method
TW202633664APending Publication Date: 2026-08-16SANIA RX LTD
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Patent Information
- Application Number
- TW114143349
- Authority / Receiving Office
- TW · TW
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2025-11-05
- Filing Date
- 2025-11-06
- Publication Date
- 2026-08-16
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Figure TWG2TA001072927_001 
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Abstract
The present disclosure relates to a chemogenetic system and method treating BMD using a gene therapy vector to deliver a therapeutic payload to specific cells (LUT neurons). Overexpression of the payload increases the sensitivity of the cells to an activator, thereby enabling lower doses of activator drugs to be administered. The disclosure also relates to vectors to deliver a nucleotide sequence to the target cells to enable overexpression thereof.
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