HUE038583T2 - Kompozíciók familiaris dysautnomia kezelésére MRNS splicing megváltoztatásával - Google Patents

Kompozíciók familiaris dysautnomia kezelésére MRNS splicing megváltoztatásával

Info

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
Application number
HUE04794054A
Other languages
English (en)
Inventor
Susan Slaugenhaupt
James Gusella
Original Assignee
Massachusetts Gen Hospital
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 Massachusetts Gen Hospital filed Critical Massachusetts Gen Hospital
Publication of HUE038583T2 publication Critical patent/HUE038583T2/hu

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic 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
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A61K31/52Purines, e.g. adenine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/335Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/35Heterocyclic 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
    • A61K31/352Heterocyclic 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 
    • A61K31/3533,4-Dihydrobenzopyrans, e.g. chroman, catechin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/335Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/35Heterocyclic 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
    • A61K31/352Heterocyclic 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 
    • A61K31/3533,4-Dihydrobenzopyrans, e.g. chroman, catechin
    • A61K31/355Tocopherols, e.g. vitamin E
    • 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
    • A61P3/04Anorexiants; Antiobesity agents
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING 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
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING 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
    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/136Screening for pharmacological compounds
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING 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
    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/156Polymorphic or mutational markers
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING 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
    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/158Expression 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)
HUE04794054A 2003-10-03 2004-10-01 Kompozíciók familiaris dysautnomia kezelésére MRNS splicing megváltoztatásával HUE038583T2 (hu)

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)

* Cited by examiner, † Cited by third party
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)

* Cited by examiner, † Cited by third party
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

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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