PMID- 22620966
OWN - NLM
STAT- MEDLINE
DCOM- 20120921
LR  - 20120524
IS  - 1744-2818 (Electronic)
IS  - 1350-6129 (Linking)
VI  - 19 Suppl 1
DP  - 2012 Jun
TI  - Potential use of glucuronylglucosyl-beta-cyclodextrin as a novel therapeutic tool 
      for familial amyloidotic polyneuropathy.
PG  - 50-2
LID - 10.3109/13506129.2012.674988 [doi]
AB  - Transthyretin (TTR)-related familial amyloidotic polyneuropathy, which is induced 
      by amyloidogenic transthyretin (ATTR), is characterized by systemic accumulation 
      of amyloid fibrils. Although it is believed that protein misfolding of monomeric 
      form of TTR is a rate-limiting step for TTR amyloid formation, no effective 
      therapy targeting this misfolding step is available. Our recent studies revealed 
      that cyclodextrins (CyDs), cyclic oligosaccharides composed of glucose units, 
      might interact with TTR and prevent the protein misfolding. In this study, we 
      focused on and elucidated the inhibitory effect of 
      6-O-alpha-(4-O-alpha-D-Glucuronyl)-D-glucosyl-beta-CyD (GUG-beta-CyD) on TTR amyloid formation. 
      Tryptophan (Trp) fluorescence and (1)H-NMR spectroscopy analyses indicated that 
      GUG-beta-CyD stabilized TTR conformation via interaction with the hydrophobic amino 
      acids of TTR. Moreover, GUG-beta-CyD suppressed TTR deposition in transgenic rats 
      possessing a human ATTR V30M gene in vivo. Collectively, these data indicate that 
      GUG-beta-CyD may inhibit TTR misfolding by stabilizing its conformation, which, in 
      turn, suppresses TTR amyloid formation.
FAU - Jono, Hirofumi
AU  - Jono H
AD  - Department of Diagnostic Medicine, Kumamoto University, Kumamoto, Japan. 
      hjono@fc.kuh.kumamoto-u.ac.jp
FAU - Anno, Takayuki
AU  - Anno T
FAU - Motoyama, Keiichi
AU  - Motoyama K
FAU - Misumi, Yohei
AU  - Misumi Y
FAU - Tasaki, Masayoshi
AU  - Tasaki M
FAU - Oshima, Toshinori
AU  - Oshima T
FAU - Mori, Yoshimasa
AU  - Mori Y
FAU - Mizuguchi, Mineyuki
AU  - Mizuguchi M
FAU - Ueda, Mitsuharu
AU  - Ueda M
FAU - Shinriki, Satoru
AU  - Shinriki S
FAU - Shono, Makoto
AU  - Shono M
FAU - Obayashi, Konen
AU  - Obayashi K
FAU - Arima, Hidetoshi
AU  - Arima H
FAU - Ando, Yukio
AU  - Ando Y
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PL  - England
TA  - Amyloid
JT  - Amyloid : the international journal of experimental and clinical investigation : 
      the official journal of the International Society of Amyloidosis
JID - 9433802
RN  - 0 (Oligosaccharides)
RN  - 0 (Prealbumin)
RN  - 0 (cyclomaltoheptaosyl-(6-1)-glucopyranosyl-(4-1)-glucopyranosiduronic acid)
SB  - IM
MH  - Amyloid Neuropathies, Familial/*drug therapy/*metabolism
MH  - Animals
MH  - Humans
MH  - Models, Theoretical
MH  - Oligosaccharides/*metabolism/*therapeutic use
MH  - Prealbumin/*metabolism
MH  - Protein Folding/drug effects
MH  - Rats
MH  - Rats, Transgenic
EDAT- 2012/06/01 06:00
MHDA- 2012/09/22 06:00
CRDT- 2012/05/25 06:00
PHST- 2012/05/25 06:00 [entrez]
PHST- 2012/06/01 06:00 [pubmed]
PHST- 2012/09/22 06:00 [medline]
AID - 10.3109/13506129.2012.674988 [doi]
PST - ppublish
SO  - Amyloid. 2012 Jun;19 Suppl 1:50-2. doi: 10.3109/13506129.2012.674988.