Hm4di mutant and use thereof in preparation of drug for treating parkinson's disease

By using a combination of hM4Di mutant and QTP derivatives, precise targeted regulation of Parkinson's disease was achieved, solving the problems of insufficient targeting and side effects of existing treatments. This significantly improved the motor and balance abilities of PD mice and avoided drug resistance and surgical side effects.

WO2026118958A1 Publication Date: 2026-06-11LIANGZHU LAB
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Patent Information

Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
LIANGZHU LAB
Filing Date
2025-11-25
Publication Date
2026-06-11

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Abstract

The present application relates to an hM4Di mutant and the use thereof in the preparation of a drug for treating Parkinson's disease. Compared with wild-type hM4Di, the hM4Di mutant comprises one or more of amino acid mutations Y439A, Y439G, W435A or W435G. The use comprises the use of an AAV expression vector containing a polynucleotide encoding the hM4Di mutant and an hSyn promoter, in combination with clozapine-N-oxide, in the preparation of a drug for treating Parkinson's disease.
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