HUE038583T2 - Kompozíciók familiaris dysautnomia kezelésére MRNS splicing megváltoztatásával - Google Patents
Kompozíciók familiaris dysautnomia kezelésére MRNS splicing megváltoztatásávalInfo
- Publication number
- HUE038583T2 HUE038583T2 HUE04794054A HUE04794054A HUE038583T2 HU E038583 T2 HUE038583 T2 HU E038583T2 HU E04794054 A HUE04794054 A HU E04794054A HU E04794054 A HUE04794054 A HU E04794054A HU E038583 T2 HUE038583 T2 HU E038583T2
- Authority
- HU
- Hungary
- Prior art keywords
- compositions
- mrna splicing
- familial dysautonomia
- treating familial
- altering mrna
- Prior art date
Links
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
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
- A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
- A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
- A61K31/52—Purines, e.g. adenine
-
- 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
- A61K31/33—Heterocyclic compounds
- A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
- A61K31/35—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom
- A61K31/352—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom condensed with carbocyclic rings, e.g. methantheline
- A61K31/353—3,4-Dihydrobenzopyrans, e.g. chroman, catechin
-
- 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
- A61K31/33—Heterocyclic compounds
- A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
- A61K31/35—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom
- A61K31/352—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom condensed with carbocyclic rings, e.g. methantheline
- A61K31/353—3,4-Dihydrobenzopyrans, e.g. chroman, catechin
- A61K31/355—Tocopherols, e.g. vitamin E
-
- 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
- A61P3/04—Anorexiants; Antiobesity agents
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
- C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/136—Screening for pharmacological compounds
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/156—Polymorphic or mutational markers
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/158—Expression markers
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Genetics & Genomics (AREA)
- Analytical Chemistry (AREA)
- Zoology (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurosurgery (AREA)
- Biomedical Technology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Pathology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Biotechnology (AREA)
- Immunology (AREA)
- Hospice & Palliative Care (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Child & Adolescent Psychology (AREA)
- Psychiatry (AREA)
- Obesity (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US50846503P | 2003-10-03 | 2003-10-03 | |
US53628704P | 2004-01-13 | 2004-01-13 |
Publications (1)
Publication Number | Publication Date |
---|---|
HUE038583T2 true HUE038583T2 (hu) | 2018-10-29 |
Family
ID=34426055
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HUE04794054A HUE038583T2 (hu) | 2003-10-03 | 2004-10-01 | Kompozíciók familiaris dysautnomia kezelésére MRNS splicing megváltoztatásával |
Country Status (8)
Country | Link |
---|---|
US (4) | US7737110B2 (hu) |
EP (1) | EP1673439B1 (hu) |
AU (1) | AU2004278773B2 (hu) |
CA (1) | CA2540681C (hu) |
HU (1) | HUE038583T2 (hu) |
IL (2) | IL174757A (hu) |
PL (1) | PL1673439T3 (hu) |
WO (1) | WO2005033290A2 (hu) |
Families Citing this family (5)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20100234402A1 (en) * | 2007-05-31 | 2010-09-16 | Gideon Dreyfuss | Methods and compositions for treating spinal muscular atrophy |
WO2013033407A2 (en) | 2011-08-30 | 2013-03-07 | The Regents Of The University Of California | Identification of small molecules that enhance therapeutic exon skipping |
JPWO2015005491A1 (ja) * | 2013-07-12 | 2017-03-02 | 国立大学法人京都大学 | 疾病の発症又は進行の一因となる異常スプライシングを抑制できる物質のスクリーニング方法 |
TW202204345A (zh) * | 2015-01-16 | 2022-02-01 | 美商通用醫院公司 | 改善mRNA剪接之化合物 |
US11987795B2 (en) | 2020-11-24 | 2024-05-21 | The Broad Institute, Inc. | Methods of modulating SLC7A11 pre-mRNA transcripts for diseases and conditions associated with expression of SLC7A11 |
Family Cites Families (6)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
DD148889A3 (de) | 1974-08-19 | 1981-06-17 | Forsch Die Gaerungsindustrie E | Verfahren zur herstellung eiweissreicher hefen |
US6238861B1 (en) | 1990-06-29 | 2001-05-29 | The Regents Of The University Of Michigan | Neurofibromatosis gene |
US5227292A (en) | 1990-07-12 | 1993-07-13 | University Of Utah | Neurofibromatosis type 1 gene |
US5605799A (en) | 1990-07-12 | 1997-02-25 | University Of Utah Research Foundation | Somatic mutations in neurofibromatosis type 1 gene in human tumors |
EP1373568B1 (en) * | 2001-01-06 | 2010-09-01 | The General Hospital Corporation | Gene for identifying individuals with familial dysautonomia |
CZ294535B6 (cs) * | 2001-08-02 | 2005-01-12 | Ústav Experimentální Botaniky Avčr | Heterocyklické sloučeniny na bázi N6-substituovaného adeninu, způsoby jejich přípravy, jejich použití pro přípravu léčiv, kosmetických přípravků a růstových regulátorů, farmaceutické přípravky, kosmetické přípravky a růstové regulátory tyto sloučeniny obsahující |
-
2004
- 2004-10-01 EP EP04794054.9A patent/EP1673439B1/en not_active Not-in-force
- 2004-10-01 US US10/956,601 patent/US7737110B2/en not_active Expired - Fee Related
- 2004-10-01 WO PCT/US2004/032554 patent/WO2005033290A2/en active Application Filing
- 2004-10-01 PL PL04794054T patent/PL1673439T3/pl unknown
- 2004-10-01 CA CA 2540681 patent/CA2540681C/en not_active Expired - Fee Related
- 2004-10-01 HU HUE04794054A patent/HUE038583T2/hu unknown
- 2004-10-01 AU AU2004278773A patent/AU2004278773B2/en not_active Ceased
-
2006
- 2006-03-30 IL IL174757A patent/IL174757A/en active IP Right Grant
-
2010
- 2010-05-04 US US12/773,137 patent/US20110136836A1/en not_active Abandoned
-
2012
- 2012-01-16 IL IL217561A patent/IL217561A/en active IP Right Grant
- 2012-08-31 US US13/601,034 patent/US8729025B2/en not_active Expired - Fee Related
-
2014
- 2014-05-19 US US14/281,065 patent/US9265766B2/en not_active Expired - Fee Related
Also Published As
Publication number | Publication date |
---|---|
CA2540681A1 (en) | 2005-04-14 |
US20120329816A1 (en) | 2012-12-27 |
CA2540681C (en) | 2014-08-12 |
US20060014763A1 (en) | 2006-01-19 |
AU2004278773B2 (en) | 2011-05-19 |
US8729025B2 (en) | 2014-05-20 |
EP1673439A2 (en) | 2006-06-28 |
IL174757A (en) | 2012-02-29 |
PL1673439T3 (pl) | 2018-05-30 |
WO2005033290A3 (en) | 2006-09-08 |
US20110136836A1 (en) | 2011-06-09 |
EP1673439A4 (en) | 2011-06-22 |
EP1673439B1 (en) | 2017-12-06 |
AU2004278773A1 (en) | 2005-04-14 |
US9265766B2 (en) | 2016-02-23 |
IL174757A0 (en) | 2006-08-20 |
US7737110B2 (en) | 2010-06-15 |
WO2005033290A2 (en) | 2005-04-14 |
IL217561A0 (en) | 2012-02-29 |
IL217561A (en) | 2014-05-28 |
US20150111902A1 (en) | 2015-04-23 |
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