JPWO2019246125A5 - - Google Patents
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- JPWO2019246125A5 JPWO2019246125A5 JP2020570457A JP2020570457A JPWO2019246125A5 JP WO2019246125 A5 JPWO2019246125 A5 JP WO2019246125A5 JP 2020570457 A JP2020570457 A JP 2020570457A JP 2020570457 A JP2020570457 A JP 2020570457A JP WO2019246125 A5 JPWO2019246125 A5 JP WO2019246125A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- domain
- polynucleotide
- raav
- nucleotide sequence
- 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.)
- Pending
Links
- 229920000023 polynucleotide Polymers 0.000 claims description 64
- 239000002157 polynucleotide Substances 0.000 claims description 64
- 229920001850 Nucleic acid sequence Polymers 0.000 claims description 46
- 239000002773 nucleotide Substances 0.000 claims description 24
- 125000003729 nucleotide group Chemical group 0.000 claims description 24
- 102000004169 proteins and genes Human genes 0.000 claims description 24
- 108090000623 proteins and genes Proteins 0.000 claims description 24
- 102100016400 HBEGF Human genes 0.000 claims description 18
- 101700029736 HBEGF Proteins 0.000 claims description 18
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 14
- 102000008000 human cationic antimicrobial protein CAP 37 Human genes 0.000 claims description 14
- 108010089633 human cationic antimicrobial protein CAP 37 Proteins 0.000 claims description 14
- 239000002245 particle Substances 0.000 claims description 13
- 241000432074 Adeno-associated virus Species 0.000 claims description 10
- 210000003205 Muscles Anatomy 0.000 claims description 7
- 102000012545 EGF-like domain Human genes 0.000 claims description 6
- 108050002150 EGF-like domain Proteins 0.000 claims description 6
- 108010085895 Laminin Proteins 0.000 claims description 6
- 230000002950 deficient Effects 0.000 claims description 6
- 108010008097 laminin alpha 2 Proteins 0.000 claims description 6
- 102000003995 transcription factors Human genes 0.000 claims description 6
- 108090000464 transcription factors Proteins 0.000 claims description 6
- 208000003627 Muscular Dystrophy Diseases 0.000 claims description 5
- 201000006938 muscular dystrophy Diseases 0.000 claims description 5
- 102100008656 FKRP Human genes 0.000 claims description 4
- 101700079499 FKRP Proteins 0.000 claims description 4
- 210000000234 Capsid Anatomy 0.000 claims description 3
- 241000699802 Cricetulus griseus Species 0.000 claims description 3
- 210000001672 Ovary Anatomy 0.000 claims description 3
- 208000008581 Brain Disease Diseases 0.000 claims description 2
- 102000007623 Dystroglycans Human genes 0.000 claims description 2
- 108010071885 Dystroglycans Proteins 0.000 claims description 2
- 206010014623 Encephalopathy Diseases 0.000 claims description 2
- 206010014625 Encephalopathy Diseases 0.000 claims description 2
- 102100008653 FKTN Human genes 0.000 claims description 2
- 101700057427 FKTN Proteins 0.000 claims description 2
- 102100011563 LAMA2 Human genes 0.000 claims description 2
- 101700027052 LAMA2 Proteins 0.000 claims description 2
- 208000002245 Muscular Dystrophy, Limb-Girdle, Type 2I Diseases 0.000 claims description 2
- 208000001847 Muscular Dystrophy, Limb-Girdle, Type 2M Diseases 0.000 claims description 2
- 102100013628 POMGNT1 Human genes 0.000 claims description 2
- 101710004588 POMGNT1 Proteins 0.000 claims description 2
- 102100006023 POMT1 Human genes 0.000 claims description 2
- 101700070013 POMT1 Proteins 0.000 claims description 2
- 102100019850 POMT2 Human genes 0.000 claims description 2
- 101700067704 POMT2 Proteins 0.000 claims description 2
- 201000006793 Walker-Warburg syndrome Diseases 0.000 claims description 2
- 201000009510 autosomal recessive limb-girdle muscular dystrophy type 2I Diseases 0.000 claims description 2
- 201000009512 autosomal recessive limb-girdle muscular dystrophy type 2K Diseases 0.000 claims description 2
- 201000009513 autosomal recessive limb-girdle muscular dystrophy type 2M Diseases 0.000 claims description 2
- 201000009511 autosomal recessive limb-girdle muscular dystrophy type 2N Diseases 0.000 claims description 2
- 201000009507 autosomal recessive limb-girdle muscular dystrophy type 2O Diseases 0.000 claims description 2
- 201000009516 autosomal recessive limb-girdle muscular dystrophy type 2P Diseases 0.000 claims description 2
- 201000009515 autosomal recessive limb-girdle muscular dystrophy type 2T Diseases 0.000 claims description 2
- 201000009514 autosomal recessive limb-girdle muscular dystrophy type 2U Diseases 0.000 claims description 2
- 201000006948 congenital merosin-deficient muscular dystrophy 1A Diseases 0.000 claims description 2
- 239000003102 growth factor Substances 0.000 claims description 2
- 201000006951 muscular dystrophy-dystroglycanopathy type B5 Diseases 0.000 claims description 2
- 201000006946 muscular dystrophy-dystroglycanopathy type B6 Diseases 0.000 claims description 2
- 235000017274 Diospyros sandwicensis Nutrition 0.000 claims 3
- 241000282838 Lama Species 0.000 claims 3
- 201000006813 Fukuyama congenital muscular dystrophy Diseases 0.000 claims 1
- 239000003814 drug Substances 0.000 description 2
- 229940079593 drugs Drugs 0.000 description 2
- 241000700605 Viruses Species 0.000 description 1
- 201000006815 congenital muscular dystrophy Diseases 0.000 description 1
- 229920001184 polypeptide Polymers 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862686522P | 2018-06-18 | 2018-06-18 | |
| US62/686,522 | 2018-06-18 | ||
| PCT/US2019/037769 WO2019246125A1 (fr) | 2018-06-18 | 2019-06-18 | Produits de virus adéno-associés de recombinaison et méthodes de traitement de dystroglycanopathies et de dystrophies musculaires déficientes en laminine |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021527418A JP2021527418A (ja) | 2021-10-14 |
| JPWO2019246125A5 true JPWO2019246125A5 (fr) | 2022-06-28 |
Family
ID=67263074
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020570457A Pending JP2021527418A (ja) | 2018-06-18 | 2019-06-18 | ジストログリカノパチーおよびラミニン欠損筋ジストロフィーを治療するための組換えアデノ随伴ウイルス生成物および方法 |
Country Status (5)
| Country | Link |
|---|---|
| EP (1) | EP3807309A1 (fr) |
| JP (1) | JP2021527418A (fr) |
| AU (1) | AU2019288291A1 (fr) |
| CA (1) | CA3104471A1 (fr) |
| WO (1) | WO2019246125A1 (fr) |
Family Cites Families (23)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| EP0733103B1 (fr) | 1993-11-09 | 2004-03-03 | Targeted Genetics Corporation | Production de titres eleves de vecteurs d'aav recombinants |
| US5837484A (en) | 1993-11-09 | 1998-11-17 | Medical College Of Ohio | Stable cell lines capable of expressing the adeno-associated virus replication gene |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| WO1996017947A1 (fr) | 1994-12-06 | 1996-06-13 | Targeted Genetics Corporation | Lignees cellulaires d'encapsidation utilisees pour la generation de titres hauts de vecteurs aav recombinants |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| AU722196B2 (en) | 1995-08-30 | 2000-07-27 | Genzyme Corporation | Chromatographic purification of adenovirus and AAV |
| AU715543B2 (en) | 1995-09-08 | 2000-02-03 | Genzyme Corporation | Improved AAV vectors for gene therapy |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| EP1696036B1 (fr) | 1996-09-06 | 2010-04-21 | The Trustees of The University of Pennsylvania | AAV recombinantes pour la production des médicaments pour la thérapie génétique de cellules musculaires |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| EP2325299A3 (fr) | 1997-09-05 | 2011-10-05 | Targeted Genetics Corporation | Procédés de génération de préparations de vecteurs AAV recombinants dont le titre est élevé et qui sont exemptes de virus assistant |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| AU2001255575B2 (en) | 2000-04-28 | 2006-08-31 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| US6962815B2 (en) | 2001-01-05 | 2005-11-08 | Children's Hopital Inc. | AAV2 vectors and methods |
| WO2013078316A1 (fr) | 2011-11-23 | 2013-05-30 | Nationwide Children's Hospital, Inc. | Administration de virus adéno-associé recombinant de polynucléotides alpha-sarcoglycanes |
| DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
| JP2015092462A (ja) | 2013-09-30 | 2015-05-14 | Tdk株式会社 | 正極及びそれを用いたリチウムイオン二次電池 |
| JP6202701B2 (ja) | 2014-03-21 | 2017-09-27 | 株式会社日立国際電気 | 基板処理装置、半導体装置の製造方法及びプログラム |
| JP6197169B2 (ja) | 2014-09-29 | 2017-09-20 | 東芝メモリ株式会社 | 半導体装置の製造方法 |
| CA2998636A1 (fr) * | 2015-09-17 | 2017-03-23 | Research Institute At Nationwide Children's Hospital | Procedes et materiaux pour therapie genique par galgt2 |
| MA45477A (fr) * | 2016-04-15 | 2019-02-20 | Res Inst Nationwide Childrens Hospital | Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire |
-
2019
- 2019-06-18 CA CA3104471A patent/CA3104471A1/fr active Pending
- 2019-06-18 EP EP19739791.2A patent/EP3807309A1/fr active Pending
- 2019-06-18 JP JP2020570457A patent/JP2021527418A/ja active Pending
- 2019-06-18 AU AU2019288291A patent/AU2019288291A1/en active Pending
- 2019-06-18 WO PCT/US2019/037769 patent/WO2019246125A1/fr unknown
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