JP2019536782A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2019536782A5 JP2019536782A5 JP2019528478A JP2019528478A JP2019536782A5 JP 2019536782 A5 JP2019536782 A5 JP 2019536782A5 JP 2019528478 A JP2019528478 A JP 2019528478A JP 2019528478 A JP2019528478 A JP 2019528478A JP 2019536782 A5 JP2019536782 A5 JP 2019536782A5
- Authority
- JP
- Japan
- Prior art keywords
- promoter
- composition according
- composition
- sequence encoding
- present
- 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
- 239000000203 mixture Substances 0.000 claims description 102
- 238000000034 method Methods 0.000 claims description 42
- 239000013598 vector Substances 0.000 claims description 37
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 27
- 229920001184 polypeptide Polymers 0.000 claims description 24
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 24
- 102100024108 Dystrophin Human genes 0.000 claims description 17
- 201000006938 muscular dystrophy Diseases 0.000 claims description 17
- 108010069091 Dystrophin Proteins 0.000 claims description 16
- 108020005004 Guide RNA Proteins 0.000 claims description 16
- 210000004027 cell Anatomy 0.000 claims description 16
- 102000001039 Dystrophin Human genes 0.000 claims description 15
- 210000000663 muscle cell Anatomy 0.000 claims description 11
- 239000013603 viral vector Substances 0.000 claims description 10
- 239000013607 AAV vector Substances 0.000 claims description 6
- 239000011734 sodium Substances 0.000 claims description 6
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 5
- 108010051583 Ventricular Myosins Proteins 0.000 claims description 5
- 230000007547 defect Effects 0.000 claims description 4
- 230000010076 replication Effects 0.000 claims description 4
- 102000007469 Actins Human genes 0.000 claims description 3
- 108010085238 Actins Proteins 0.000 claims description 3
- 101710158332 Diuretic hormone Proteins 0.000 claims description 3
- 101710204261 Diuretic hormone class 2 Proteins 0.000 claims description 3
- DGAQECJNVWCQMB-PUAWFVPOSA-M Ilexoside XXIX Chemical compound C[C@@H]1CC[C@@]2(CC[C@@]3(C(=CC[C@H]4[C@]3(CC[C@@H]5[C@@]4(CC[C@@H](C5(C)C)OS(=O)(=O)[O-])C)C)[C@@H]2[C@]1(C)O)C)C(=O)O[C@H]6[C@@H]([C@H]([C@@H]([C@H](O6)CO)O)O)O.[Na+] DGAQECJNVWCQMB-PUAWFVPOSA-M 0.000 claims description 3
- 102100026925 Myosin regulatory light chain 2, ventricular/cardiac muscle isoform Human genes 0.000 claims description 3
- 101000987816 Triticum aestivum 16.9 kDa class I heat shock protein 1 Proteins 0.000 claims description 3
- 102000013640 alpha-Crystallin B Chain Human genes 0.000 claims description 3
- 108010051585 alpha-Crystallin B Chain Proteins 0.000 claims description 3
- 230000002490 cerebral effect Effects 0.000 claims description 3
- 210000005260 human cell Anatomy 0.000 claims description 3
- 230000001939 inductive effect Effects 0.000 claims description 3
- 102000006495 integrins Human genes 0.000 claims description 3
- 108010044426 integrins Proteins 0.000 claims description 3
- 239000002502 liposome Substances 0.000 claims description 3
- 108010065781 myosin light chain 2 Proteins 0.000 claims description 3
- 239000002105 nanoparticle Substances 0.000 claims description 3
- 239000013612 plasmid Substances 0.000 claims description 3
- 229910052708 sodium Inorganic materials 0.000 claims description 3
- 241000604451 Acidaminococcus Species 0.000 claims description 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 2
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims description 2
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 2
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 2
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims description 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 2
- 241000649045 Adeno-associated virus 10 Species 0.000 claims description 2
- 241000649046 Adeno-associated virus 11 Species 0.000 claims description 2
- 241000702421 Dependoparvovirus Species 0.000 claims description 2
- 108700024394 Exon Proteins 0.000 claims description 2
- 241001112693 Lachnospiraceae Species 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 125000003729 nucleotide group Chemical group 0.000 claims description 2
- 238000003776 cleavage reaction Methods 0.000 claims 2
- 238000012224 gene deletion Methods 0.000 claims 1
- 208000024891 symptom Diseases 0.000 description 10
- 210000003205 muscle Anatomy 0.000 description 9
- 239000003550 marker Substances 0.000 description 6
- 210000001087 myotubule Anatomy 0.000 description 6
- 210000001519 tissue Anatomy 0.000 description 4
- 230000000926 neurological effect Effects 0.000 description 3
- 230000000750 progressive effect Effects 0.000 description 3
- 208000010428 Muscle Weakness Diseases 0.000 description 2
- 206010028372 Muscular weakness Diseases 0.000 description 2
- 206010033799 Paralysis Diseases 0.000 description 2
- 230000000735 allogeneic effect Effects 0.000 description 2
- 101150015424 dmd gene Proteins 0.000 description 2
- 238000002347 injection Methods 0.000 description 2
- 239000007924 injection Substances 0.000 description 2
- 210000002966 serum Anatomy 0.000 description 2
- 201000006347 Intellectual Disability Diseases 0.000 description 1
- 208000034819 Mobility Limitation Diseases 0.000 description 1
- 108091034117 Oligonucleotide Proteins 0.000 description 1
- 108020005067 RNA Splice Sites Proteins 0.000 description 1
- 208000000875 Spinal Curvatures Diseases 0.000 description 1
- 230000002159 abnormal effect Effects 0.000 description 1
- 230000014461 bone development Effects 0.000 description 1
- 230000009194 climbing Effects 0.000 description 1
- 230000007812 deficiency Effects 0.000 description 1
- 238000003745 diagnosis Methods 0.000 description 1
- 230000001747 exhibiting effect Effects 0.000 description 1
- 230000002068 genetic effect Effects 0.000 description 1
- 210000005003 heart tissue Anatomy 0.000 description 1
- 238000000338 in vitro Methods 0.000 description 1
- 238000001727 in vivo Methods 0.000 description 1
- 238000011065 in-situ storage Methods 0.000 description 1
- 238000001802 infusion Methods 0.000 description 1
- 238000010255 intramuscular injection Methods 0.000 description 1
- 239000007927 intramuscular injection Substances 0.000 description 1
- 238000001990 intravenous administration Methods 0.000 description 1
- 230000007775 late Effects 0.000 description 1
- 210000004962 mammalian cell Anatomy 0.000 description 1
- 108020004999 messenger RNA Proteins 0.000 description 1
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 230000035772 mutation Effects 0.000 description 1
- 210000004197 pelvis Anatomy 0.000 description 1
- 239000002243 precursor Substances 0.000 description 1
- 210000003314 quadriceps muscle Anatomy 0.000 description 1
- 210000002027 skeletal muscle Anatomy 0.000 description 1
- 210000001057 smooth muscle myoblast Anatomy 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662426853P | 2016-11-28 | 2016-11-28 | |
| US62/426,853 | 2016-11-28 | ||
| PCT/US2017/063468 WO2018098480A1 (en) | 2016-11-28 | 2017-11-28 | Prevention of muscular dystrophy by crispr/cpf1-mediated gene editing |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019536782A JP2019536782A (ja) | 2019-12-19 |
| JP2019536782A5 true JP2019536782A5 (OSRAM) | 2021-01-14 |
Family
ID=60703123
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019528478A Pending JP2019536782A (ja) | 2016-11-28 | 2017-11-28 | ＣＲＩＳＰＲ／Ｃｐｆ１媒介性遺伝子編集による筋ジストロフィーの予防 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20200046854A1 (OSRAM) |
| EP (1) | EP3545090A1 (OSRAM) |
| JP (1) | JP2019536782A (OSRAM) |
| CN (1) | CN110382695A (OSRAM) |
| AU (1) | AU2017364106A1 (OSRAM) |
| CA (1) | CA3044531A1 (OSRAM) |
| MX (1) | MX2019006157A (OSRAM) |
| WO (1) | WO2018098480A1 (OSRAM) |
Families Citing this family (65)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2734621B1 (en) | 2011-07-22 | 2019-09-04 | President and Fellows of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| EP2841572B1 (en) | 2012-04-27 | 2019-06-19 | Duke University | Genetic correction of mutated genes |
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | President And Fellows Of Harvard College | Methods for identifying a target site of a cas9 nuclease |
| US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9340800B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | Extended DNA-sensing GRNAS |
| US9840699B2 (en) | 2013-12-12 | 2017-12-12 | President And Fellows Of Harvard College | Methods for nucleic acid editing |
| WO2016022363A2 (en) | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| WO2016130600A2 (en) | 2015-02-09 | 2016-08-18 | Duke University | Compositions and methods for epigenome editing |
| WO2017066497A2 (en) | 2015-10-13 | 2017-04-20 | Duke University | Genome engineering with type i crispr systems in eukaryotic cells |
| IL258821B (en) | 2015-10-23 | 2022-07-01 | Harvard College | Nucleobase editors and their uses |
| ES2905558T3 (es) | 2015-11-13 | 2022-04-11 | Avellino Lab Usa Inc | Procedimientos para el tratamiento de las distrofias corneales |
| AU2016362282B2 (en) | 2015-11-30 | 2023-03-16 | Duke University | Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use |
| US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| EP4275747A3 (en) * | 2016-07-19 | 2024-01-24 | Duke University | Therapeutic applications of cpf1-based genome editing |
| KR20250103795A (ko) | 2016-08-03 | 2025-07-07 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| CA3033327A1 (en) | 2016-08-09 | 2018-02-15 | President And Fellows Of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| WO2020225754A1 (en) * | 2019-05-06 | 2020-11-12 | Mcmullen Tara | Crispr gene editing for autosomal dominant diseases |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| JP7588390B2 (ja) | 2016-10-14 | 2024-11-22 | プレジデント アンド フェローズ オブ ハーバード カレッジ | 核酸塩基エディターのａａｖ送達 |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| WO2018165504A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| CN110662556A (zh) | 2017-03-09 | 2020-01-07 | 哈佛大学的校长及成员们 | 癌症疫苗 |
| JP2020510439A (ja) | 2017-03-10 | 2020-04-09 | プレジデント アンド フェローズ オブ ハーバード カレッジ | シトシンからグアニンへの塩基編集因子 |
| BR112019019655A2 (pt) | 2017-03-23 | 2020-04-22 | Harvard College | editores de nucleobase que compreendem proteínas de ligação a dna programáveis por ácido nucleico |
| KR102758434B1 (ko) * | 2017-03-30 | 2025-01-21 | 고쿠리츠 다이가쿠 호진 교토 다이가쿠 | 게놈 편집에 의한 엑손 스키핑 유도 방법 |
| WO2018209320A1 (en) | 2017-05-12 | 2018-11-15 | President And Fellows Of Harvard College | Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation |
| JP2020534795A (ja) | 2017-07-28 | 2020-12-03 | プレジデント アンド フェローズ オブ ハーバード カレッジ | ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物 |
| US11319532B2 (en) | 2017-08-30 | 2022-05-03 | President And Fellows Of Harvard College | High efficiency base editors comprising Gam |
| KR20250107288A (ko) | 2017-10-16 | 2025-07-11 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| WO2020028832A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| WO2020122104A1 (ja) * | 2018-12-11 | 2020-06-18 | 国立大学法人京都大学 | ゲノムｄｎａに欠失を誘導する方法 |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| CA3127527A1 (en) * | 2019-02-12 | 2020-08-20 | Universita Degli Studi Di Trento | Cas12a guide rna molecules and uses thereof |
| EP3942042A1 (en) | 2019-03-19 | 2022-01-26 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| EP3952884A4 (en) * | 2019-04-12 | 2023-03-22 | Duke University | Crispr/cas-based base editing composition for restoring dystrophin function |
| EP3956349A1 (en) | 2019-04-17 | 2022-02-23 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| CN110499333A (zh) * | 2019-08-01 | 2019-11-26 | 广州德赫生物科技有限公司 | 用于修复dmd基因突变的核酸序列及系统 |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| CA3172542A1 (en) * | 2020-03-04 | 2021-09-10 | Regeneron Pharmaceuticals, Inc. | A rodent model of b4galt1-mediated functions |
| WO2021216674A1 (en) * | 2020-04-24 | 2021-10-28 | University Of Massachusetts | Improved cas 12a/nls mediated therapeutic gene editing platforms |
| WO2021226558A1 (en) | 2020-05-08 | 2021-11-11 | The Broad Institute, Inc. | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| WO2021231575A1 (en) * | 2020-05-12 | 2021-11-18 | Myogene Bio Llc | Immunosuppressive agents and viral delivery re-dosing methods for gene therapy |
| TW202218686A (zh) * | 2020-09-09 | 2022-05-16 | 美商維泰克斯製藥公司 | 用於治療杜興氏肌肉失養症(duchenne muscular dystrophy)之組合物及方法 |
| WO2022140340A1 (en) * | 2020-12-22 | 2022-06-30 | Vertex Pharmaceuticals Incorporated | Compositions comprising an rna guide targeting dmd and uses thereof |
| US20240226334A1 (en) * | 2021-05-25 | 2024-07-11 | The Board Of Regents Of The University Of Texas System | Correction of duchenne muscular dystrophy mutations with all-in-one adeno-associated virus-delivered single-cut crispr |
| EP4366784A1 (en) | 2021-07-09 | 2024-05-15 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating dystrophinopathies |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| KR20240035826A (ko) * | 2021-07-09 | 2024-03-18 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 디스트로핀병증을 치료하기 위한 그의 용도 |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| CN114214360A (zh) * | 2021-12-27 | 2022-03-22 | 西安英创生物技术有限公司 | 先天性肌无力小鼠模型、其构建方法及应用 |
| EP4215614A1 (en) | 2022-01-24 | 2023-07-26 | Dynacure | Combination therapy for dystrophin-related diseases |
| EP4490292A1 (en) * | 2022-03-08 | 2025-01-15 | Vertex Pharmaceuticals Incorporated | Precise excisions of portions of exon 44, 50, and 53 for treatment of duchenne muscular dystrophy |
| CN119731321A (zh) | 2022-06-24 | 2025-03-28 | 图恩疗法股份有限公司 | 通过靶向基因阻遏减少低密度脂蛋白的组合物、系统和方法 |
| CN120648680A (zh) * | 2024-03-15 | 2025-09-16 | 迦进生物医药(上海)有限公司 | 治疗杜兴型肌营养不良症和贝克型肌营养不良症的药物 |
| CN118086408A (zh) * | 2024-04-25 | 2024-05-28 | 华南理工大学 | 一种可实现靶向肌肉细胞基因编辑的仿病毒颗粒及其制备方法和应用 |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK99653C (da) | 1959-06-24 | 1964-08-31 | Skaanska Aettikfabriken Ab | Pynteliste til brug som sammenfojningsliste, hjørneliste eller endeliste ved montering af beklædningsplader. |
| EP0273085A1 (en) | 1986-12-29 | 1988-07-06 | IntraCel Corporation | A method for internalizing nucleic acids into eukaryotic cells |
| SMT201900159T1 (it) * | 2008-10-24 | 2019-05-10 | Sarepta Therapeutics Inc | Composizioni di salto di esone per dmd |
| SI3401400T1 (sl) | 2012-05-25 | 2019-10-30 | Univ California | Postopki in sestavki za RNA usmerjeno modifikacijo tarčne DNA in za RNA usmerjeno modulacijo prepisovanja |
| HK1220154A1 (zh) * | 2013-03-15 | 2017-04-28 | Sarepta Therapeutics, Inc. | 改进的用於治疗肌营养不良的组合物 |
| KR20230136697A (ko) * | 2013-06-05 | 2023-09-26 | 듀크 유니버시티 | Rna-가이드 유전자 편집 및 유전자 조절 |
| DK3116533T3 (da) * | 2014-03-12 | 2020-08-24 | Prec Biosciences Inc | Deletion af dystrophingenexon under anvendelse af genmanipulerede nukleaser |
| CA2959130A1 (en) * | 2014-08-11 | 2016-02-18 | The Board Of Regents Of The University Of Texas System | Prevention of muscular dystrophy by crispr/cas9-mediated gene editing |
| WO2016025468A2 (en) * | 2014-08-11 | 2016-02-18 | The Board Of Trustees Of The University Of Illinois | Devices and related methods for epidermal characterization of biofluids |
| US9790490B2 (en) * | 2015-06-18 | 2017-10-17 | The Broad Institute Inc. | CRISPR enzymes and systems |
| KR102699944B1 (ko) * | 2015-08-25 | 2024-09-13 | 듀크 유니버시티 | Rna-가이드된 엔도뉴클레아제를 이용하는 게놈 조작에서 특이성을 개선하는 조성물 및 방법 |
| CA2996982A1 (en) * | 2015-09-23 | 2017-03-30 | Universite Laval | Modification of the dystrophin gene and uses thereof |
| CN108513546A (zh) * | 2015-10-28 | 2018-09-07 | 克里斯珀医疗股份公司 | 用于治疗杜氏肌营养不良症的材料和方法 |
| EP4275747A3 (en) * | 2016-07-19 | 2024-01-24 | Duke University | Therapeutic applications of cpf1-based genome editing |
-
2017
- 2017-11-28 WO PCT/US2017/063468 patent/WO2018098480A1/en not_active Ceased
- 2017-11-28 AU AU2017364106A patent/AU2017364106A1/en not_active Abandoned
- 2017-11-28 CN CN201780084906.8A patent/CN110382695A/zh active Pending
- 2017-11-28 US US16/464,124 patent/US20200046854A1/en not_active Abandoned
- 2017-11-28 JP JP2019528478A patent/JP2019536782A/ja active Pending
- 2017-11-28 CA CA3044531A patent/CA3044531A1/en not_active Abandoned
- 2017-11-28 MX MX2019006157A patent/MX2019006157A/es unknown
- 2017-11-28 EP EP17817498.3A patent/EP3545090A1/en not_active Withdrawn
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2019536782A5 (OSRAM) | ||
| JP2020503869A5 (OSRAM) | ||
| CN109069672B (zh) | 腺相关病毒载体传递微肌营养不良蛋白以治疗肌营养不良症 | |
| ES2535877T3 (es) | Genes de Factor VIII y Factor IX modificados y vectores para la terapia génica | |
| JP2023503637A (ja) | マイクロジストロフィン遺伝子治療コンストラクト及びその使用 | |
| JP2020513811A5 (OSRAM) | ||
| JP2018515096A (ja) | 神経成長因子シグナルペプチド及び副甲状腺ホルモンを含むａａｖ分離株及び融合タンパク質 | |
| EP2933335A1 (en) | A method of treating peripheral neuropathies and motor neuron diseases | |
| JP7541027B2 (ja) | 筋肉発現のためのハイブリッドプロモーター | |
| JP2020513831A (ja) | ＭｅＣＰ２発現カセット | |
| AU2016355343A1 (en) | Compositions and methods for correction of heritable ocular disease | |
| ES2946415T3 (es) | Mini-GDE para el tratamiento de la enfermedad de almacenamiento de glucógeno III | |
| JP7671790B2 (ja) | 眼疾患の遺伝子治療 | |
| CN119137273A (zh) | 包括工程化核内小RNA（snRNA）的组合物和方法 | |
| JP2025093922A (ja) | Ｃｌｎ６ポリヌクレオチドのアデノ随伴ウイルス送達 | |
| JP2022519596A (ja) | Ｃｌｎ３ポリヌクレオチドのアデノ随伴ウイルス送達 | |
| US20250230443A1 (en) | Products and methods for inhibition of expression of dynamin-1 variants | |
| EP4536686B1 (en) | Peptide-modified aav capsid with enhanced muscle transduction efficiency | |
| AU2020366242A1 (en) | Materials and methods for the treatment of disorders associated mutations in the IRF2BPL gene | |
| AU2020385387A1 (en) | Materials and methods for treatment of disorders associated with the IGHMBP2 gene | |
| WO2025232917A1 (en) | Novel liver detargeted aav variants and uses thereof | |
| TW202449145A (zh) | 具有改良負載基因表現率及降低基因毒性之腺相關病毒之經修飾複合體平台 | |
| JP2025534666A (ja) | 操作された核酸調節エレメントならびにその使用方法 | |
| WO2025023319A1 (ja) | 脳指向性ａａｖ変異体 | |
| JP2024515612A (ja) | 球脊髄性筋萎縮症（ｓｂｍａ）の治療に有用な組成物 |