ATE443764T1 - Allelspezifische rna-interferenz - Google Patents
Allelspezifische rna-interferenzInfo
- Publication number
- ATE443764T1 ATE443764T1 AT03783121T AT03783121T ATE443764T1 AT E443764 T1 ATE443764 T1 AT E443764T1 AT 03783121 T AT03783121 T AT 03783121T AT 03783121 T AT03783121 T AT 03783121T AT E443764 T1 ATE443764 T1 AT E443764T1
- Authority
- AT
- Austria
- Prior art keywords
- wild
- mutant
- gene
- type
- expression
- Prior art date
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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
-
- 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
-
- 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
- A61P25/16—Anti-Parkinson drugs
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
- C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
- C12Y115/01001—Superoxide dismutase (1.15.1.1)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
- C12N2310/111—Antisense spanning the whole gene, or a large part of it
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Veterinary Medicine (AREA)
- Molecular Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biochemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Animal Behavior & Ethology (AREA)
- General Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Psychology (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US42350702P | 2002-11-04 | 2002-11-04 | |
US48828303P | 2003-07-18 | 2003-07-18 | |
PCT/US2003/035009 WO2004042027A2 (en) | 2002-11-04 | 2003-11-04 | Allele-specific rna interference |
Publications (1)
Publication Number | Publication Date |
---|---|
ATE443764T1 true ATE443764T1 (de) | 2009-10-15 |
Family
ID=32314488
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
AT03783121T ATE443764T1 (de) | 2002-11-04 | 2003-11-04 | Allelspezifische rna-interferenz |
Country Status (9)
Country | Link |
---|---|
US (1) | US20040192629A1 (de) |
EP (1) | EP1567539B1 (de) |
JP (1) | JP2006505288A (de) |
AT (1) | ATE443764T1 (de) |
AU (1) | AU2003290586B2 (de) |
CA (1) | CA2504915A1 (de) |
DE (1) | DE60329416D1 (de) |
ES (1) | ES2334125T3 (de) |
WO (1) | WO2004042027A2 (de) |
Families Citing this family (95)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20030228320A1 (en) * | 2000-08-18 | 2003-12-11 | Ashdown Martin Leonard | Retroviral immunotherapy |
DE10163098B4 (de) * | 2001-10-12 | 2005-06-02 | Alnylam Europe Ag | Verfahren zur Hemmung der Replikation von Viren |
AUPS054702A0 (en) * | 2002-02-14 | 2002-03-07 | Immunaid Pty Ltd | Cancer therapy |
AU2003265978A1 (en) | 2002-05-03 | 2003-11-17 | Duke University | A method of regulating gene expression |
US20080176812A1 (en) * | 2002-08-05 | 2008-07-24 | Davidson Beverly L | Allele-specific silencing of disease genes |
US20040023390A1 (en) * | 2002-08-05 | 2004-02-05 | Davidson Beverly L. | SiRNA-mediated gene silencing with viral vectors |
US20080274989A1 (en) | 2002-08-05 | 2008-11-06 | University Of Iowa Research Foundation | Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof |
US20040241854A1 (en) | 2002-08-05 | 2004-12-02 | Davidson Beverly L. | siRNA-mediated gene silencing |
US20050042646A1 (en) * | 2002-08-05 | 2005-02-24 | Davidson Beverly L. | RNA interference suppresion of neurodegenerative diseases and methods of use thereof |
US20050255086A1 (en) * | 2002-08-05 | 2005-11-17 | Davidson Beverly L | Nucleic acid silencing of Huntington's Disease gene |
US20050106731A1 (en) * | 2002-08-05 | 2005-05-19 | Davidson Beverly L. | siRNA-mediated gene silencing with viral vectors |
WO2004014933A1 (en) | 2002-08-07 | 2004-02-19 | University Of Massachusetts | Compositions for rna interference and methods of use thereof |
US7892793B2 (en) | 2002-11-04 | 2011-02-22 | University Of Massachusetts | Allele-specific RNA interference |
US7951935B2 (en) | 2002-11-14 | 2011-05-31 | Dharmacon, Inc. | siRNA targeting v-myc myelocytomatosis viral oncogene homolog (MYC) |
JP2006507841A (ja) * | 2002-11-14 | 2006-03-09 | ダーマコン, インコーポレイテッド | 機能的siRNAおよび超機能的siRNA |
US9771586B2 (en) | 2002-11-14 | 2017-09-26 | Thermo Fisher Scientific Inc. | RNAi targeting ZNF205 |
WO2006006948A2 (en) | 2002-11-14 | 2006-01-19 | Dharmacon, Inc. | METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY |
US7781575B2 (en) | 2002-11-14 | 2010-08-24 | Dharmacon, Inc. | siRNA targeting tumor protein 53 (p53) |
US20090227780A1 (en) * | 2002-11-14 | 2009-09-10 | Dharmacon, Inc. | siRNA targeting connexin 43 |
US7691998B2 (en) * | 2002-11-14 | 2010-04-06 | Dharmacon, Inc. | siRNA targeting nucleoporin 62kDa (Nup62) |
US20100113307A1 (en) * | 2002-11-14 | 2010-05-06 | Dharmacon, Inc. | siRNA targeting vascular endothelial growth factor (VEGF) |
US9839649B2 (en) | 2002-11-14 | 2017-12-12 | Thermo Fisher Scientific Inc. | Methods and compositions for selecting siRNA of improved functionality |
US9719092B2 (en) | 2002-11-14 | 2017-08-01 | Thermo Fisher Scientific Inc. | RNAi targeting CNTD2 |
US7612196B2 (en) | 2002-11-14 | 2009-11-03 | Dharmacon, Inc. | siRNA targeting cyclin-dependent kinase inhibitor 1B (p27, Kip1) (CDKN1B) |
US9228186B2 (en) | 2002-11-14 | 2016-01-05 | Thermo Fisher Scientific Inc. | Methods and compositions for selecting siRNA of improved functionality |
US10011836B2 (en) | 2002-11-14 | 2018-07-03 | Thermo Fisher Scientific Inc. | Methods and compositions for selecting siRNA of improved functionality |
US7592442B2 (en) * | 2002-11-14 | 2009-09-22 | Dharmacon, Inc. | siRNA targeting ribonucleotide reductase M2 polypeptide (RRM2 or RNR-R2) |
US7635770B2 (en) * | 2002-11-14 | 2009-12-22 | Dharmacon, Inc. | siRNA targeting protein kinase N-3 (PKN-3) |
US8198427B1 (en) | 2002-11-14 | 2012-06-12 | Dharmacon, Inc. | SiRNA targeting catenin, beta-1 (CTNNB1) |
US7977471B2 (en) * | 2002-11-14 | 2011-07-12 | Dharmacon, Inc. | siRNA targeting TNFα |
US7619081B2 (en) * | 2002-11-14 | 2009-11-17 | Dharmacon, Inc. | siRNA targeting coatomer protein complex, subunit beta 2 (COPB2) |
US9879266B2 (en) | 2002-11-14 | 2018-01-30 | Thermo Fisher Scientific Inc. | Methods and compositions for selecting siRNA of improved functionality |
US9719094B2 (en) | 2002-11-14 | 2017-08-01 | Thermo Fisher Scientific Inc. | RNAi targeting SEC61G |
US20080268457A1 (en) * | 2002-11-14 | 2008-10-30 | Dharmacon, Inc. | siRNA targeting forkhead box P3 (FOXP3) |
WO2004046324A2 (en) * | 2002-11-15 | 2004-06-03 | University Of Massachusetts | Allele-targeted rna interference |
WO2004047764A2 (en) * | 2002-11-22 | 2004-06-10 | University Of Massachusetts | Modulation of hiv replication by rna interference |
US20040181821A1 (en) * | 2003-03-14 | 2004-09-16 | Jiadong Zhou | siRNA research tool kit |
US8309704B2 (en) * | 2003-06-02 | 2012-11-13 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNAi |
US7750144B2 (en) * | 2003-06-02 | 2010-07-06 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
DK1633767T3 (en) * | 2003-06-02 | 2019-03-25 | Univ Massachusetts | METHODS AND COMPOSITIONS FOR MANAGING THE EFFECT OF RNA SILENCING |
US20050130184A1 (en) * | 2003-07-18 | 2005-06-16 | University Of Massachusetts | Enhanced promoters for synthesis of small hairpin RNA |
US8680063B2 (en) * | 2003-09-12 | 2014-03-25 | University Of Massachusetts | RNA interference for the treatment of gain-of-function disorders |
DK2821085T3 (da) * | 2003-09-12 | 2020-08-03 | Univ Massachusetts | Rna-interferens til behandling af "gain-of-function"-forstyrrelser |
PL1692516T3 (pl) | 2003-10-24 | 2011-05-31 | Immunaid Pty Ltd | Sposób terapii |
US20060134787A1 (en) | 2004-12-22 | 2006-06-22 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended siRNA |
WO2005097817A2 (en) | 2004-04-05 | 2005-10-20 | Alnylam Pharmaceuticals, Inc. | Process and reagents for oligonucleotide synthesis and purification |
WO2005096781A2 (en) * | 2004-04-06 | 2005-10-20 | University Of Massachusetts | Methods and compositions for treating gain-of-function disorders using rna interference |
JP4584986B2 (ja) * | 2004-04-27 | 2010-11-24 | アルニラム ファーマスーティカルズ インコーポレイテッド | 2−アリールプロピル部分を含む1本鎖及び2本鎖オリゴヌクレオチド |
EP1750776A2 (de) | 2004-04-30 | 2007-02-14 | Alnylam Pharmaceuticals Inc. | Oligonukleotide mit c5-modifiziertem pyrimidin |
WO2006088490A2 (en) * | 2004-06-30 | 2006-08-24 | Alnylam Pharmaceuticals, Inc. | Oligonucleotides comprising a non-phosphate backbone linkage |
WO2006093526A2 (en) | 2004-07-21 | 2006-09-08 | Alnylam Pharmaceuticals, Inc. | Oligonucleotides comprising a modified or non-natural nucleobase |
AU2005330637B2 (en) | 2004-08-04 | 2012-09-20 | Alnylam Pharmaceuticals, Inc. | Oligonucleotides comprising a ligand tethered to a modified or non-natural nucleobase |
WO2006060454A2 (en) * | 2004-12-02 | 2006-06-08 | B-Bridge International, Inc. | Methods of designing small interfering rnas, antisense polynucleotides, and other hybridizing polynucleotides |
WO2006066203A2 (en) * | 2004-12-16 | 2006-06-22 | Alsgen, Llc | Small interfering rna (sirna) molecules for modulating superoxide dismutase (sod) |
JP4853892B2 (ja) * | 2005-04-13 | 2012-01-11 | 独立行政法人国立精神・神経医療研究センター | 変異対立遺伝子に対する特異的なRNAiの評価方法 |
CA2606002A1 (en) * | 2005-04-28 | 2006-11-02 | Benitec Limited | Multiple-rnai expression cassettes for simultaneous delivery of rnai agents related to heterozygotic expression patterns |
US20070105803A1 (en) | 2005-08-18 | 2007-05-10 | Muthiah Manoharan | Methods and compositions for treating neurological disease |
WO2007087451A2 (en) * | 2006-01-25 | 2007-08-02 | University Of Massachusetts | Compositions and methods for enhancing discriminatory rna interference |
GB0605337D0 (en) * | 2006-03-17 | 2006-04-26 | Genomica Sau | Treatment of CNS conditions |
WO2008143774A2 (en) * | 2007-05-01 | 2008-11-27 | University Of Massachusetts | Methods and compositions for locating snp heterozygosity for allele specific diagnosis and therapy |
CN104975020B (zh) | 2008-02-11 | 2020-01-17 | 菲奥医药公司 | 经修饰的RNAi多核苷酸及其用途 |
JP2011521649A (ja) * | 2008-05-30 | 2011-07-28 | イェール ユニバーシティー | 遺伝子発現を改変するための標的化オリゴヌクレオチド組成物 |
WO2009150156A1 (en) * | 2008-06-13 | 2009-12-17 | Riboxx Gmbh | Method for enzymatic synthesis of chemically modified rna |
WO2010008582A2 (en) | 2008-07-18 | 2010-01-21 | Rxi Pharmaceuticals Corporation | Phagocytic cell drug delivery system |
EP2949752B1 (de) | 2008-09-22 | 2017-12-20 | RXi Pharmaceuticals Corporation | Selbstfreisetzende rnai-verbindungen von reduzierter grösse |
US8802646B2 (en) | 2008-10-08 | 2014-08-12 | Trustees Of Dartmouth College | Method for selectively inhibiting the activity of ACAT1 in the treatment of alzheimer's disease |
US20100183558A1 (en) | 2008-10-17 | 2010-07-22 | Zhennan Lai | Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules |
US9074211B2 (en) | 2008-11-19 | 2015-07-07 | Rxi Pharmaceuticals Corporation | Inhibition of MAP4K4 through RNAI |
WO2010078536A1 (en) | 2009-01-05 | 2010-07-08 | Rxi Pharmaceuticals Corporation | Inhibition of pcsk9 through rnai |
WO2010090762A1 (en) | 2009-02-04 | 2010-08-12 | Rxi Pharmaceuticals Corporation | Rna duplexes with single stranded phosphorothioate nucleotide regions for additional functionality |
AU2010251880B2 (en) | 2009-05-27 | 2015-07-30 | Biotempus Pty Ltd | Methods of treating diseases |
BR112012007137A2 (pt) | 2009-09-30 | 2015-09-15 | Harvard College | metodos para modulacao da autofagia por meio da modulacao de produtos genicos inibidores da autofagia |
US9799416B2 (en) * | 2009-11-06 | 2017-10-24 | Terrapower, Llc | Methods and systems for migrating fuel assemblies in a nuclear fission reactor |
WO2011119887A1 (en) | 2010-03-24 | 2011-09-29 | Rxi Pharmaceuticals Corporation | Rna interference in dermal and fibrotic indications |
RU2615143C2 (ru) | 2010-03-24 | 2017-04-04 | Адвирна | Самодоставляющие PHKi соединения уменьшенного размера |
EP3578183B1 (de) | 2010-03-24 | 2021-09-08 | Phio Pharmaceuticals Corp. | Rna-interferenz bei augenerkrankungen |
EP2623600A4 (de) * | 2010-09-30 | 2014-11-26 | Lsip Llc | Hemmer der expression dominant mutierter gene |
GB201105137D0 (en) * | 2011-03-28 | 2011-05-11 | Isis Innovation | Therapeutic molecules for use in the suppression of Parkinson's disease |
JP2015529222A (ja) * | 2012-09-06 | 2015-10-05 | トラスティーズ・オブ・ダートマウス・カレッジ | アルツハイマー病の処置におけるacat1の活性を選択的に阻害するための方法 |
WO2015153800A2 (en) | 2014-04-01 | 2015-10-08 | Isis Pharmaceuticals, Inc. | Compositions for modulating sod-1 expression |
US10900039B2 (en) | 2014-09-05 | 2021-01-26 | Phio Pharmaceuticals Corp. | Methods for treating aging and skin disorders using nucleic acids targeting Tyr or MMP1 |
US10808247B2 (en) | 2015-07-06 | 2020-10-20 | Phio Pharmaceuticals Corp. | Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach |
JP6983752B2 (ja) | 2015-07-06 | 2021-12-17 | フィオ ファーマシューティカルズ コーポレーションPhio Pharmaceuticals Corp. | スーパーオキシドディスムターゼ1(sod1)を標的とする核酸分子 |
WO2017007994A1 (en) | 2015-07-08 | 2017-01-12 | American Gene Technologies International Inc. | Hiv pre-immunization and immunotherapy |
WO2017070151A1 (en) | 2015-10-19 | 2017-04-27 | Rxi Pharmaceuticals Corporation | Reduced size self-delivering nucleic acid compounds targeting long non-coding rna |
US10137144B2 (en) | 2016-01-15 | 2018-11-27 | American Gene Technologies International Inc. | Methods and compositions for the activation of gamma-delta T-cells |
AU2017207906B2 (en) | 2016-01-15 | 2021-03-11 | American Gene Technologies International Inc. | Methods and compositions for the activation of gamma-delta T-cells |
WO2017139065A1 (en) | 2016-02-08 | 2017-08-17 | American Gene Technologies International Inc. | Hiv vaccination and immunotherapy |
US10767183B2 (en) | 2016-03-09 | 2020-09-08 | American Gene Technologies International Inc. | Combination vectors and methods for treating cancer |
US11976292B2 (en) | 2016-06-08 | 2024-05-07 | American Gene Technologies International Inc. | Non-integrating viral delivery system and methods related thereto |
CA3028982A1 (en) | 2016-07-08 | 2018-01-11 | American Gene Technologies International Inc. | Hiv pre-immunization and immunotherapy |
JP7176756B2 (ja) | 2016-07-21 | 2022-11-22 | アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド | パーキンソン病を処置するためのウイルスベクター |
CA3057142A1 (en) | 2017-04-03 | 2018-10-11 | American Gene Technologies International Inc. | Compositions and methods for treating phenylketonuria |
US11352646B2 (en) | 2018-11-05 | 2022-06-07 | American Gene Technologies International Inc. | Vector system for expressing regulatory RNA |
IL304880A (en) | 2021-02-12 | 2023-10-01 | Alnylam Pharmaceuticals Inc | Superoxide dismutase 1 (SOD1) IRNA compositions and methods of using them to treat or prevent superoxide dismutase 1- (SOD1-) associated neurodegenerative diseases |
Family Cites Families (16)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US6358932B1 (en) * | 1994-05-31 | 2002-03-19 | Isis Pharmaceticals, Inc. | Antisense oligonucleotide inhibition of raf gene expression |
CA2287508A1 (en) * | 1997-04-21 | 1998-10-29 | The University Of Florida | Materials and methods for ribozyme treatment of retinal diseases |
US7829693B2 (en) * | 1999-11-24 | 2010-11-09 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of a target gene |
US20030190635A1 (en) * | 2002-02-20 | 2003-10-09 | Mcswiggen James A. | RNA interference mediated treatment of Alzheimer's disease using short interfering RNA |
US20040219671A1 (en) * | 2002-02-20 | 2004-11-04 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of parkinson disease using short interfering nucleic acid (siNA) |
US20050137155A1 (en) * | 2001-05-18 | 2005-06-23 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of Parkinson disease using short interfering nucleic acid (siNA) |
DE10163098B4 (de) * | 2001-10-12 | 2005-06-02 | Alnylam Europe Ag | Verfahren zur Hemmung der Replikation von Viren |
US20030180756A1 (en) * | 2002-03-21 | 2003-09-25 | Yang Shi | Compositions and methods for suppressing eukaryotic gene expression |
US20050042646A1 (en) * | 2002-08-05 | 2005-02-24 | Davidson Beverly L. | RNA interference suppresion of neurodegenerative diseases and methods of use thereof |
US20040023390A1 (en) * | 2002-08-05 | 2004-02-05 | Davidson Beverly L. | SiRNA-mediated gene silencing with viral vectors |
US20040241854A1 (en) * | 2002-08-05 | 2004-12-02 | Davidson Beverly L. | siRNA-mediated gene silencing |
US20050106731A1 (en) * | 2002-08-05 | 2005-05-19 | Davidson Beverly L. | siRNA-mediated gene silencing with viral vectors |
WO2004046324A2 (en) * | 2002-11-15 | 2004-06-03 | University Of Massachusetts | Allele-targeted rna interference |
US20050130184A1 (en) * | 2003-07-18 | 2005-06-16 | University Of Massachusetts | Enhanced promoters for synthesis of small hairpin RNA |
US20050130919A1 (en) * | 2003-07-18 | 2005-06-16 | University Of Massachusetts | Regulatable promoters for synthesis of small hairpin RNA |
WO2007087451A2 (en) * | 2006-01-25 | 2007-08-02 | University Of Massachusetts | Compositions and methods for enhancing discriminatory rna interference |
-
2003
- 2003-11-04 AT AT03783121T patent/ATE443764T1/de not_active IP Right Cessation
- 2003-11-04 US US10/700,816 patent/US20040192629A1/en not_active Abandoned
- 2003-11-04 CA CA002504915A patent/CA2504915A1/en not_active Abandoned
- 2003-11-04 JP JP2005502260A patent/JP2006505288A/ja active Pending
- 2003-11-04 AU AU2003290586A patent/AU2003290586B2/en not_active Ceased
- 2003-11-04 ES ES03783121T patent/ES2334125T3/es not_active Expired - Lifetime
- 2003-11-04 WO PCT/US2003/035009 patent/WO2004042027A2/en active Application Filing
- 2003-11-04 EP EP03783121A patent/EP1567539B1/de not_active Expired - Lifetime
- 2003-11-04 DE DE60329416T patent/DE60329416D1/de not_active Expired - Lifetime
Also Published As
Publication number | Publication date |
---|---|
JP2006505288A (ja) | 2006-02-16 |
AU2003290586A1 (en) | 2004-06-07 |
US20040192629A1 (en) | 2004-09-30 |
CA2504915A1 (en) | 2004-05-21 |
EP1567539B1 (de) | 2009-09-23 |
EP1567539A2 (de) | 2005-08-31 |
EP1567539A4 (de) | 2006-07-26 |
AU2003290586B2 (en) | 2009-07-02 |
DE60329416D1 (de) | 2009-11-05 |
WO2004042027A2 (en) | 2004-05-21 |
WO2004042027A3 (en) | 2004-07-15 |
ES2334125T3 (es) | 2010-03-05 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
ATE443764T1 (de) | Allelspezifische rna-interferenz | |
WO2007044362A3 (en) | Allele-specific rna interference | |
Yu et al. | Triplet repeat–derived siRNAs enhance RNA–mediated toxicity in a Drosophila model for myotonic dystrophy | |
Fujiwara et al. | Developmental hyperbilirubinemia and CNS toxicity in mice humanized with the UDP glucuronosyltransferase 1 (UGT1) locus | |
Powell et al. | R-loop formation at Snord116 mediates topotecan inhibition of Ube3a-antisense and allele-specific chromatin decondensation | |
Gu et al. | tRNA modifications regulate translation during cellular stress | |
Hagerman et al. | Fragile X–associated tremor/ataxia syndrome | |
Wang et al. | MiR-122 inhibits cell proliferation and tumorigenesis of breast cancer by targeting IGF1R | |
Zinad et al. | Natural antisense transcripts at the interface between host genome and mobile genetic elements | |
Gasparini et al. | Protective role of miR-155 in breast cancer through RAD51 targeting impairs homologous recombination after irradiation | |
Li | Chromatin remodeling by the small RNA machinery in mammalian cells | |
Yan et al. | MicroRNA-34a inhibits the proliferation and metastasis of osteosarcoma cells both in vitro and in vivo | |
Tazawa et al. | Tumor-suppressive miR-34a induces senescence-like growth arrest through modulation of the E2F pathway in human colon cancer cells | |
MX2007010608A (es) | Inhibicion mediada por i-arn de proteina i relacionada con frizzled para tratamiento de glaucoma. | |
Pu et al. | MiR-1254 suppresses HO-1 expression through seed region-dependent silencing and non-seed interaction with TFAP2A transcript to attenuate NSCLC growth | |
Böhm et al. | Extracellular localization of catalase is associated with the transformed state of malignant cells | |
WO2005096781A3 (en) | Methods and compositions for treating gain-of-function disorders using rna interference | |
Wang et al. | Therapeutic Gene Silencing Delivered by a Chemically Modified Small Interfering RNA against Mutant SOD1 Slows Amyotrophic Lateral Sclerosis Progression*♦ | |
Haupenthal et al. | RNAse A‐like enzymes in serum inhibit the anti‐neoplastic activity of siRNA targeting polo‐like kinase 1 | |
Lyu et al. | Reciprocal regulation of autism-related genes MeCP2 and PTEN via microRNAs | |
WO2005076999A3 (en) | Methods and compositions for combination rnai therapeutics | |
Haque et al. | ZFR coordinates crosstalk between RNA decay and transcription in innate immunity | |
Towler et al. | The 3'-5'exoribonuclease Dis3 regulates the expression of specific microRNAs in Drosophila wing imaginal discs | |
WO2005112636A3 (en) | COMPOSITIONS AND METHODS FOR siRNA INHIBITION OF PRIMATE POLYOMAVIRUS GENES | |
Hanzlikova et al. | Pathogenic ARH3 mutations result in ADP-ribose chromatin scars during DNA strand break repair |
Legal Events
Date | Code | Title | Description |
---|---|---|---|
RER | Ceased as to paragraph 5 lit. 3 law introducing patent treaties |