Protein therapy for Unverricht-Lundborg disease using cystatin B transduction by TAT-PTD - Is it that simple?

被引：6

作者：

Andrade, Danielle M.

Scherer, Stephen W.

Minassian, Berge A.

机构：

[1] Hosp Sick Children, Program Genet & Genom Biol, Res Inst, Toronto, ON M5G 1X8, Canada

[2] Univ Toronto, Dept Mol & Med Genet, Toronto, ON, Canada

[3] Hosp Sick Children, Div Neurol, Dept Paediat, Toronto, ON M5G 1X8, Canada

[4] Hosp Sick Children, Dept Genet, Toronto, ON M5G 1X8, Canada

来源：

EPILEPSY RESEARCH | 2006年 / 72卷 / 01期

基金：

加拿大健康研究院;

关键词：

progressive myoclonus epilepsy; Unverrricht-Lundborg; protein therapy; blood-brain-barrier; cell-permeable peptide; TAT-PTD;

D O I：

10.1016/j.eplepsyres.2006.07.009

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

In this work we analysed the characteristics of the cell-permeable peptide TAT-PTD fused to cystatin B (CSTB) to evaluate its potential for protein therapy of Unverricht-Lundborg (UL) epilepsy. TAT-PTD-CSTB does not penetrate the cells despite initial evidence of time and concentration-dependent transduction. Therefore, it cannot be used as a form of replacement of the intracytoplasmic protein missing in UL. Importantly, we discuss precautions to avoid false-positive results when working with TAT-PTD for protein therapy of neurological diseases. (c) 2006 Elsevier B.V. All rights reserved.

引用

页码：75 / 79

页数：5

共 12 条

[1] HIV-1 Tat protein stimulates in vivo vascular permeability and lymphomononuclear cell recruitment
Arese, M
Ferrandi, C
Primo, L
Camussi, G
Bussolino, F
[J]. JOURNAL OF IMMUNOLOGY, 2001, 166 (02) : 1380 - 1388
[2] TAT-mediated protein transduction into mammalian cells
Becker-Hapak, M
McAllister, SS
Dowdy, SF
[J]. METHODS, 2001, 24 (03) : 247 - 256
[3] COLIGAN J, 1995, CURRENT PROTOCOLS PR, V1, P1
[4] KOSKINIEMI M, 1974, ACTA NEUROL SCAND, V50, P307
[5] Unstable insertion in the 5' flanking region of the cystatin B gene is the most common mutation in progressive myoclonus epilepsy type 1, EPM1
Lafreniere, RG
Rochefort, DL
Chretien, N
Rommens, JM
Cochius, JI
Kalviainen, R
Nousiainen, U
Patry, G
Farrell, K
Soderfeldt, B
Federico, A
Hale, BR
Cossio, OH
Sorensen, T
Pouliot, MA
Kmiec, T
Uldall, P
Janszky, J
Pranzatelli, MR
Andermann, F
Andermann, E
Rouleau, GA
[J]. NATURE GENETICS, 1997, 15 (03) : 298 - 302
[6] LOCALIZATION OF THE EPM1 GENE FOR PROGRESSIVE MYOCLONUS EPILEPSY ON CHROMOSOME-21 - LINKAGE DISEQUILIBRIUM ALLOWS HIGH-RESOLUTION MAPPING
LEHESJOKI, AE
KOSKINIEMI, M
NORIO, R
TIRRITO, S
SISTONEN, P
LANDER, E
DELACHAPELLE, A
[J]. HUMAN MOLECULAR GENETICS, 1993, 2 (08) : 1229 - 1234
[7] Positively charged DNA-binding proteins cause apparent cell membrane translocation
Lundberg, M
Johansson, M
[J]. BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, 2002, 291 (02) : 367 - 371
[8] Minassian BA, 2001, ANN NEUROL, V49, P271, DOI 10.1002/1531-8249(20010201)49:2<271::AID-ANA52>3.0.CO
[9] 2-D
[10] Mutations in the gene encoding cystatin B in progressive myoclonus epilepsy (EPM1)
Pennacchio, LA
Lehesjoki, AE
Stone, NE
Willour, VL
Virtaneva, K
Miao, JM
DAmato, E
Ramirez, L
Faham, M
Koskiniemi, M
Warrington, JA
Norio, R
delaChapelle, A
Cox, DR
Myers, RM
[J]. SCIENCE, 1996, 271 (5256) : 1731 - 1734

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