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Article

Chemical chaperone therapy: clinical effect in murine G(M1)-gangliosidosis.

Annals of neurology - 1 Dec 2007

Suzuki Yoshiyuki, Ichinomiya Satoshi, Kurosawa Mieko, Ohkubo Masato, Watanabe Hiroshi, Iwasaki Hiroyuki, Matsuda Junichiro, Noguchi Yoko, Takimoto Kazuhiro, Itoh Masayuki, Tabe Miho, Iida Masami, Kubo Takatoshi, Ogawa Seiichiro, Nanba Eiji, Higaki Katsumi, Ohno Kousaku, Brady Roscoe O

Abstract excerpt

Certain low-molecular-weight substrate analogs act both as in vitro competitive inhibitors of lysosomal hydrolases and as intracellular enhancers (chemical chaperones) by stabilization of mutant proteins. In this study, we performed oral administration of a chaperone compound N-octyl-4-epi-beta-v...

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Identifiers

PMID: 17994547
DOI: 10.1002/ana.21284

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 4 Sept 2026. Prepared record publication-17994547-2026-09-04.2. Manifest publication-graph-cohort-2026-09-04.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 4 Sept 2026.

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