Abstract: The invention provides agents (e.g., antisense oligonucleotides (ASOs), a CRISPR/Cas-based base editing system) capable of increasing expression of RNA-binding motif protein 3 (RBM3) by targeting a poison exon of RBM3, exon 3a, or a splice site thereof. Also disclosed are methods for increasing expression of RBM3 in a cell, methods of treating or preventing a disease affected by RMB3 expression in a subject, or methods for providing neuroprotective treatment to a subject.
| # | Name | Date |
|---|---|---|
| 1 | 202447054848-STATEMENT OF UNDERTAKING (FORM 3) [18-07-2024(online)].pdf | 2024-07-18 |
| 3 | 202447054848-Sequence Listing in PDF [18-07-2024(online)].pdf | 2024-07-18 |
| 4 | 202447054848-REQUEST FOR EXAMINATION (FORM-18) [18-07-2024(online)].pdf | 2024-07-18 |
| 5 | 202447054848-PROOF OF RIGHT [18-07-2024(online)].pdf | 2024-07-18 |
| 6 | 202447054848-PRIORITY DOCUMENTS [18-07-2024(online)].pdf | 2024-07-18 |
| 7 | 202447054848-FORM FOR SMALL ENTITY(FORM-28) [18-07-2024(online)].pdf | 2024-07-18 |
| 8 | 202447054848-FORM 18 [18-07-2024(online)].pdf | 2024-07-18 |
| 9 | 202447054848-FORM 1 [18-07-2024(online)].pdf | 2024-07-18 |
| 10 | 202447054848-EVIDENCE FOR REGISTRATION UNDER SSI(FORM-28) [18-07-2024(online)].pdf | 2024-07-18 |
| 11 | 202447054848-DRAWINGS [18-07-2024(online)].pdf | 2024-07-18 |
| 12 | 202447054848-DECLARATION OF INVENTORSHIP (FORM 5) [18-07-2024(online)].pdf | 2024-07-18 |
| 13 | 202447054848-COMPLETE SPECIFICATION [18-07-2024(online)].pdf | 2024-07-18 |
| 14 | 202447054848-FORM-26 [19-07-2024(online)].pdf | 2024-07-19 |
| 15 | 202447054848-FORM 3 [26-07-2024(online)].pdf | 2024-07-26 |