CA2292874A1 - Traitement de maladies hereditaires avec la gentamicine - Google Patents
Traitement de maladies hereditaires avec la gentamicine Download PDFInfo
- Publication number
- CA2292874A1 CA2292874A1 CA002292874A CA2292874A CA2292874A1 CA 2292874 A1 CA2292874 A1 CA 2292874A1 CA 002292874 A CA002292874 A CA 002292874A CA 2292874 A CA2292874 A CA 2292874A CA 2292874 A1 CA2292874 A1 CA 2292874A1
- Authority
- CA
- Canada
- Prior art keywords
- gentamicin
- muscularly
- aminoglycoside
- stop codon
- administered intra
- Prior art date
- Legal status (The legal status 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 status listed.)
- Abandoned
Links
- CEAZRRDELHUEMR-URQXQFDESA-N Gentamicin Chemical compound O1[C@H](C(C)NC)CC[C@@H](N)[C@H]1O[C@H]1[C@H](O)[C@@H](O[C@@H]2[C@@H]([C@@H](NC)[C@@](C)(O)CO2)O)[C@H](N)C[C@@H]1N CEAZRRDELHUEMR-URQXQFDESA-N 0.000 title claims abstract description 25
- 229930182566 Gentamicin Natural products 0.000 title claims abstract description 25
- 229960002518 gentamicin Drugs 0.000 title claims abstract description 25
- 208000024556 Mendelian disease Diseases 0.000 title abstract description 3
- 238000011282 treatment Methods 0.000 title description 7
- 208000026350 Inborn Genetic disease Diseases 0.000 title description 2
- 238000000034 method Methods 0.000 claims abstract description 12
- 108020004705 Codon Proteins 0.000 claims abstract description 10
- 239000002647 aminoglycoside antibiotic agent Substances 0.000 claims abstract description 10
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims abstract description 9
- 229940126574 aminoglycoside antibiotic Drugs 0.000 claims abstract description 8
- 241000699670 Mus sp. Species 0.000 claims description 13
- 201000010099 disease Diseases 0.000 claims description 13
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 13
- 108090000623 proteins and genes Proteins 0.000 claims description 12
- 102000004169 proteins and genes Human genes 0.000 claims description 10
- RDEIXVOBVLKYNT-VQBXQJRRSA-N (2r,3r,4r,5r)-2-[(1s,2s,3r,4s,6r)-4,6-diamino-3-[(2r,3r,6s)-3-amino-6-(1-aminoethyl)oxan-2-yl]oxy-2-hydroxycyclohexyl]oxy-5-methyl-4-(methylamino)oxane-3,5-diol;(2r,3r,4r,5r)-2-[(1s,2s,3r,4s,6r)-4,6-diamino-3-[(2r,3r,6s)-3-amino-6-(aminomethyl)oxan-2-yl]o Chemical compound OS(O)(=O)=O.O1C[C@@](O)(C)[C@H](NC)[C@@H](O)[C@H]1O[C@@H]1[C@@H](O)[C@H](O[C@@H]2[C@@H](CC[C@@H](CN)O2)N)[C@@H](N)C[C@H]1N.O1C[C@@](O)(C)[C@H](NC)[C@@H](O)[C@H]1O[C@@H]1[C@@H](O)[C@H](O[C@@H]2[C@@H](CC[C@H](O2)C(C)N)N)[C@@H](N)C[C@H]1N.O1[C@H](C(C)NC)CC[C@@H](N)[C@H]1O[C@H]1[C@H](O)[C@@H](O[C@@H]2[C@@H]([C@@H](NC)[C@@](C)(O)CO2)O)[C@H](N)C[C@@H]1N RDEIXVOBVLKYNT-VQBXQJRRSA-N 0.000 claims description 9
- 108020004707 nucleic acids Proteins 0.000 claims description 8
- 102000039446 nucleic acids Human genes 0.000 claims description 8
- 150000007523 nucleic acids Chemical class 0.000 claims description 8
- 108020004485 Nonsense Codon Proteins 0.000 claims description 7
- 229940126575 aminoglycoside Drugs 0.000 claims description 7
- 230000037396 body weight Effects 0.000 claims description 7
- 229940079593 drug Drugs 0.000 claims description 6
- 239000003814 drug Substances 0.000 claims description 6
- 230000035772 mutation Effects 0.000 abstract description 15
- 108010069091 Dystrophin Proteins 0.000 abstract description 12
- 102000001039 Dystrophin Human genes 0.000 abstract description 10
- 210000001087 myotubule Anatomy 0.000 abstract description 6
- 230000000694 effects Effects 0.000 abstract description 5
- 230000002028 premature Effects 0.000 abstract description 5
- 150000001413 amino acids Chemical class 0.000 abstract description 4
- 238000010171 animal model Methods 0.000 abstract description 2
- 208000028782 Hereditary disease Diseases 0.000 abstract 1
- 210000003205 muscle Anatomy 0.000 description 6
- 241000282412 Homo Species 0.000 description 4
- 108010079245 Cystic Fibrosis Transmembrane Conductance Regulator Proteins 0.000 description 3
- 102000012605 Cystic Fibrosis Transmembrane Conductance Regulator Human genes 0.000 description 3
- 201000003883 Cystic fibrosis Diseases 0.000 description 3
- 239000008280 blood Substances 0.000 description 3
- 210000004369 blood Anatomy 0.000 description 3
- 102100023419 Cystic fibrosis transmembrane conductance regulator Human genes 0.000 description 2
- 101000907783 Homo sapiens Cystic fibrosis transmembrane conductance regulator Proteins 0.000 description 2
- 241000699666 Mus <mouse, genus> Species 0.000 description 2
- 238000012217 deletion Methods 0.000 description 2
- 230000037430 deletion Effects 0.000 description 2
- 238000002474 experimental method Methods 0.000 description 2
- 238000003364 immunohistochemistry Methods 0.000 description 2
- 238000012744 immunostaining Methods 0.000 description 2
- 238000001727 in vivo Methods 0.000 description 2
- 238000011534 incubation Methods 0.000 description 2
- 238000007918 intramuscular administration Methods 0.000 description 2
- 239000012528 membrane Substances 0.000 description 2
- 210000002027 skeletal muscle Anatomy 0.000 description 2
- 238000010186 staining Methods 0.000 description 2
- 238000007920 subcutaneous administration Methods 0.000 description 2
- 238000013519 translation Methods 0.000 description 2
- GLDQAMYCGOIJDV-UHFFFAOYSA-M 2,3-dihydroxybenzoate Chemical compound OC1=CC=CC(C([O-])=O)=C1O GLDQAMYCGOIJDV-UHFFFAOYSA-M 0.000 description 1
- 101150029409 CFTR gene Proteins 0.000 description 1
- 108700024394 Exon Proteins 0.000 description 1
- 208000031220 Hemophilia Diseases 0.000 description 1
- 208000009292 Hemophilia A Diseases 0.000 description 1
- 206010029155 Nephropathy toxic Diseases 0.000 description 1
- 206010033109 Ototoxicity Diseases 0.000 description 1
- GLDQAMYCGOIJDV-UHFFFAOYSA-N Pyrocatechuic acid Natural products OC(=O)C1=CC=CC(O)=C1O GLDQAMYCGOIJDV-UHFFFAOYSA-N 0.000 description 1
- 210000001766 X chromosome Anatomy 0.000 description 1
- 230000009471 action Effects 0.000 description 1
- 125000000266 alpha-aminoacyl group Chemical group 0.000 description 1
- 230000008901 benefit Effects 0.000 description 1
- 230000003115 biocidal effect Effects 0.000 description 1
- 230000015572 biosynthetic process Effects 0.000 description 1
- 230000017531 blood circulation Effects 0.000 description 1
- 210000000424 bronchial epithelial cell Anatomy 0.000 description 1
- 210000004413 cardiac myocyte Anatomy 0.000 description 1
- 210000004027 cell Anatomy 0.000 description 1
- 210000000170 cell membrane Anatomy 0.000 description 1
- 239000003795 chemical substances by application Substances 0.000 description 1
- 150000001875 compounds Chemical class 0.000 description 1
- 230000001268 conjugating effect Effects 0.000 description 1
- 230000008602 contraction Effects 0.000 description 1
- 229940071221 dihydroxybenzoate Drugs 0.000 description 1
- 239000006185 dispersion Substances 0.000 description 1
- 239000000835 fiber Substances 0.000 description 1
- 238000002347 injection Methods 0.000 description 1
- 239000007924 injection Substances 0.000 description 1
- 238000010255 intramuscular injection Methods 0.000 description 1
- 238000001990 intravenous administration Methods 0.000 description 1
- 239000003446 ligand Substances 0.000 description 1
- 239000002502 liposome Substances 0.000 description 1
- 230000001404 mediated effect Effects 0.000 description 1
- 210000004165 myocardium Anatomy 0.000 description 1
- 230000007694 nephrotoxicity Effects 0.000 description 1
- 231100000417 nephrotoxicity Toxicity 0.000 description 1
- 231100000252 nontoxic Toxicity 0.000 description 1
- 230000003389 potentiating effect Effects 0.000 description 1
- 230000000750 progressive effect Effects 0.000 description 1
- 230000009467 reduction Effects 0.000 description 1
- 230000008439 repair process Effects 0.000 description 1
- 230000001629 suppression Effects 0.000 description 1
- 238000003786 synthesis reaction Methods 0.000 description 1
- 230000009885 systemic effect Effects 0.000 description 1
- 230000008685 targeting Effects 0.000 description 1
- 238000012360 testing method Methods 0.000 description 1
- 231100000331 toxic Toxicity 0.000 description 1
- 230000002588 toxic effect Effects 0.000 description 1
- 231100000419 toxicity Toxicity 0.000 description 1
- 230000001988 toxicity Effects 0.000 description 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
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7028—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages
- A61K31/7034—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin
- A61K31/7036—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin having at least one amino group directly attached to the carbocyclic ring, e.g. streptomycin, gentamycin, amikacin, validamycin, fortimicins
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Chemical & Material Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Physical Education & Sports Medicine (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Organic Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CA002292874A CA2292874A1 (fr) | 1998-12-22 | 1999-12-22 | Traitement de maladies hereditaires avec la gentamicine |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CA2,256,855 | 1998-12-22 | ||
| CA002256855A CA2256855A1 (fr) | 1998-12-22 | 1998-12-22 | Traitement de maladies hereditaires avec la gentamicine |
| CA002292874A CA2292874A1 (fr) | 1998-12-22 | 1999-12-22 | Traitement de maladies hereditaires avec la gentamicine |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA2292874A1 true CA2292874A1 (fr) | 2000-06-22 |
Family
ID=31888819
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA002292874A Abandoned CA2292874A1 (fr) | 1998-12-22 | 1999-12-22 | Traitement de maladies hereditaires avec la gentamicine |
Country Status (1)
| Country | Link |
|---|---|
| CA (1) | CA2292874A1 (fr) |
Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FR2900339A1 (fr) * | 2006-04-28 | 2007-11-02 | Univ Claude Bernard Lyon I Eta | Utilisation d'un compose qui induit une augmentation du taux de serotonine libre dans l'organisme, pour la preparation d'un medicament destine a traiter des degenerescences du muscle squelettique |
| FR2900338A1 (fr) * | 2006-04-28 | 2007-11-02 | Univ Claude Bernard Lyon I Eta | Utilisation d'un compose tricyclique, pour la preparation d'un medicament destine a traiter des degenerescences du muscle squelettique |
-
1999
- 1999-12-22 CA CA002292874A patent/CA2292874A1/fr not_active Abandoned
Cited By (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FR2900339A1 (fr) * | 2006-04-28 | 2007-11-02 | Univ Claude Bernard Lyon I Eta | Utilisation d'un compose qui induit une augmentation du taux de serotonine libre dans l'organisme, pour la preparation d'un medicament destine a traiter des degenerescences du muscle squelettique |
| FR2900338A1 (fr) * | 2006-04-28 | 2007-11-02 | Univ Claude Bernard Lyon I Eta | Utilisation d'un compose tricyclique, pour la preparation d'un medicament destine a traiter des degenerescences du muscle squelettique |
| WO2007125246A1 (fr) * | 2006-04-28 | 2007-11-08 | Universite Claude Bernard Lyon 1 | Utilisation d'un compose tricyclique, pour la preparation d'un medicament destine a traiter des degenerescences du muscle squelettique |
| WO2007125245A1 (fr) * | 2006-04-28 | 2007-11-08 | Universite Claude Bernard Lyon 1 | Utilisation d'un compose qui induit une augmentation du taux de serotonine libre dans l'organisme, pour la preparation d'un medicament destine a traiter des degenerescences du muscle squelettique |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request | ||
| FZDE | Dead |