JP2018538261A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2018538261A5 JP2018538261A5 JP2018524411A JP2018524411A JP2018538261A5 JP 2018538261 A5 JP2018538261 A5 JP 2018538261A5 JP 2018524411 A JP2018524411 A JP 2018524411A JP 2018524411 A JP2018524411 A JP 2018524411A JP 2018538261 A5 JP2018538261 A5 JP 2018538261A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- item
- subject
- seq
- recombinant aav
- 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.)
- Granted
Links
- 239000000203 mixture Substances 0.000 claims description 116
- 239000003963 antioxidant agent Substances 0.000 claims description 63
- 235000006708 antioxidants Nutrition 0.000 claims description 63
- 230000003078 antioxidant effect Effects 0.000 claims description 56
- 239000013607 AAV vector Substances 0.000 claims description 38
- 150000007523 nucleic acids Chemical class 0.000 claims description 30
- 201000006938 muscular dystrophy Diseases 0.000 claims description 27
- 239000002773 nucleotide Substances 0.000 claims description 24
- 125000003729 nucleotide group Chemical group 0.000 claims description 24
- 208000021642 Muscular disease Diseases 0.000 claims description 21
- 201000009623 Myopathy Diseases 0.000 claims description 21
- 210000003205 muscle Anatomy 0.000 claims description 20
- GVJHHUAWPYXKBD-UHFFFAOYSA-N (±)-α-Tocopherol Chemical compound OC1=C(C)C(C)=C2OC(CCCC(C)CCCC(C)CCCC(C)C)(C)CCC2=C1C GVJHHUAWPYXKBD-UHFFFAOYSA-N 0.000 claims description 16
- ACTIUHUUMQJHFO-UHFFFAOYSA-N Coenzym Q10 Natural products COC1=C(OC)C(=O)C(CC=C(C)CCC=C(C)CCC=C(C)CCC=C(C)CCC=C(C)CCC=C(C)CCC=C(C)CCC=C(C)CCC=C(C)CCC=C(C)C)=C(C)C1=O ACTIUHUUMQJHFO-UHFFFAOYSA-N 0.000 claims description 16
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 16
- 235000017471 coenzyme Q10 Nutrition 0.000 claims description 16
- 229940110767 coenzyme Q10 Drugs 0.000 claims description 16
- ACTIUHUUMQJHFO-UPTCCGCDSA-N coenzyme Q10 Chemical compound COC1=C(OC)C(=O)C(C\C=C(/C)CC\C=C(/C)CC\C=C(/C)CC\C=C(/C)CC\C=C(/C)CC\C=C(/C)CC\C=C(/C)CC\C=C(/C)CC\C=C(/C)CCC=C(C)C)=C(C)C1=O ACTIUHUUMQJHFO-UPTCCGCDSA-N 0.000 claims description 16
- AGBQKNBQESQNJD-UHFFFAOYSA-M lipoate Chemical compound [O-]C(=O)CCCCC1CCSS1 AGBQKNBQESQNJD-UHFFFAOYSA-M 0.000 claims description 16
- 235000019136 lipoic acid Nutrition 0.000 claims description 16
- 229960002663 thioctic acid Drugs 0.000 claims description 16
- 229940088594 vitamin Drugs 0.000 claims description 16
- 229930003231 vitamin Natural products 0.000 claims description 16
- 239000011782 vitamin Substances 0.000 claims description 16
- 235000013343 vitamin Nutrition 0.000 claims description 16
- 108020004707 nucleic acids Proteins 0.000 claims description 13
- 102000039446 nucleic acids Human genes 0.000 claims description 13
- 108090000623 proteins and genes Proteins 0.000 claims description 13
- 102000004169 proteins and genes Human genes 0.000 claims description 13
- 102100036524 Anoctamin-5 Human genes 0.000 claims description 12
- 101000928364 Homo sapiens Anoctamin-5 Proteins 0.000 claims description 12
- 230000000694 effects Effects 0.000 claims description 12
- 206010028289 Muscle atrophy Diseases 0.000 claims description 11
- 230000001684 chronic effect Effects 0.000 claims description 11
- 239000012634 fragment Substances 0.000 claims description 11
- 201000000585 muscular atrophy Diseases 0.000 claims description 11
- 108091033319 polynucleotide Proteins 0.000 claims description 10
- 102000040430 polynucleotide Human genes 0.000 claims description 10
- 239000002157 polynucleotide Substances 0.000 claims description 10
- 150000003722 vitamin derivatives Chemical class 0.000 claims description 10
- 101150008952 ANO5 gene Proteins 0.000 claims description 8
- 101100269860 Homo sapiens ANO5 gene Proteins 0.000 claims description 8
- 229930003427 Vitamin E Natural products 0.000 claims description 8
- 235000021466 carotenoid Nutrition 0.000 claims description 8
- 150000001747 carotenoids Chemical class 0.000 claims description 8
- 229930003935 flavonoid Natural products 0.000 claims description 8
- 235000017173 flavonoids Nutrition 0.000 claims description 8
- 150000002215 flavonoids Chemical class 0.000 claims description 8
- WIGCFUFOHFEKBI-UHFFFAOYSA-N gamma-tocopherol Natural products CC(C)CCCC(C)CCCC(C)CCCC1CCC2C(C)C(O)C(C)C(C)C2O1 WIGCFUFOHFEKBI-UHFFFAOYSA-N 0.000 claims description 8
- 229910052500 inorganic mineral Inorganic materials 0.000 claims description 8
- 239000011707 mineral Substances 0.000 claims description 8
- 235000010755 mineral Nutrition 0.000 claims description 8
- 230000035772 mutation Effects 0.000 claims description 8
- 150000008442 polyphenolic compounds Chemical class 0.000 claims description 8
- 235000013824 polyphenols Nutrition 0.000 claims description 8
- 210000002027 skeletal muscle Anatomy 0.000 claims description 8
- 235000019165 vitamin E Nutrition 0.000 claims description 8
- 229940046009 vitamin E Drugs 0.000 claims description 8
- 239000011709 vitamin E Substances 0.000 claims description 8
- 208000006304 Bethlem myopathy Diseases 0.000 claims description 7
- 102100036912 Desmin Human genes 0.000 claims description 7
- 108010044052 Desmin Proteins 0.000 claims description 7
- 101001023021 Homo sapiens LIM domain-binding protein 3 Proteins 0.000 claims description 7
- 102100035112 LIM domain-binding protein 3 Human genes 0.000 claims description 7
- 201000009342 Limb-girdle muscular dystrophy Diseases 0.000 claims description 7
- 208000022583 Qualitative or quantitative defects of dysferlin Diseases 0.000 claims description 7
- 208000037328 Qualitative or quantitative defects of sarcoglycan Diseases 0.000 claims description 7
- 210000005045 desmin Anatomy 0.000 claims description 7
- 208000002086 myofibrillar myopathy Diseases 0.000 claims description 7
- 208000010532 sarcoglycanopathy Diseases 0.000 claims description 7
- 238000010255 intramuscular injection Methods 0.000 claims description 5
- 238000010253 intravenous injection Methods 0.000 claims description 5
- 230000004220 muscle function Effects 0.000 claims description 4
- 230000036542 oxidative stress Effects 0.000 claims description 4
- 238000000034 method Methods 0.000 description 46
- 239000003814 drug Substances 0.000 description 9
- 241000702421 Dependoparvovirus Species 0.000 description 7
- 238000002360 preparation method Methods 0.000 description 7
- 108010085238 Actins Proteins 0.000 description 6
- 108090000362 Lymphotoxin-beta Proteins 0.000 description 6
- 230000000747 cardiac effect Effects 0.000 description 6
- 241001655883 Adeno-associated virus - 1 Species 0.000 description 4
- 241000702423 Adeno-associated virus - 2 Species 0.000 description 4
- 241000580270 Adeno-associated virus - 4 Species 0.000 description 4
- 241001634120 Adeno-associated virus - 5 Species 0.000 description 4
- 241000972680 Adeno-associated virus - 6 Species 0.000 description 4
- 241001164823 Adeno-associated virus - 7 Species 0.000 description 4
- 241001164825 Adeno-associated virus - 8 Species 0.000 description 4
- 241000649045 Adeno-associated virus 10 Species 0.000 description 4
- 241000649046 Adeno-associated virus 11 Species 0.000 description 4
- 241000649047 Adeno-associated virus 12 Species 0.000 description 4
- 241000300529 Adeno-associated virus 13 Species 0.000 description 4
- 102000004420 Creatine Kinase Human genes 0.000 description 3
- 108010042126 Creatine kinase Proteins 0.000 description 3
- 108700028146 Genetic Enhancer Elements Proteins 0.000 description 3
- 206010021143 Hypoxia Diseases 0.000 description 3
- 108091027981 Response element Proteins 0.000 description 3
- 108010065729 Troponin I Proteins 0.000 description 3
- 239000003623 enhancer Substances 0.000 description 3
- 239000003862 glucocorticoid Substances 0.000 description 3
- 210000000663 muscle cell Anatomy 0.000 description 3
- 230000009756 muscle regeneration Effects 0.000 description 3
- 208000011580 syndromic disease Diseases 0.000 description 3
- 102000004168 Dysferlin Human genes 0.000 description 2
- 108090000620 Dysferlin Proteins 0.000 description 2
- 239000003795 chemical substances by application Substances 0.000 description 2
- 230000001747 exhibiting effect Effects 0.000 description 2
- 230000001172 regenerating effect Effects 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021086103A JP7307121B2 (ja) | 2015-11-12 | 2021-05-21 | 筋ジストロフィーの治療方法 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562254539P | 2015-11-12 | 2015-11-12 | |
| US62/254,539 | 2015-11-12 | ||
| US201662419793P | 2016-11-09 | 2016-11-09 | |
| US62/419,793 | 2016-11-09 | ||
| PCT/US2016/061703 WO2017083776A1 (en) | 2015-11-12 | 2016-11-11 | Methods of treating muscular dystrophy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021086103A Division JP7307121B2 (ja) | 2015-11-12 | 2021-05-21 | 筋ジストロフィーの治療方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018538261A JP2018538261A (ja) | 2018-12-27 |
| JP2018538261A5 true JP2018538261A5 (enExample) | 2019-12-19 |
| JP6889717B2 JP6889717B2 (ja) | 2021-06-18 |
Family
ID=58696162
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018524411A Active JP6889717B2 (ja) | 2015-11-12 | 2016-11-11 | 筋ジストロフィーの治療方法 |
| JP2021086103A Active JP7307121B2 (ja) | 2015-11-12 | 2021-05-21 | 筋ジストロフィーの治療方法 |
| JP2023076538A Withdrawn JP2023099184A (ja) | 2015-11-12 | 2023-05-08 | 筋ジストロフィーの治療方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021086103A Active JP7307121B2 (ja) | 2015-11-12 | 2021-05-21 | 筋ジストロフィーの治療方法 |
| JP2023076538A Withdrawn JP2023099184A (ja) | 2015-11-12 | 2023-05-08 | 筋ジストロフィーの治療方法 |
Country Status (21)
| Country | Link |
|---|---|
| US (2) | US11820972B2 (enExample) |
| EP (1) | EP3373980B1 (enExample) |
| JP (3) | JP6889717B2 (enExample) |
| KR (1) | KR102821290B1 (enExample) |
| CN (1) | CN108883200B (enExample) |
| AU (3) | AU2016354561B2 (enExample) |
| BR (1) | BR112018009657A2 (enExample) |
| CA (1) | CA3005240A1 (enExample) |
| CO (1) | CO2018005928A2 (enExample) |
| DK (1) | DK3373980T3 (enExample) |
| EA (1) | EA201891134A1 (enExample) |
| ES (1) | ES3034857T3 (enExample) |
| FI (1) | FI3373980T3 (enExample) |
| HR (1) | HRP20250923T1 (enExample) |
| IL (1) | IL259178B (enExample) |
| MX (1) | MX2018005882A (enExample) |
| RU (1) | RU2761264C2 (enExample) |
| SG (1) | SG11201803971UA (enExample) |
| TN (2) | TN2018000163A1 (enExample) |
| WO (1) | WO2017083776A1 (enExample) |
| ZA (1) | ZA201803080B (enExample) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11338045B2 (en) | 2017-03-17 | 2022-05-24 | Newcastle University | Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy |
| EP3687565A1 (en) | 2017-09-27 | 2020-08-05 | Alexion Pharmaceuticals, Inc. | Methods of improving cardiovascular function and treating cardiovascular disease using a recombinant ectonucleotide pyrophosphatase phosphodiesterase (npp1) |
| BR112020007790A2 (pt) | 2017-10-20 | 2020-10-20 | Research Institute At Nationwide Children's Hospital | métodos e materiais para terapia genética com nt-3 |
| US12491265B2 (en) | 2018-06-18 | 2025-12-09 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
| CA3124963A1 (en) * | 2018-12-31 | 2020-07-09 | Research Institute At Nationwide Children's Hospital | Dux4 rna silencing using rna targeting crispr-cas13b |
| TW202208630A (zh) | 2020-06-15 | 2022-03-01 | 美國全美兒童醫院之研究學會 | 針對肌肉營養不良症的腺相關病毒載體遞送 |
| KR102732922B1 (ko) * | 2021-10-29 | 2024-11-20 | 충남대학교 산학협력단 | 과산화수소수 처리에 의해 유용성분 함량이 증진된 상추의 재배방법 |
| CN117223686B (zh) * | 2023-11-15 | 2024-01-23 | 四川省农业机械科学研究院 | 一种扩座除砂一体化蚕盘及养蚕方法 |
Family Cites Families (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IE58011B1 (en) | 1983-05-27 | 1993-06-16 | Texas A & M Univ Sys | Method for producing a recombinant baculovirus expression vector |
| DK518384A (da) | 1984-01-31 | 1985-07-01 | Idaho Res Found | Vektor til fremstilling af et gen-produkt i insektceller, fremgangsmaade til dens fremstilling samt dens anvendelse |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US6962815B2 (en) | 2001-01-05 | 2005-11-08 | Children's Hopital Inc. | AAV2 vectors and methods |
| WO2003074714A1 (en) | 2002-03-05 | 2003-09-12 | Stichting Voor De Technische Wetenschappen | Baculovirus expression system |
| JP2006121961A (ja) * | 2004-10-28 | 2006-05-18 | Univ Of Tokushima | 顎骨骨幹異形成症ｇｄｄの原因遺伝子ｇｄｄ１とその用途 |
| EP2052088A2 (en) * | 2006-08-02 | 2009-04-29 | Genizon Biosciences | Genemap of the human genes associated with psoriasis |
| FR2919305B1 (fr) | 2007-07-26 | 2009-09-18 | Genethon Ass Loi De 1901 | Vecteurs viraux adeno-associes pour l'expression de la dysferline. |
| GB0715087D0 (en) | 2007-08-03 | 2007-09-12 | Summit Corp Plc | Drug combinations for the treatment of duchenne muscular dystrophy |
| US20100026655A1 (en) | 2008-07-31 | 2010-02-04 | Avago Technologies Ecbu Ip (Singapore) Pte. Ltd. | Capacitive Touchscreen or Touchpad for Finger or Stylus |
| WO2011044138A1 (en) * | 2009-10-05 | 2011-04-14 | Catabasis Pharmaceuticals, Inc. | Lipoic acid acylated salicylate derivatives and their uses |
| WO2011056867A1 (en) | 2009-11-03 | 2011-05-12 | The Regents Of The University Of California | High brightness light emitting diode covered by zinc oxide layers on multiple surfaces grown in low temperature aqueous solution |
| WO2013078316A1 (en) * | 2011-11-23 | 2013-05-30 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| WO2013151663A1 (en) | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of membrane proteins |
| US20150247196A1 (en) * | 2012-09-07 | 2015-09-03 | INSERM (Institut National de la Santé et de la Recherche Médicale) | In vitro genetic diagnostic of inherited neuromuscular disorders |
| US9624282B2 (en) | 2012-11-26 | 2017-04-18 | The Curators Of The University Of Missouri | Microdystrophin peptides and methods for treating muscular dystrophy using the same |
| ITTO20130669A1 (it) | 2013-08-05 | 2015-02-06 | Consiglio Nazionale Ricerche | Vettore adeno-associato ricombinante muscolo-specifico e suo impiego nel trattamento di patologie muscolari |
| PT2859896T (pt) * | 2013-10-08 | 2018-04-26 | Ystem S R L | Composições farmacêuticas para o tratamento de distúrbios musculares |
| US9850497B2 (en) * | 2013-11-04 | 2017-12-26 | Regents Of The University Of Minnesota | Gene targeting methods and tools |
-
2016
- 2016-11-11 RU RU2018121289A patent/RU2761264C2/ru active
- 2016-11-11 AU AU2016354561A patent/AU2016354561B2/en active Active
- 2016-11-11 CN CN201680079597.0A patent/CN108883200B/zh active Active
- 2016-11-11 JP JP2018524411A patent/JP6889717B2/ja active Active
- 2016-11-11 KR KR1020187016152A patent/KR102821290B1/ko active Active
- 2016-11-11 SG SG11201803971UA patent/SG11201803971UA/en unknown
- 2016-11-11 IL IL259178A patent/IL259178B/en unknown
- 2016-11-11 CA CA3005240A patent/CA3005240A1/en active Pending
- 2016-11-11 HR HRP20250923TT patent/HRP20250923T1/hr unknown
- 2016-11-11 ES ES16865168T patent/ES3034857T3/es active Active
- 2016-11-11 TN TNP/2018/000163A patent/TN2018000163A1/en unknown
- 2016-11-11 EP EP16865168.5A patent/EP3373980B1/en active Active
- 2016-11-11 FI FIEP16865168.5T patent/FI3373980T3/fi active
- 2016-11-11 US US15/775,566 patent/US11820972B2/en active Active
- 2016-11-11 DK DK16865168.5T patent/DK3373980T3/da active
- 2016-11-11 WO PCT/US2016/061703 patent/WO2017083776A1/en not_active Ceased
- 2016-11-11 MX MX2018005882A patent/MX2018005882A/es unknown
- 2016-11-11 EA EA201891134A patent/EA201891134A1/ru unknown
- 2016-11-11 BR BR112018009657-1A patent/BR112018009657A2/pt active Search and Examination
- 2016-11-11 TN TNP/2019/000279A patent/TN2019000279A1/en unknown
-
2018
- 2018-05-10 ZA ZA2018/03080A patent/ZA201803080B/en unknown
- 2018-06-08 CO CONC2018/0005928A patent/CO2018005928A2/es unknown
-
2021
- 2021-05-21 JP JP2021086103A patent/JP7307121B2/ja active Active
-
2022
- 2022-03-02 AU AU2022201427A patent/AU2022201427B2/en active Active
-
2023
- 2023-05-08 JP JP2023076538A patent/JP2023099184A/ja not_active Withdrawn
- 2023-11-17 US US18/513,227 patent/US20240191250A1/en active Pending
-
2025
- 2025-07-22 AU AU2025206419A patent/AU2025206419A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2018538261A5 (enExample) | ||
| JP2019513393A5 (enExample) | ||
| JP2019513779A5 (enExample) | ||
| IL259178B (en) | A vector encoding ano5 for the treatment of muscular dystrophy | |
| JP7094236B2 (ja) | 変異ａａｖ、及び、細胞、臓器並びに組織への遺伝子導入のための組成物、方法並びに使用法 | |
| JP2020513811A5 (enExample) | ||
| JP2019513399A5 (enExample) | ||
| JP2019513389A5 (enExample) | ||
| Pryadkina et al. | A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequence | |
| FI3442600T3 (fi) | Adeno-assosioidulla virusvektorilla b-sarkoglykaanin ja mikro-rna-29:n toimittaminen ja lihasdystrofian hoito | |
| WO2016077687A4 (en) | Compositions and methods of treating amyotrophic lateral sclerosis (als) | |
| IL320714A (en) | Gene therapy for limb-girdle muscular dystrophy type 2c | |
| JP2021500352A5 (enExample) | ||
| JP2013531471A5 (enExample) | ||
| RU2018116076A (ru) | Модифицированные гены атаксии фридрейха и векторы для генной терапии | |
| BR112014025985A2 (pt) | composição e métodos para transferência de gene altamente eficaz com o uso de variantes de capsídeo de aav | |
| JP2023544683A (ja) | ペプチド改変されたａａｖカプシド | |
| RU2020109343A (ru) | Полинуклеотиды aadc для лечения болезни паркинсона | |
| HRP20201200T1 (hr) | OLIGONUKLEOTIDNI SPOJEVI ZA CILJANJE HUNTINGTIN mRNK | |
| JP2017510298A5 (enExample) | ||
| HRP20231077T1 (hr) | Genska terapija za pigmentni retinitis | |
| JP2017512466A5 (enExample) | ||
| MX375360B (es) | Vectores de virus adeno-asociado para el tratamiento de la enfermedad de almacenamiento de glucógeno. | |
| JP2017535266A5 (enExample) | ||
| RU2015144234A (ru) | Композиции и способы лечения mps1 |