TW202216244A - 有用於治療夏馬杜三氏病之組成物 - Google Patents
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- C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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- C12Y—ENZYMES
- C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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| EP4634394A2 (en) | 2022-12-17 | 2025-10-22 | 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 |
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| WO2020047268A1 (en) * | 2018-08-29 | 2020-03-05 | Research Institute At Nationwide Children's Hospital | Products and methods for inhibition of expression of mutant gars protein |
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