HIGHLIGHTS
- who: Advance access, publication and March, from the Huashan Hospital Fudan University, Shanghai, China have published the research: Protein & Cell, in the Journal: (JOURNAL) of 13/03/2023
- what: Unlike the ASO-based gene therapy approaches that require multiple intrathecal injections per year, this study showed that a single peripheral injection could potentially treat HD, providing a much more desirable scenario for potential clinical application; (iii) Potential safety issues were systematically evaluated, including deep-sequencing and whole-genome sequencing that reveal minimal off-target effects and strong animal safety data that encourages further clinical trials . . .
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