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A Bioinspired Platform for Effective Delivery of Protein Therapeutics to the Central Nervous System.

ABSTRACT: Central nervous system (CNS) diseases are the leading cause of morbidity and mortality; their treatment, however, remains constrained by the blood-brain barrier (BBB) that impedes the access of most therapeutics to the brain. A CNS delivery platform for protein therapeutics, which is achieved by encapsulating the proteins within nanocapsules that contain choline and acetylcholine analogues, is reported herein. Mediated by nicotinic acetylcholine receptors and choline transporters, such nanocapsules can effectively penetrate the BBB and deliver the therapeutics to the CNS, as demonstrated in mice and non-human primates. This universal platform, in general, enables the delivery of any protein therapeutics of interest to the brain, opening a new avenue for the treatment of CNS diseases.

SUBMITTER: Wu D 

PROVIDER: S-EPMC6701476 | biostudies-literature | 2019 May

REPOSITORIES: biostudies-literature

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A Bioinspired Platform for Effective Delivery of Protein Therapeutics to the Central Nervous System.

Wu Di D   Qin Meng M   Xu Duo D   Wang Lan L   Liu Chaoyong C   Ren Jie J   Zhou George G   Chen Chen C   Yang Fengmei F   Li Yanyan Y   Zhao Yuan Y   Huang Ruyi R   Pourtaheri Sina S   Kang Chunsheng C   Kamata Masakazu M   Chen Irvin S Y ISY   He Zhanlong Z   Wen Jing J   Chen Wei W   Lu Yunfeng Y  

Advanced materials (Deerfield Beach, Fla.) 20190225 18

Central nervous system (CNS) diseases are the leading cause of morbidity and mortality; their treatment, however, remains constrained by the blood-brain barrier (BBB) that impedes the access of most therapeutics to the brain. A CNS delivery platform for protein therapeutics, which is achieved by encapsulating the proteins within nanocapsules that contain choline and acetylcholine analogues, is reported herein. Mediated by nicotinic acetylcholine receptors and choline transporters, such nanocapsu  ...[more]

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