CA3185281A1 - Compositions useful for treatment of charcot-marie-tooth disease - Google Patents
Compositions useful for treatment of charcot-marie-tooth diseaseInfo
- Publication number
- CA3185281A1 CA3185281A1 CA3185281A CA3185281A CA3185281A1 CA 3185281 A1 CA3185281 A1 CA 3185281A1 CA 3185281 A CA3185281 A CA 3185281A CA 3185281 A CA3185281 A CA 3185281A CA 3185281 A1 CA3185281 A1 CA 3185281A1
- Authority
- CA
- Canada
- Prior art keywords
- seq
- sequence
- mirna
- coding sequence
- 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.)
- Pending
Links
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- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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| US202163173045P | 2021-04-09 | 2021-04-09 | |
| US63/173,045 | 2021-04-09 | ||
| PCT/US2021/041406 WO2022015715A1 (en) | 2020-07-13 | 2021-07-13 | Compositions useful for treatment of charcot-marie-tooth disease |
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| WO2023049846A1 (en) * | 2021-09-24 | 2023-03-30 | The Trustees Of The University Of Pennsylvania | Compositions useful for treatment of charcot-marie-tooth disease |
| WO2024130070A2 (en) | 2022-12-17 | 2024-06-20 | The Trustees Of The University Of Pennsylvania | Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof |
| WO2024130067A2 (en) | 2022-12-17 | 2024-06-20 | The Trustees Of The University Of Pennsylvania | Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same |
| WO2024155966A1 (en) * | 2023-01-20 | 2024-07-25 | Loma Linda University Health | Methods and compositions for treatment of niemann-pick disease type c and charcot-marie-tooth disease |
| WO2024261139A1 (en) | 2023-06-21 | 2024-12-26 | Fundació Institut De Recerca Biomèdica (Irb Barcelona) | Variants of mfn2 and their use in the treatment/prevention of diseases associated with alterations in endoplasmatic reticulum function |
| AR133098A1 (es) | 2023-06-29 | 2025-08-27 | Univ Pennsylvania | Aav mutante con motivos dirigidos al sistema nervioso central y composiciones que lo contienen |
| WO2025106661A1 (en) | 2023-11-14 | 2025-05-22 | The Trustees Of The University Of Pennsylvania | Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof |
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| MX346493B (es) | 2001-11-13 | 2017-03-21 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| JP4810062B2 (ja) | 2001-12-17 | 2011-11-09 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | アデノ随伴ウイルス（ａａｖ）血清型８の配列 |
| CN102199626B (zh) | 2003-09-30 | 2015-06-24 | 宾夕法尼亚大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
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| CN104910025B (zh) | 2008-11-07 | 2019-07-16 | 麻省理工学院 | 氨基醇类脂质和其用途 |
| EP2553106A2 (en) | 2010-03-29 | 2013-02-06 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| EP2558074B1 (en) | 2010-04-08 | 2018-06-06 | The Trustees of Princeton University | Preparation of lipid nanoparticles |
| WO2012075040A2 (en) | 2010-11-30 | 2012-06-07 | Shire Human Genetic Therapies, Inc. | mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES |
| EP2717893B1 (en) | 2011-06-08 | 2019-05-08 | Translate Bio, Inc. | Lipid nanoparticle compositions and methods for mrna delivery |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
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| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
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| EP3387118B1 (en) | 2015-12-11 | 2022-04-06 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aavrh10 |
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| CN118956781A (zh) | 2017-02-28 | 2024-11-15 | 宾夕法尼亚州大学信托人 | 腺相关病毒(aav)进化枝f载体及其用途 |
| WO2018168956A1 (ja) | 2017-03-16 | 2018-09-20 | 株式会社デンソー | 自己位置推定装置 |
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| BR112023000578A2 (pt) | 2023-05-09 |
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