN107362371A (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) PL3520823T3 (pt)
PT (4) PT1879624E (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
PL1879623T3 (pl) * 2005-05-02 2013-03-29 Genzyme Corp Terapia genowa zaburzeń rdzenia kręgowego
AU2006243776A1 (en) 2005-05-02 2006-11-09 Genzyme Corporation Gene therapy for neurometabolic disorders
ES2725552T3 (es) 2006-06-07 2019-09-24 Genzyme Corp Terapia génica para esclerosis lateral amiotrófica y otros trastornos de la médula espinal
PL2158322T3 (pl) * 2007-06-06 2017-10-31 Genzyme Corp Terapia genowa dla lizosomalnych chorób spichrzeniowych
EP2687609B1 (en) 2008-11-10 2017-01-04 The United States of America, as represented by The Secretary, Department of Health and Human Services Method for treating solid tumor
ES2686504T3 (es) 2009-05-02 2018-10-18 Genzyme Corporation Terapia génica para trastornos neurodegenerativos
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
US20130090374A1 (en) * 2011-04-20 2013-04-11 Miguel Sena-Esteves Methods for the treatment of tay-sachs disease, sandhoff disease, and gm1-gangliosidosis
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
CN112553229A (zh) 2014-11-05 2021-03-26 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
CN107207556B (zh) 2014-11-14 2020-12-08 沃雅戈治疗公司 调节性多核苷酸
RU2020108189A (ru) 2014-11-14 2020-03-11 Вояджер Терапьютикс, Инк. Композиции и способы лечения бокового амиотрофического склероза (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
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
US11951121B2 (en) 2016-05-18 2024-04-09 Voyager Therapeutics, Inc. Compositions and methods for treating Huntington's disease
JP7066635B2 (ja) 2016-05-18 2022-05-13 ボイジャー セラピューティクス インコーポレイテッド 調節性ポリヌクレオチド
WO2018044933A1 (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
US11752181B2 (en) 2017-05-05 2023-09-12 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
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
CN110809583A (zh) 2017-06-07 2020-02-18 瑞泽恩制药公司 用于内化酶的组合物和方法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CN111132626B (zh) 2017-07-17 2024-01-30 沃雅戈治疗公司 轨迹阵列引导系统
WO2019028306A2 (en) 2017-08-03 2019-02-07 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
AU2018352236A1 (en) 2017-10-16 2020-04-23 The Curators Of The University Of Missouri 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
AU687829B2 (en) 1993-06-24 1998-03-05 Advec, Inc. Adenovirus vectors for gene therapy
SK283703B6 (sk) 1993-10-25 2003-12-02 Canji, Inc. Rekombinantný adenovírusový vektor a jeho použitie
DK0733103T3 (da) 1993-11-09 2004-07-12 Univ Johns Hopkins Fremstilling af höje titre af rekombinante AAV vektorer
US7252989B1 (en) 1994-04-04 2007-08-07 Board Of Regents, The University Of Texas System Adenovirus supervector system
ATE386131T1 (de) 1994-04-13 2008-03-15 Univ Rockefeller Aav-vermittelte überbringung von dna in zellen des nervensystems
WO1997039789A1 (en) 1996-04-22 1997-10-30 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
DE60115070T2 (de) 2000-09-18 2006-07-27 Genzyme Corp., Cambridge Expressionsvektoren mit hybriden ubiquitin-promotoren
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
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
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
AU2003220115A1 (en) * 2002-05-20 2003-12-12 Board Of Regents, The University Of Texas System Methods and compositions for delivering enzymes and nucleic acid molecules to brain, bone, and other tissues
SI1620133T1 (sl) * 2003-05-01 2016-03-31 Genzyme Corporation Genska terapija za nevrometabolne motnje
US20040258666A1 (en) * 2003-05-01 2004-12-23 Passini Marco A. Gene therapy for neurometabolic disorders
US7740168B2 (en) 2003-08-18 2010-06-22 Visa U.S.A. Inc. Method and system for generating a dynamic verification value
US20070275449A1 (en) 2003-10-15 2007-11-29 Vector 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
AU2006243776A1 (en) 2005-05-02 2006-11-09 Genzyme Corporation Gene therapy for neurometabolic disorders
PL1879623T3 (pl) 2005-05-02 2013-03-29 Genzyme Corp Terapia genowa zaburzeń rdzenia kręgowego
MX360595B (es) 2006-02-08 2018-11-09 Genzyme Corp Terapia génica para la enfermedad de niemann-pick de tipo a.
JP2014513727A (ja) 2011-05-16 2014-06-05 ジェンザイム・コーポレーション Cxcr4拮抗薬の使用
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
EP1879624A4 (en) 2009-03-25
PL3058959T3 (pl) 2019-07-31
PL3520823T3 (pl) 2021-11-22
ES2887076T3 (es) 2021-12-21
ATE525092T1 (de) 2011-10-15
EP3520823B1 (en) 2021-06-30
CN101212988A (zh) 2008-07-02
JP2014037418A (ja) 2014-02-27
PL2420256T3 (pl) 2016-12-30
CN104306986A (zh) 2015-01-28
PT2420256T (pt) 2016-09-13
EP3058959A1 (en) 2016-08-24
CA2998603A1 (en) 2006-11-09
JP5829372B2 (ja) 2015-12-09
IL239167A0 (en) 2015-07-30
EP2420256A1 (en) 2012-02-22
US10632213B2 (en) 2020-04-28
PT3058959T (pt) 2019-05-24
AU2006243776A1 (en) 2006-11-09
CA2607173A1 (en) 2006-11-09
US20140356327A9 (en) 2014-12-04
US20080292593A1 (en) 2008-11-27
EP3058959B1 (en) 2019-01-30
US20200306387A1 (en) 2020-10-01
PL1879624T3 (pl) 2012-02-29
WO2006119458A9 (en) 2007-02-15
CN107362371A (zh) 2017-11-21
JP6338560B2 (ja) 2018-06-06
CN107007842A (zh) 2017-08-04
EP2420256B1 (en) 2016-06-22
US11957765B2 (en) 2024-04-16
WO2006119458A1 (en) 2006-11-09
JP2016041706A (ja) 2016-03-31
EP1879624B1 (en) 2011-09-21
JP2008540445A (ja) 2008-11-20
IL187078A (en) 2015-06-30
IL187078A0 (en) 2008-02-09
CA2607173C (en) 2018-04-24
CA2998603C (en) 2019-07-23
EP3520823A1 (en) 2019-08-07
PT1879624E (pt) 2012-02-02
EP1879624A1 (en) 2008-01-23
BRPI0611379A2 (pt) 2010-08-31

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
HRP20171165T1 (hr) Genska terapija za lizosomske bolesti nakupljanja
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)
HK1103363A1 (en) Treatment of glycogen storage diseases type ii ii
MX2009002816A (es) Proteinas de fusion de albumina.
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
WO2007035722A8 (en) Methods and compositions for stimulating neurogenesis and inhibiting neuronal degeneration
Mendell et al. 480. Gene therapy for spinal muscular atrophy type 1 shows potential to improve survival and motor functional outcomes
WO2007132292A3 (en) Therapy for alzheimer's disease
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
WO2002029091A3 (en) Methods and compositions for the treatment of inflammatory diseases
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
WO2001055410A3 (en) Ceramidase compositions and methods based thereon
WO2006044665A3 (en) Neuroprotective spirostenol pharmaceutical compositions
WO2005074605A3 (en) Recombinant molecular products for expression of heme oxygenase-1, heme oxygenase-2, and/or biliverdin reductase for treatment of neurodegenerative diseases or disorders
WO2003105761A3 (en) IMMUNOTOXIN AS THERAPEUTIC AGENT AND USES THEREOF