Huntington's Disease: Current and Future Therapeutic Prospects

被引：26

作者：

Kieburtz, Karl ^{[1
,2
]}

Reilmann, Ralf ^{[3
]}

Olanow, C. Warren ^{[2
,4
]}

机构：

[1] Univ Rochester, Rochester, NY 14627 USA

[2] Clintrex LLC, Sarasota, FL USA

[3] George Huntington Inst, Munster, Germany

[4] Mt Sinai Sch Med, New York, NY USA

来源：

MOVEMENT DISORDERS | 2018年 / 33卷 / 07期

关键词：

Huntington's Disease; Therapy; PLACEBO-CONTROLLED TRIAL; YAC128 MOUSE MODEL; DOUBLE-BLIND; MUTANT HUNTINGTIN; CEREBROSPINAL-FLUID; PARKINSONS-DISEASE; MINIPIG MODEL; CELL MODELS; INHIBITOR; PREMANIFEST;

D O I：

10.1002/mds.27363

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

Huntington's disease is a progressive neurodegenerative disorder for which therapies are woefully inadequate and do not prevent inevitable progression. Currently approved therapies are primarily aimed at treating chorea, but do not address the more clinically meaningful motor, behavioral, and cognitive features of the disease. However, there are a number of promising new therapies that are currently being studied in the laboratory, and in the clinic. This article will review the wide variety of therapies currently being tested, the advances in clinical trials and end points, and the many potentially relevant new targets. (C) 2018 International Parkinson and Movement Disorder Society

引用

页码：1033 / 1041

页数：9

共 92 条

[1] Safety, tolerability, and efficacy of PBT2 in Huntington's disease: a phase 2, randomised, double-blind, placebo-controlled trial [J].

Angus, D. ;

Herd, C. ;

Stone, C. ;

Stout, J. ;

Wieler, M. ;

Reilmann, R. ;

Ritchie, C. W. ;

Dorsey, E. R. ;

Helles, K. ;

Kayson, E. ;

Oakes, D. ;

Rosas, H. D. ;

Vaughan, C. ;

Panegyres, P. K. ;

Ames, D. ;

Goh, A. ;

Agarwal, P. ;

Churchyard, A. ;

Murathodizic, M. ;

Chua, P. ;

Germaine, D. ;

Lim, L. ;

Mack, H. ;

Loy, C. ;

Griffith, J. ;

Mitchell, P. ;

Corey-Bloom, J. ;

Gluhm, S. ;

Goldstein, J. ;

Levi, L. ;

Margolis, R. ;

Yoritomo, N. ;

Janicki, S. ;

Marder, K. ;

Clouse, R. ;

Singer, C. ;

Moore, H. ;

Padron, N. ;

Kostyk, S. ;

Daley, A. ;

Segro, V. ;

Kumar, R. ;

Anderson, K. ;

Drazinic, C. ;

Hennig, B. ;

Nance, M. ;

Molho, E. ;

Criswell, S. ;

LeDoux, M. S. ;

Guyot, S. .

LANCET NEUROLOGY, 2015, 14 (01) :39-47

[2]

[Anonymous], NEUOLOGY

[3] Huntingtin-Lowering Strategies in Huntington's Disease: Antisense Oligonucleotides, Small RNAs, and Gene Editing [J].

Aronin, Neil ;

DiFiglia, Marian .

MOVEMENT DISORDERS, 2014, 29 (11) :1455-1461

[4] Dimebon Slows Progression of Proteinopathy in γ-Synuclein Transgenic Mice [J].

Bachurin, Sergey O. ;

Shelkovnikova, Tatyana A. ;

Ustyugov, Alexey A. ;

Peters, Owen ;

Khritankova, Ina ;

Afanasieva, Marina A. ;

Tarasova, Tatyana V. ;

Alentov, Igor I. ;

Buchman, Vladimir L. ;

Ninkina, Natalia N. .

NEUROTOXICITY RESEARCH, 2012, 22 (01) :33-42

[5] The protective effects of cystamine in the R6/2 Huntington's disease mouse involve mechanisms other than the inhibition of tissue transglutaminase [J].

Bailey, Craig D. C. ;

Johnson, Gail V. W. .

NEUROBIOLOGY OF AGING, 2006, 27 (06) :871-879

[6] Mouse models of triplet repeat diseases [J].

Bates, GP ;

Gonitel, R .

MOLECULAR BIOTECHNOLOGY, 2006, 32 (02) :147-158

[7] A Transgenic Minipig Model of Huntington's Disease [J].

Baxa, Monika ;

Hruska-Plochan, Marian ;

Juhas, Stefan ;

Vodicka, Petr ;

Pavlok, Antonin ;

Juhasova, Jana ;

Miyanohara, Atsushi ;

Nejime, Tetsuya ;

Klima, Jiri ;

Macakova, Monika ;

Marsala, Silvia ;

Weiss, Andreas ;

Kubickova, Svatava ;

Musilova, Petra ;

Vrtel, Radek ;

Sontag, Emily M. ;

Thompson, Leslie M. ;

Schier, Jan ;

Hansikova, Hana ;

Howland, David S. ;

Cattaneo, Elena ;

DiFiglia, Marian ;

Marsala, Martin ;

Motlik, Jan .

JOURNAL OF HUNTINGTONS DISEASE, 2013, 2 (01) :47-68

[8] Tapping linked to function and structure in premanifest and symptomatic Huntington disease [J].

Bechtel, N. ;

Scahill, R. I. ;

Rosas, H. D. ;

Acharya, T. ;

van den Bogaard, S. J. A. ;

Jauffret, C. ;

Say, M. J. ;

Sturrock, A. ;

Johnson, H. ;

Onorato, C. E. ;

Salat, D. H. ;

Durr, A. ;

Leavitt, B. R. ;

Roos, R. A. C. ;

Landwehrmeyer, G. B. ;

Langbehn, D. R. ;

Stout, J. C. ;

Tabrizi, S. J. ;

Reilmann, R. .

NEUROLOGY, 2010, 75 (24) :2150-2160

[9] Nonallele-specific Silencing of Mutant and Wild-type Huntingtin Demonstrates Therapeutic Efficacy in Huntington's Disease Mice [J].

Boudreau, Ryan L. ;

McBride, Jodi L. ;

Martins, Ines ;

Shen, Shihao ;

Xing, Yi ;

Carter, Barrie J. ;

Davidson, Beverly L. .

MOLECULAR THERAPY, 2009, 17 (06) :1053-1063

[10] Pharmacological properties of BN82451: A novel multitargeting neuroprotective agent [J].

Chabrier, Pierre Etienne ;

Auguet, Michel .

CNS DRUG REVIEWS, 2007, 13 (03) :317-332

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