JPWO2020123645A5 - - Google Patents
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- JPWO2020123645A5 JPWO2020123645A5 JP2021533367A JP2021533367A JPWO2020123645A5 JP WO2020123645 A5 JPWO2020123645 A5 JP WO2020123645A5 JP 2021533367 A JP2021533367 A JP 2021533367A JP 2021533367 A JP2021533367 A JP 2021533367A JP WO2020123645 A5 JPWO2020123645 A5 JP WO2020123645A5
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- JP
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- Prior art keywords
- recombinant viral
- viral vector
- sequence
- gene
- vector
- 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.)
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- 239000013603 viral vector Substances 0.000 claims description 74
- 108700011259 MicroRNAs Proteins 0.000 claims description 22
- 108090000623 proteins and genes Proteins 0.000 claims description 21
- 239000002679 microRNA Substances 0.000 claims description 20
- 108091033319 polynucleotide Proteins 0.000 claims description 20
- 102000040430 polynucleotide Human genes 0.000 claims description 20
- 239000002157 polynucleotide Substances 0.000 claims description 20
- 230000000692 anti-sense effect Effects 0.000 claims description 18
- 239000000203 mixture Substances 0.000 claims description 16
- 102000001039 Dystrophin Human genes 0.000 claims description 14
- 108010069091 Dystrophin Proteins 0.000 claims description 14
- 239000013598 vector Substances 0.000 claims description 14
- 108091033409 CRISPR Proteins 0.000 claims description 12
- 230000002950 deficient Effects 0.000 claims description 12
- 239000013607 AAV vector Substances 0.000 claims description 11
- 210000004027 cell Anatomy 0.000 claims description 10
- 108091027967 Small hairpin RNA Proteins 0.000 claims description 9
- 102000004169 proteins and genes Human genes 0.000 claims description 9
- 238000010354 CRISPR gene editing Methods 0.000 claims description 8
- 208000037141 Congenital muscular dystrophy, Fukuyama type Diseases 0.000 claims description 8
- 201000006813 Fukuyama congenital muscular dystrophy Diseases 0.000 claims description 8
- 108091030071 RNAI Proteins 0.000 claims description 8
- 201000006793 Walker-Warburg syndrome Diseases 0.000 claims description 8
- 230000009368 gene silencing by RNA Effects 0.000 claims description 8
- 238000000034 method Methods 0.000 claims description 8
- 210000003205 muscle Anatomy 0.000 claims description 8
- 208000025855 muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A, 4 Diseases 0.000 claims description 8
- 230000001105 regulatory effect Effects 0.000 claims description 8
- 102000009023 sarcolipin Human genes 0.000 claims description 8
- 108010088766 sarcolipin Proteins 0.000 claims description 8
- 108700024394 Exon Proteins 0.000 claims description 7
- 101000616435 Homo sapiens Gamma-sarcoglycan Proteins 0.000 claims description 7
- 239000004055 small Interfering RNA Substances 0.000 claims description 7
- 108020005345 3' Untranslated Regions Proteins 0.000 claims description 6
- 108091026890 Coding region Proteins 0.000 claims description 6
- 108010059343 MM Form Creatine Kinase Proteins 0.000 claims description 6
- 108010057466 NF-kappa B Proteins 0.000 claims description 6
- 102000003945 NF-kappa B Human genes 0.000 claims description 6
- 201000006948 congenital merosin-deficient muscular dystrophy 1A Diseases 0.000 claims description 6
- 108091047189 miR-29c stem-loop Proteins 0.000 claims description 6
- 108091054490 miR-29c-2 stem-loop Proteins 0.000 claims description 6
- 201000006938 muscular dystrophy Diseases 0.000 claims description 6
- 239000008194 pharmaceutical composition Substances 0.000 claims description 6
- 102100021792 Gamma-sarcoglycan Human genes 0.000 claims description 5
- 108010085238 Actins Proteins 0.000 claims description 4
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 4
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 4
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 4
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 4
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 4
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims description 4
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 4
- 241000649045 Adeno-associated virus 10 Species 0.000 claims description 4
- 241000649046 Adeno-associated virus 11 Species 0.000 claims description 4
- 241000649047 Adeno-associated virus 12 Species 0.000 claims description 4
- 241000300529 Adeno-associated virus 13 Species 0.000 claims description 4
- 101150086792 CLCN1 gene Proteins 0.000 claims description 4
- 108700004991 Cas12a Proteins 0.000 claims description 4
- 102000015225 Connective Tissue Growth Factor Human genes 0.000 claims description 4
- 108010039419 Connective Tissue Growth Factor Proteins 0.000 claims description 4
- 241000702421 Dependoparvovirus Species 0.000 claims description 4
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims description 4
- 102100032248 Dysferlin Human genes 0.000 claims description 4
- 208000037149 Facioscapulohumeral dystrophy Diseases 0.000 claims description 4
- 102100029100 Hematopoietic prostaglandin D synthase Human genes 0.000 claims description 4
- 101710082112 Hematopoietic prostaglandin D synthase Proteins 0.000 claims description 4
- HNDVDQJCIGZPNO-YFKPBYRVSA-N L-histidine Chemical compound OC(=O)[C@@H](N)CC1=CN=CN1 HNDVDQJCIGZPNO-YFKPBYRVSA-N 0.000 claims description 4
- 102100022745 Laminin subunit alpha-2 Human genes 0.000 claims description 4
- TWRXJAOTZQYOKJ-UHFFFAOYSA-L Magnesium chloride Chemical compound [Mg+2].[Cl-].[Cl-] TWRXJAOTZQYOKJ-UHFFFAOYSA-L 0.000 claims description 4
- 102000005604 Myosin Heavy Chains Human genes 0.000 claims description 4
- 108010084498 Myosin Heavy Chains Proteins 0.000 claims description 4
- 108010052185 Myotonin-Protein Kinase Proteins 0.000 claims description 4
- 102100031754 Ribitol-5-phosphate transferase FKTN Human genes 0.000 claims description 4
- 101710087566 Ribitol-5-phosphate transferase FKTN Proteins 0.000 claims description 4
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims description 4
- 108020000411 Toll-like receptor Proteins 0.000 claims description 4
- 102000002689 Toll-like receptor Human genes 0.000 claims description 4
- 239000007864 aqueous solution Substances 0.000 claims description 4
- 230000000747 cardiac effect Effects 0.000 claims description 4
- 239000003085 diluting agent Substances 0.000 claims description 4
- 208000008570 facioscapulohumeral muscular dystrophy Diseases 0.000 claims description 4
- 230000002757 inflammatory effect Effects 0.000 claims description 4
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 4
- 108010029731 6-phosphogluconolactonase Proteins 0.000 claims description 3
- 208000033136 Gamma-sarcoglycan-related limb-girdle muscular dystrophy R5 Diseases 0.000 claims description 3
- 108010018962 Glucosephosphate Dehydrogenase Proteins 0.000 claims description 3
- 101001016184 Homo sapiens Dysferlin Proteins 0.000 claims description 3
- 201000009562 autosomal recessive limb-girdle muscular dystrophy type 2C Diseases 0.000 claims description 3
- 201000006815 congenital muscular dystrophy Diseases 0.000 claims description 3
- 238000010362 genome editing Methods 0.000 claims description 3
- MZOFCQQQCNRIBI-VMXHOPILSA-N (3s)-4-[[(2s)-1-[[(2s)-1-[[(1s)-1-carboxy-2-hydroxyethyl]amino]-4-methyl-1-oxopentan-2-yl]amino]-5-(diaminomethylideneamino)-1-oxopentan-2-yl]amino]-3-[[2-[[(2s)-2,6-diaminohexanoyl]amino]acetyl]amino]-4-oxobutanoic acid Chemical compound OC[C@@H](C(O)=O)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CCCN=C(N)N)NC(=O)[C@H](CC(O)=O)NC(=O)CNC(=O)[C@@H](N)CCCCN MZOFCQQQCNRIBI-VMXHOPILSA-N 0.000 claims description 2
- 201000006935 Becker muscular dystrophy Diseases 0.000 claims description 2
- 101100381481 Caenorhabditis elegans baz-2 gene Proteins 0.000 claims description 2
- 102000008186 Collagen Human genes 0.000 claims description 2
- 108010035532 Collagen Proteins 0.000 claims description 2
- 102000004420 Creatine Kinase Human genes 0.000 claims description 2
- 108010042126 Creatine kinase Proteins 0.000 claims description 2
- 108090000695 Cytokines Proteins 0.000 claims description 2
- 102000004127 Cytokines Human genes 0.000 claims description 2
- 101150083642 DYSF gene Proteins 0.000 claims description 2
- 108090000620 Dysferlin Proteins 0.000 claims description 2
- 102000016942 Elastin Human genes 0.000 claims description 2
- 108010014258 Elastin Proteins 0.000 claims description 2
- 102000004190 Enzymes Human genes 0.000 claims description 2
- 108090000790 Enzymes Proteins 0.000 claims description 2
- 102000010834 Extracellular Matrix Proteins Human genes 0.000 claims description 2
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- 101150091020 FKTN gene Proteins 0.000 claims description 2
- 108700028146 Genetic Enhancer Elements Proteins 0.000 claims description 2
- 108020005004 Guide RNA Proteins 0.000 claims description 2
- 102000003964 Histone deacetylase Human genes 0.000 claims description 2
- 108090000353 Histone deacetylase Proteins 0.000 claims description 2
- 102100039999 Histone deacetylase 2 Human genes 0.000 claims description 2
- 101001035011 Homo sapiens Histone deacetylase 2 Proteins 0.000 claims description 2
- 101100127663 Homo sapiens LAMA2 gene Proteins 0.000 claims description 2
- 101000972491 Homo sapiens Laminin subunit alpha-2 Proteins 0.000 claims description 2
- 206010021143 Hypoxia Diseases 0.000 claims description 2
- -1 IL-1β Proteins 0.000 claims description 2
- 108010002352 Interleukin-1 Proteins 0.000 claims description 2
- 108090001005 Interleukin-6 Proteins 0.000 claims description 2
- 101150089565 LAMA2 gene Proteins 0.000 claims description 2
- 201000009342 Limb-girdle muscular dystrophy Diseases 0.000 claims description 2
- 102000004472 Myostatin Human genes 0.000 claims description 2
- 108010056852 Myostatin Proteins 0.000 claims description 2
- 206010068871 Myotonic dystrophy Diseases 0.000 claims description 2
- 102000018658 Myotonin-Protein Kinase Human genes 0.000 claims description 2
- 108010076504 Protein Sorting Signals Proteins 0.000 claims description 2
- 101100372762 Rattus norvegicus Flt1 gene Proteins 0.000 claims description 2
- 108091027981 Response element Proteins 0.000 claims description 2
- 102000004887 Transforming Growth Factor beta Human genes 0.000 claims description 2
- 108090001012 Transforming Growth Factor beta Proteins 0.000 claims description 2
- 108060008682 Tumor Necrosis Factor Proteins 0.000 claims description 2
- 102000000852 Tumor Necrosis Factor-alpha Human genes 0.000 claims description 2
- 102000011016 Type 5 Cyclic Nucleotide Phosphodiesterases Human genes 0.000 claims description 2
- 108010037581 Type 5 Cyclic Nucleotide Phosphodiesterases Proteins 0.000 claims description 2
- 102000016548 Vascular Endothelial Growth Factor Receptor-1 Human genes 0.000 claims description 2
- 108010053096 Vascular Endothelial Growth Factor Receptor-1 Proteins 0.000 claims description 2
- 102000005789 Vascular Endothelial Growth Factors Human genes 0.000 claims description 2
- 108010019530 Vascular Endothelial Growth Factors Proteins 0.000 claims description 2
- 230000003213 activating effect Effects 0.000 claims description 2
- 230000002411 adverse Effects 0.000 claims description 2
- 210000004899 c-terminal region Anatomy 0.000 claims description 2
- 229920001436 collagen Polymers 0.000 claims description 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 2
- 208000035475 disorder Diseases 0.000 claims description 2
- 239000002552 dosage form Substances 0.000 claims description 2
- 229920002549 elastin Polymers 0.000 claims description 2
- 239000003623 enhancer Substances 0.000 claims description 2
- 210000002744 extracellular matrix Anatomy 0.000 claims description 2
- 239000003862 glucocorticoid Substances 0.000 claims description 2
- 229960002885 histidine Drugs 0.000 claims description 2
- 239000003112 inhibitor Substances 0.000 claims description 2
- 230000037431 insertion Effects 0.000 claims description 2
- 238000003780 insertion Methods 0.000 claims description 2
- 238000010255 intramuscular injection Methods 0.000 claims description 2
- 239000007927 intramuscular injection Substances 0.000 claims description 2
- 238000010253 intravenous injection Methods 0.000 claims description 2
- 230000002934 lysing effect Effects 0.000 claims description 2
- 229910001629 magnesium chloride Inorganic materials 0.000 claims description 2
- 108020004999 messenger RNA Proteins 0.000 claims description 2
- 108091028606 miR-1 stem-loop Proteins 0.000 claims description 2
- 108091079012 miR-133a Proteins 0.000 claims description 2
- 108091024038 miR-133a stem-loop Proteins 0.000 claims description 2
- 108091063796 miR-206 stem-loop Proteins 0.000 claims description 2
- 108091055059 miR-30c stem-loop Proteins 0.000 claims description 2
- 210000000663 muscle cell Anatomy 0.000 claims description 2
- 230000035772 mutation Effects 0.000 claims description 2
- 239000002773 nucleotide Substances 0.000 claims description 2
- 125000003729 nucleotide group Chemical group 0.000 claims description 2
- 238000007911 parenteral administration Methods 0.000 claims description 2
- 230000008488 polyadenylation Effects 0.000 claims description 2
- 208000022587 qualitative or quantitative defects of dystrophin Diseases 0.000 claims description 2
- 108700015048 receptor decoy activity proteins Proteins 0.000 claims description 2
- 108020003175 receptors Proteins 0.000 claims description 2
- 102000005962 receptors Human genes 0.000 claims description 2
- 230000019491 signal transduction Effects 0.000 claims description 2
- 210000002027 skeletal muscle Anatomy 0.000 claims description 2
- 239000011780 sodium chloride Substances 0.000 claims description 2
- 238000007910 systemic administration Methods 0.000 claims description 2
- ZRKFYGHZFMAOKI-QMGMOQQFSA-N tgfbeta Chemical compound C([C@H](NC(=O)[C@H](C(C)C)NC(=O)CNC(=O)[C@H](CCC(O)=O)NC(=O)[C@H](CCCNC(N)=N)NC(=O)[C@H](CC(N)=O)NC(=O)[C@H](CC(C)C)NC(=O)[C@H]([C@@H](C)O)NC(=O)[C@H](CCC(O)=O)NC(=O)[C@H]([C@@H](C)O)NC(=O)[C@H](CC(C)C)NC(=O)CNC(=O)[C@H](C)NC(=O)[C@H](CO)NC(=O)[C@H](CCC(N)=O)NC(=O)[C@@H](NC(=O)[C@H](C)NC(=O)[C@H](C)NC(=O)[C@@H](NC(=O)[C@H](CC(C)C)NC(=O)[C@@H](N)CCSC)C(C)C)[C@@H](C)CC)C(=O)N[C@@H]([C@@H](C)O)C(=O)N[C@@H](C(C)C)C(=O)N[C@@H](CC=1C=CC=CC=1)C(=O)N[C@@H](C)C(=O)N1[C@@H](CCC1)C(=O)N[C@@H]([C@@H](C)O)C(=O)N[C@@H](CC(N)=O)C(=O)N[C@@H](CCC(O)=O)C(=O)N[C@@H](C)C(=O)N[C@@H](CC=1C=CC=CC=1)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](C)C(=O)N[C@@H](CC(C)C)C(=O)N1[C@@H](CCC1)C(=O)N1[C@@H](CCC1)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CCC(O)=O)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CO)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CC(C)C)C(O)=O)C1=CC=C(O)C=C1 ZRKFYGHZFMAOKI-QMGMOQQFSA-N 0.000 claims description 2
- 102100035172 Glucose-6-phosphate 1-dehydrogenase Human genes 0.000 claims 2
- 206010010356 Congenital anomaly Diseases 0.000 claims 1
- 102000004168 Dysferlin Human genes 0.000 claims 1
- 201000005973 campomelic dysplasia Diseases 0.000 claims 1
- 208000019484 coronary microvascular disease Diseases 0.000 claims 1
- 230000000694 effects Effects 0.000 claims 1
- 108020004459 Small interfering RNA Proteins 0.000 description 3
- 239000002924 silencing RNA Substances 0.000 description 3
- 102100031126 6-phosphogluconolactonase Human genes 0.000 description 2
- 239000012190 activator Substances 0.000 description 1
- 208000015181 infectious disease Diseases 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862778646P | 2018-12-12 | 2018-12-12 | |
| US62/778,646 | 2018-12-12 | ||
| PCT/US2019/065718 WO2020123645A1 (en) | 2018-12-12 | 2019-12-11 | Combination therapy for treating muscular dystrophy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022513456A JP2022513456A (ja) | 2022-02-08 |
| JPWO2020123645A5 true JPWO2020123645A5 (enrdf_load_stackoverflow) | 2022-12-20 |
Family
ID=71076668
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021533367A Pending JP2022513456A (ja) | 2018-12-12 | 2019-12-11 | 筋ジストロフィーの治療のための併用療法 |
Country Status (9)
Families Citing this family (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019012336A2 (en) | 2017-03-17 | 2019-01-17 | Newcastle University | ADENO-ASSOCIATED VIRAL VECTOR DELIVERY OF A MICRO-DYSTROPHIN FRAGMENT FOR TREATING MUSCLE DYSTROPHY |
| JP7361701B2 (ja) | 2018-01-31 | 2023-10-16 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 肢帯型筋ジストロフィー２ｃ型のための遺伝子治療 |
| SG11202012715UA (en) | 2018-06-29 | 2021-01-28 | Res Inst Nationwide Childrens Hospital | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a |
| TW202045531A (zh) | 2019-02-26 | 2020-12-16 | 美國全美兒童醫院之研究學會 | Β-肌聚糖之腺相關病毒載體遞送及肌肉營養不良症之治療 |
| HUE066215T2 (hu) | 2019-08-21 | 2024-07-28 | Res Institute At Nationwide | Alfa-szarkoglikán bevitele adenoasszociáltvírus-vektorral és izomdisztrófia kezelése |
| AU2021205965A1 (en) * | 2020-01-10 | 2022-09-01 | Solid Biosciences Inc. | Viral vector for combination therapy |
| MX2022016332A (es) | 2020-06-15 | 2023-04-03 | Res Inst Nationwide Childrens Hospital | Administracion de vectores de virus adenoasociados para distrofias musculares. |
| EP4086276A1 (en) | 2021-05-03 | 2022-11-09 | Université d'Aix-Marseille | Composition for treating dysferlinopathy |
| EP4108263A3 (en) | 2021-06-02 | 2023-03-22 | Research Institute at Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a |
| US20240390522A1 (en) * | 2021-09-28 | 2024-11-28 | University Of Florida Research Foundation, Incorporated | Gene therapy for duchenne muscular dystrophy |
| JP2023059858A (ja) | 2021-10-15 | 2023-04-27 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 自己相補的アデノ随伴ウイルスベクター及び筋ジストロフィーの治療におけるその使用 |
| WO2023196853A1 (en) * | 2022-04-05 | 2023-10-12 | Astellas Gene Therapies, Inc. | Compositions and methods for the treatment of muscular dystrophies |
| WO2023225592A2 (en) * | 2022-05-18 | 2023-11-23 | Inadcure Foundation Inc. | Gene therapies for treatment of infantile neuroaxonal dystrophy |
| AU2023356650A1 (en) * | 2022-10-06 | 2025-04-17 | Solid Biosciences Inc. | Dual transfection vector |
| CN117959464A (zh) * | 2022-10-27 | 2024-05-03 | 苏州新芽基因生物技术有限公司 | 用于疾病治疗的基因编辑器和抗纤维化抑制剂的核酸药物组合物 |
| CN116218849B (zh) * | 2023-01-18 | 2024-01-23 | 昆明理工大学 | 针对utrophin激活治疗杜氏肌营养不良的sgRNA和应用 |
| WO2024220592A2 (en) * | 2023-04-18 | 2024-10-24 | Research Institute At Nationwide Children's Hospital, Inc. | Gene therapy for treating limb girdle muscular dystrophy r9 and congenital muscular dystrophy 1c |
| WO2024229164A2 (en) * | 2023-05-03 | 2024-11-07 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase |
| CN116926125B (zh) * | 2023-09-07 | 2024-06-11 | 昆明理工大学 | 一种抑制炎症与基因编辑同时进行的基因载体 |
| WO2025085731A1 (en) * | 2023-10-18 | 2025-04-24 | Children's National Medical Center | TRANSFORMING GROWTH FACTOR ß (TGF-ß) OLIGONUCLEOTIDE COMBINATION THERAPY FOR TREATING MUSCULOSKELETAL DISEASES |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8314290B2 (en) * | 2004-12-21 | 2012-11-20 | Monsanto Technology Llc | Temporal regulation of gene expression by MicroRNAs |
| CN102311973A (zh) * | 2010-07-05 | 2012-01-11 | 北京五加和分子医学研究所有限公司 | 一种新型miRNA和编码蛋白基因共表达载体 |
| JP2014103905A (ja) * | 2012-11-28 | 2014-06-09 | Tokai Univ | 標的遺伝子のノックダウン方法およびそのためのＲＮＡｉ用発現ベクター |
| EP3097197B1 (en) * | 2014-01-21 | 2020-12-16 | Vrije Universiteit Brussel | Muscle-specific nucleic acid regulatory elements and methods and use thereof |
| KR102244434B1 (ko) * | 2014-08-11 | 2021-04-23 | 삼성전자주식회사 | 재조합 벡터 및 이를 이용한 목적 폴리펩타이드의 생산 방법 |
| AU2016206518B2 (en) * | 2015-01-16 | 2020-03-05 | University Of Washington | Novel micro-dystrophins and related methods of use |
| US10543260B2 (en) * | 2015-07-22 | 2020-01-28 | The Board Of Trustees Of The Leland Stanford Junior University | Replacement gene tolerizing vectors and methods of use thereof |
| WO2017095967A2 (en) * | 2015-11-30 | 2017-06-08 | Duke University | Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use |
| 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 |
| CN110475573A (zh) * | 2017-01-23 | 2019-11-19 | 新泽西鲁特格斯州立大学 | 用于减少肌脂蛋白表达以及预防和治疗肌营养不良症和心肌病的组合物及其使用方法 |
| WO2018200419A1 (en) * | 2017-04-23 | 2018-11-01 | The Trustees Of The University Of Pennsylvania | Viral vectors comprising engineered aav capsids and compositions containing the same |
| EP3697429A1 (en) * | 2017-10-20 | 2020-08-26 | Genethon | Use of syncytin for targeting drug and gene delivery to regenerating muscle tissue |
-
2019
- 2019-12-11 WO PCT/US2019/065718 patent/WO2020123645A1/en unknown
- 2019-12-11 KR KR1020217021723A patent/KR20210124969A/ko active Pending
- 2019-12-11 AU AU2019395388A patent/AU2019395388A1/en active Pending
- 2019-12-11 US US17/312,259 patent/US20220031865A1/en active Pending
- 2019-12-11 SG SG11202105873SA patent/SG11202105873SA/en unknown
- 2019-12-11 CN CN201980091591.9A patent/CN113646004A/zh active Pending
- 2019-12-11 CA CA3123003A patent/CA3123003A1/en active Pending
- 2019-12-11 EP EP19895501.5A patent/EP3893940A4/en active Pending
- 2019-12-11 JP JP2021533367A patent/JP2022513456A/ja active Pending
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