IL135578A0 - Compositions and methods for treating lysosomal storage disease - Google Patents
Compositions and methods for treating lysosomal storage diseaseInfo
- Publication number
- IL135578A0 IL135578A0 IL13557898A IL13557898A IL135578A0 IL 135578 A0 IL135578 A0 IL 135578A0 IL 13557898 A IL13557898 A IL 13557898A IL 13557898 A IL13557898 A IL 13557898A IL 135578 A0 IL135578 A0 IL 135578A0
- Authority
- IL
- Israel
- Prior art keywords
- compositions
- methods
- lysosomal storage
- storage disease
- treating lysosomal
- Prior art date
Links
- 208000015439 Lysosomal storage disease Diseases 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 108090000790 Enzymes Proteins 0.000 abstract 3
- 102000004190 Enzymes Human genes 0.000 abstract 3
- 108700019146 Transgenes Proteins 0.000 abstract 2
- 230000002132 lysosomal effect Effects 0.000 abstract 2
- 230000002950 deficient Effects 0.000 abstract 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- 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
- 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
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
- C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
-
- 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
- C12N2799/022—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from an adenovirus
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Epidemiology (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Microbiology (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Enzymes And Modification Thereof (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US6352797P | 1997-10-29 | 1997-10-29 | |
PCT/US1998/022886 WO2000009153A1 (en) | 1997-10-29 | 1998-10-29 | Compositions and methods for treating lysosomal storage disease |
Publications (1)
Publication Number | Publication Date |
---|---|
IL135578A0 true IL135578A0 (en) | 2001-05-20 |
Family
ID=22049811
Family Applications (3)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
IL13557898A IL135578A0 (en) | 1997-10-29 | 1998-10-29 | Compositions and methods for treating lysosomal storage disease |
IL135578A IL135578A (en) | 1997-10-29 | 2000-04-10 | Use of a vector for the manufacture of a medicament for treating gaucher disease |
IL177228A IL177228A0 (en) | 1997-10-29 | 2006-08-02 | Use of a vector for the manufacture of a medicament for treating gaucher disease |
Family Applications After (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
IL135578A IL135578A (en) | 1997-10-29 | 2000-04-10 | Use of a vector for the manufacture of a medicament for treating gaucher disease |
IL177228A IL177228A0 (en) | 1997-10-29 | 2006-08-02 | Use of a vector for the manufacture of a medicament for treating gaucher disease |
Country Status (10)
Country | Link |
---|---|
US (3) | US6066626A (xx) |
EP (2) | EP1027069B1 (xx) |
JP (2) | JP2002522509A (xx) |
AT (1) | ATE333893T1 (xx) |
AU (1) | AU734290B2 (xx) |
CA (1) | CA2305768A1 (xx) |
DE (1) | DE69835367T2 (xx) |
ES (1) | ES2268799T3 (xx) |
IL (3) | IL135578A0 (xx) |
WO (1) | WO2000009153A1 (xx) |
Families Citing this family (83)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5362442A (en) * | 1993-07-22 | 1994-11-08 | 2920913 Canada Inc. | Method for sterilizing products with gamma radiation |
US20040067157A1 (en) * | 1993-07-22 | 2004-04-08 | Clearant, Inc. | Methods for sterilizing biological materials |
US6705323B1 (en) * | 1995-06-07 | 2004-03-16 | Conceptus, Inc. | Contraceptive transcervical fallopian tube occlusion devices and methods |
CA2305768A1 (en) * | 1997-10-29 | 2000-02-24 | Genzyme Corporation | Compositions and methods for treating lysosomal storage disease |
AU764686B2 (en) | 1998-08-28 | 2003-08-28 | Duke University | Adenoviruses deleted in the IVa2, 100K, polymerase and/or preterminal protein sequences |
WO2001036603A2 (en) * | 1999-11-17 | 2001-05-25 | Avigen, Inc. | Recombinant adeno-associated virus virions for the treatment of lysosomal disorders |
JP2003523330A (ja) | 2000-02-04 | 2003-08-05 | チルドレンズ・ホスピタル・リサーチ・ファウンデイション | アテローム性動脈硬化症および関連疾患のための脂質加水分解治療 |
AU2001237050A1 (en) * | 2000-02-17 | 2001-08-27 | Genzyme Corporation | Genetic modification of the lung as a portal for gene delivery |
WO2001060377A2 (en) * | 2000-02-17 | 2001-08-23 | Genzyme Corporation | Methods for treatment of lysosomal storage diseases using biphosphonates |
US20040086420A1 (en) * | 2000-03-23 | 2004-05-06 | Macphee Martin J. | Methods for sterilizing serum or plasma |
US20040204379A1 (en) * | 2000-06-19 | 2004-10-14 | Cheng Seng H. | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
US20020095135A1 (en) | 2000-06-19 | 2002-07-18 | David Meeker | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
US7723296B2 (en) | 2001-01-18 | 2010-05-25 | Genzyme Corporation | Methods for introducing mannose-6-phosphate and other oligosaccharides onto glycoproteins and its application thereof |
JP2004522763A (ja) * | 2001-02-20 | 2004-07-29 | ユーエイビー リサーチ ファンデーション | リソソーム蓄積症のアミノグリコシド治療 |
US6682695B2 (en) * | 2001-03-23 | 2004-01-27 | Clearant, Inc. | Methods for sterilizing biological materials by multiple rates |
US20040005309A1 (en) * | 2002-05-29 | 2004-01-08 | Symbiontics, Inc. | Targeted therapeutic proteins |
EP1974752B1 (en) * | 2001-04-30 | 2012-09-26 | BioMarin Pharmaceutical Inc. | Subcellular targeting of therapeutic proteins |
US7560424B2 (en) | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
US7629309B2 (en) | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
US6946126B2 (en) * | 2001-06-04 | 2005-09-20 | Duke University | Replicating adenovirus vectors |
US6696060B2 (en) * | 2001-06-14 | 2004-02-24 | Clearant, Inc. | Methods for sterilizing preparations of monoclonal immunoglobulins |
US20030031584A1 (en) * | 2001-08-10 | 2003-02-13 | Wilson Burgess | Methods for sterilizing biological materials using dipeptide stabilizers |
US6946098B2 (en) | 2001-08-10 | 2005-09-20 | Clearant, Inc. | Methods for sterilizing biological materials |
US6749851B2 (en) | 2001-08-31 | 2004-06-15 | Clearant, Inc. | Methods for sterilizing preparations of digestive enzymes |
US7252799B2 (en) * | 2001-08-31 | 2007-08-07 | Clearant, Inc. | Methods for sterilizing preparations containing albumin |
US20110091353A1 (en) * | 2001-09-24 | 2011-04-21 | Wilson Burgess | Methods for Sterilizing Tissue |
US20030095890A1 (en) * | 2001-09-24 | 2003-05-22 | Shirley Miekka | Methods for sterilizing biological materials containing non-aqueous solvents |
US6783968B2 (en) | 2001-09-24 | 2004-08-31 | Clearant, Inc. | Methods for sterilizing preparations of glycosidases |
US20030185702A1 (en) * | 2002-02-01 | 2003-10-02 | Wilson Burgess | Methods for sterilizing tissue |
US20030072761A1 (en) * | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
US20030124023A1 (en) * | 2001-12-21 | 2003-07-03 | Wilson Burgess | Method of sterilizing heart valves |
US20030180181A1 (en) * | 2002-02-01 | 2003-09-25 | Teri Greib | Methods for sterilizing tissue |
US7090836B2 (en) * | 2002-06-21 | 2006-08-15 | Institut Pasteur | Vector for expressing α-L-iduronidase and method of treating MPS I by stereotactic injection into the brain of a mammal |
US20040013562A1 (en) * | 2002-07-18 | 2004-01-22 | Wilson Burgess | Methods for sterilizing milk. |
US6908591B2 (en) * | 2002-07-18 | 2005-06-21 | Clearant, Inc. | Methods for sterilizing biological materials by irradiation over a temperature gradient |
CN1756558B (zh) * | 2003-01-31 | 2012-04-25 | 纽约大学西奈山医学院 | 酶和酶的活性部位特异性陪伴分子的组合 |
US7446098B2 (en) * | 2003-02-18 | 2008-11-04 | Mount Sinai School Of Medicine Of New York University | Combination therapy for treating protein deficiencies |
US20040258666A1 (en) * | 2003-05-01 | 2004-12-23 | Passini Marco A. | Gene therapy for neurometabolic disorders |
EP1682504B1 (en) * | 2003-11-12 | 2012-12-26 | Amicus Therapeutics Inc. | Hydroxy piperidine derivatives to treat gaucher disease |
US20050244400A1 (en) * | 2004-02-10 | 2005-11-03 | Zystor Therapeutics, Inc. | Acid alpha-glucosidase and fragments thereof |
US8889127B2 (en) * | 2004-07-01 | 2014-11-18 | Icahn School Of Medicine At Mount Sinai | Targeted protein replacement for the treatment of lysosomal storage disorders |
EP1961816B1 (en) | 2005-11-18 | 2015-09-02 | Tokyo Metropolitan Institute of Medical Science | Novel highly functional enzyme having modified substrate-specificity |
BRPI0707590A2 (pt) | 2006-02-08 | 2011-05-10 | Genzyme Corp | terapia gÊnica para a doenÇa de niemann-pick do tipo a |
EP2099523A2 (en) * | 2006-11-13 | 2009-09-16 | ZyStor Therapeutics , Inc. | Methods for treating pompe disease |
CN101631794B (zh) | 2007-01-18 | 2013-01-02 | 建新公司 | 包含氨基氧基基团的寡糖及其轭合物 |
WO2008143354A1 (ja) | 2007-05-18 | 2008-11-27 | Tokyo Metropolitan Organization For Medical Research | 酵素補充療法用医薬組成物 |
US8252789B2 (en) | 2007-11-29 | 2012-08-28 | Neuraltus Pharmaceuticals, Inc. | Compositions and methods for treating lysosomal disorders |
AU2009244148B2 (en) * | 2008-05-07 | 2014-10-09 | Biomarin Pharmaceutical Inc. | Lysosomal targeting peptides and uses thereof |
US20100119502A1 (en) * | 2008-11-11 | 2010-05-13 | Amicus Therapeutics, Inc. | Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease |
CN103432566A (zh) | 2008-12-16 | 2013-12-11 | 建新公司 | 寡糖-蛋白缀合物 |
US8785168B2 (en) | 2009-06-17 | 2014-07-22 | Biomarin Pharmaceutical Inc. | Formulations for lysosomal enzymes |
WO2011028941A2 (en) | 2009-09-04 | 2011-03-10 | The United States Of America, As Represented By The Secretary Department Of Health & Human Services | Disabling autophagy as a treatment for lysosomal storage diseases |
MX338426B (es) | 2010-04-23 | 2016-04-15 | Synageva Biopharma Corp | Enzima de enfermedad del almacenamiento lisosomico. |
NZ700824A (en) | 2010-09-09 | 2016-03-31 | Synageva Biopharma Corp | Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients |
RS55405B1 (sr) | 2010-11-08 | 2017-04-28 | Amicus Therapeutics Inc | Varijantni, rekombinantni proteini beta-glukocerebrozidaze sa povećanom stabilnošću i povećanom zadržanom katalitičkom aktivnošću |
US20120178105A1 (en) * | 2011-01-10 | 2012-07-12 | Genzyme Corporation | Detection of globotriaosylceramide (glc) in human urine samples using an antibody sandwich |
WO2012112681A1 (en) | 2011-02-15 | 2012-08-23 | Shire Human Genetic Therapies, Inc. | Methods for treating lysosomal acid lipase deficiency |
SI2685986T1 (sl) | 2011-03-18 | 2020-03-31 | Genzyme Corporation | Inhibitor glukozilceramid sintaze |
JP2014513952A (ja) | 2011-04-22 | 2014-06-19 | ジェンザイム・コーポレーション | 促進されたプロセシングを備えた修飾された酸性アルファグルコシダーゼ |
JP6184945B2 (ja) | 2011-06-08 | 2017-08-23 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | mRNA送達のための脂質ナノ粒子組成物および方法 |
GB201115280D0 (en) * | 2011-09-05 | 2011-10-19 | Alligator Bioscience Ab | Antibodies, uses and methods |
ES2791760T3 (es) | 2011-09-07 | 2020-11-05 | Sinai School Medicine | Ceramidasa y diferenciación celular |
US9492514B2 (en) | 2012-06-01 | 2016-11-15 | Icahn School Of Medicine At Mount Sinai | Ceramide levels in the treatment and prevention of infections |
MA37975B2 (fr) | 2012-09-11 | 2021-03-31 | Genzyme Corp | Inhibiteurs de synthase de glucosylcéramide |
ES2968649T3 (es) | 2012-12-07 | 2024-05-13 | Translate Bio Inc | Nanopartículas lipídicas para la administración de ARNm en los pulmones |
WO2014160390A1 (en) | 2013-03-14 | 2014-10-02 | Icahn School Of Medicine At Mount Sinai | Therapeutic acid ceramidase compositions and methods of making and using them |
JO3713B1 (ar) | 2013-03-15 | 2021-01-31 | Genzyme Corp | أشكال ملح (s)-كوينوكليدين-3-يل(2-(2-(4-فلوروفينيل)ثيازول-4-يل)بروبان-2-يل)كارباميت |
TW202332774A (zh) | 2013-10-23 | 2023-08-16 | 美商健臻公司 | 重組醣蛋白及其用途 |
US9981021B1 (en) | 2015-04-09 | 2018-05-29 | Kinetiq, Inc. | Subcutaneous therapeutic enzyme formulations, uses, and methods for generating thereof |
GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
US11219695B2 (en) * | 2016-10-20 | 2022-01-11 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of Fabry disease |
WO2020046132A1 (en) | 2018-08-31 | 2020-03-05 | Leiden University | Pharmacological chaperones for enzyme treatment therapy |
NL2021840B1 (en) | 2018-10-19 | 2020-05-13 | Univ Leiden | Pharmacological Chaperones For Enzyme Treatment Therapy |
US20220001027A1 (en) * | 2018-11-15 | 2022-01-06 | President And Fellows Of Harvard College | Genetically Engineered Skin Cells for the Systemic In Vivo Treatment of Deficient Enzymes, Factors or Proteins |
IL288284B2 (en) | 2019-05-22 | 2024-06-01 | Massachusetts Inst Technology | Circular Rana preparations and methods |
CN114786710A (zh) * | 2019-10-17 | 2022-07-22 | 西湖生物医药科技(杭州)有限公司 | 工程改造红细胞用于治疗溶酶体贮积病 |
EP3920976B1 (en) | 2019-12-04 | 2023-07-19 | Orna Therapeutics, Inc. | Circular rna compositions and methods |
AU2021215396A1 (en) | 2020-02-03 | 2022-09-29 | Genzyme Corporation | Methods for treating neurological symptoms associated with lysosomal storage diseases |
US20240245805A1 (en) | 2020-03-20 | 2024-07-25 | Orna Therapeutics, Inc. | Circular rna compositions and methods |
WO2021236855A1 (en) | 2020-05-19 | 2021-11-25 | Orna Therapeutics, Inc. | Circular rna compositions and methods |
US11857512B2 (en) | 2020-07-24 | 2024-01-02 | Genzyme Corporation | Pharmaceutical compositions comprising venglustat |
TW202317767A (zh) | 2021-06-10 | 2023-05-01 | 美商歐納醫療公司 | 環狀rna組合物及方法 |
US20240052049A1 (en) | 2022-06-24 | 2024-02-15 | Orna Therapeutics, Inc. | Circular rna encoding chimeric antigen receptors targeting bcma |
Family Cites Families (29)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US4647447A (en) | 1981-07-24 | 1987-03-03 | Schering Aktiengesellschaft | Diagnostic media |
US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
US5672344A (en) | 1987-12-30 | 1997-09-30 | The Regents Of The University Of Michigan | Viral-mediated gene transfer system |
US5236838A (en) * | 1988-12-23 | 1993-08-17 | Genzyme Corporation | Enzymatically active recombinant glucocerebrosidase |
US5179023A (en) | 1989-03-24 | 1993-01-12 | Research Corporation Technologies, Inc. | Recombinant α-galactosidase a therapy for Fabry disease |
US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
US5670488A (en) | 1992-12-03 | 1997-09-23 | Genzyme Corporation | Adenovirus vector for gene therapy |
US5401650A (en) | 1990-10-24 | 1995-03-28 | The Mount Sinai School Of Medicine Of The City University Of New York | Cloning and expression of biologically active α-galactosidase A |
US5283185A (en) | 1991-08-28 | 1994-02-01 | University Of Tennessee Research Corporation | Method for delivering nucleic acids into cells |
US5252479A (en) | 1991-11-08 | 1993-10-12 | Research Corporation Technologies, Inc. | Safe vector for gene therapy |
US5911983A (en) * | 1992-06-26 | 1999-06-15 | University Of Pittsburgh | Gene therapy for Gaucher disease using retroviral vectors |
US5872005A (en) * | 1994-11-03 | 1999-02-16 | Cell Genesys Inc. | Packaging cell lines for adeno-associated viral vectors |
US6071890A (en) | 1994-12-09 | 2000-06-06 | Genzyme Corporation | Organ-specific targeting of cationic amphiphile/DNA complexes for gene therapy |
US5840710A (en) * | 1994-12-09 | 1998-11-24 | Genzyme Corporation | Cationic amphiphiles containing ester or ether-linked lipophilic groups for intracellular delivery of therapeutic molecules |
US5767099A (en) | 1994-12-09 | 1998-06-16 | Genzyme Corporation | Cationic amphiphiles containing amino acid or dervatized amino acid groups for intracellular delivery of therapeutic molecules |
US5719131A (en) * | 1994-12-09 | 1998-02-17 | Genzyme Corporation | Cationic amphiphiles containing dialkylamine lipophilic groups for intracellular delivery of therapeutic molecules |
US5707618A (en) | 1995-03-24 | 1998-01-13 | Genzyme Corporation | Adenovirus vectors for gene therapy |
WO1997009441A2 (en) | 1995-09-08 | 1997-03-13 | Genzyme Corporation | Improved aav vectors for gene therapy |
US5962313A (en) * | 1996-01-18 | 1999-10-05 | Avigen, Inc. | Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme |
AU4352997A (en) | 1996-08-30 | 1998-03-19 | Genzyme Corporation | Inhibition of primary and/or secondary immune response to repeat adenoviral vector administration using cd40l specific antibodies |
AU4424497A (en) * | 1996-09-13 | 1998-04-02 | Transkaryotic Therapies, Inc. | Therapy for alpha-galactosidase a deficiency |
US5981275A (en) * | 1997-04-14 | 1999-11-09 | Genzyme Corporation | Transgene expression system for increased persistence |
GB9708526D0 (en) * | 1997-04-25 | 1997-06-18 | Royal Free Hosp School Med | Eukaryotic gene expression cassette and uses thereof |
CA2305768A1 (en) * | 1997-10-29 | 2000-02-24 | Genzyme Corporation | Compositions and methods for treating lysosomal storage disease |
US5879005A (en) * | 1998-02-26 | 1999-03-09 | Baca; Ron | Wargame playing system |
CN105246194B (zh) | 2011-11-15 | 2018-07-03 | 昂宝电子(上海)有限公司 | 用于各种操作模式中的恒流控制的led照明系统和方法 |
US9807841B2 (en) | 2012-07-12 | 2017-10-31 | Hubbell Incorporated | Circuit for expanding the dimming range of an LED lamp |
KR101690305B1 (ko) | 2014-10-16 | 2016-12-27 | 주식회사 솔루엠 | 컨버터 |
CN104409036B (zh) | 2014-12-16 | 2017-01-11 | 上海天马微电子有限公司 | 一种显示面板和显示装置 |
-
1998
- 1998-10-29 CA CA002305768A patent/CA2305768A1/en not_active Abandoned
- 1998-10-29 EP EP98956290A patent/EP1027069B1/en not_active Expired - Lifetime
- 1998-10-29 WO PCT/US1998/022886 patent/WO2000009153A1/en active Application Filing
- 1998-10-29 AT AT98956290T patent/ATE333893T1/de not_active IP Right Cessation
- 1998-10-29 ES ES98956290T patent/ES2268799T3/es not_active Expired - Lifetime
- 1998-10-29 JP JP2000564655A patent/JP2002522509A/ja not_active Withdrawn
- 1998-10-29 US US09/182,245 patent/US6066626A/en not_active Expired - Fee Related
- 1998-10-29 EP EP09175718A patent/EP2147681A1/en not_active Withdrawn
- 1998-10-29 AU AU12847/99A patent/AU734290B2/en not_active Ceased
- 1998-10-29 DE DE69835367T patent/DE69835367T2/de not_active Expired - Lifetime
- 1998-10-29 IL IL13557898A patent/IL135578A0/xx active IP Right Review Request
-
2000
- 2000-04-10 IL IL135578A patent/IL135578A/en not_active IP Right Cessation
-
2002
- 2002-09-13 US US10/244,700 patent/US20030087868A1/en not_active Abandoned
-
2006
- 2006-08-02 IL IL177228A patent/IL177228A0/en unknown
-
2008
- 2008-10-14 US US12/251,451 patent/US20100041151A1/en not_active Abandoned
-
2009
- 2009-09-28 JP JP2009222892A patent/JP2010031023A/ja not_active Withdrawn
Also Published As
Publication number | Publication date |
---|---|
WO2000009153A1 (en) | 2000-02-24 |
EP1027069B1 (en) | 2006-07-26 |
JP2010031023A (ja) | 2010-02-12 |
IL177228A0 (en) | 2006-12-10 |
US20100041151A1 (en) | 2010-02-18 |
EP1027069A1 (en) | 2000-08-16 |
AU734290B2 (en) | 2001-06-07 |
DE69835367D1 (de) | 2006-09-07 |
US6066626A (en) | 2000-05-23 |
IL135578A (en) | 2007-03-08 |
EP2147681A1 (en) | 2010-01-27 |
CA2305768A1 (en) | 2000-02-24 |
US20030087868A1 (en) | 2003-05-08 |
JP2002522509A (ja) | 2002-07-23 |
AU1284799A (en) | 2000-03-06 |
DE69835367T2 (de) | 2007-08-02 |
ATE333893T1 (de) | 2006-08-15 |
ES2268799T3 (es) | 2007-03-16 |
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KB | Patent renewed |