Therapeutic Advances for Huntington's Disease

被引:64
|
作者
Kumar, Ashok [1 ]
Kumar, Vijay [2 ]
Singh, Kritanjali [3 ]
Kumar, Sukesh [4 ]
Kim, You-Sam [2 ]
Lee, Yun-Mi [2 ]
Kim, Jong-Joo [2 ]
机构
[1] Sanjay Gandhi Postgrad Inst Med Sci, Dept Genet, Lucknow 226014, Uttar Pradesh, India
[2] Yeungnam Univ, Dept Biotechnol, Gyongsan 38541, Gyeongbuk, South Korea
[3] Swami Vivekanand Subharti Univ, Subharti Med Coll, Cent Res Stn, Meerut 250002, Uttar Pradesh, India
[4] Magadh Univ, Nalanda Coll, PG Dept Bot, Bodh Gaya 824234, Bihar, India
关键词
Huntington's disease; CAG repeat; mutant huntingtin (mHTT); therapeutics; neurodegeneration; TRANSGENIC MOUSE MODEL; PLACEBO-CONTROLLED TRIAL; STRIATAL CELL-DEATH; MUTANT HUNTINGTIN; DOUBLE-BLIND; ANTISENSE OLIGONUCLEOTIDES; IMPROVES MOTOR; COENZYME Q(10); GENE-THERAPY; EICOSAPENTAENOIC ACID;
D O I
10.3390/brainsci10010043
中图分类号
Q189 [神经科学];
学科分类号
071006 ;
摘要
Huntington's disease (HD) is a progressive neurological disease that is inherited in an autosomal fashion. The cause of disease pathology is an expansion of cytosine-adenine-guanine (CAG) repeats within the huntingtin gene (HTT) on chromosome 4 (4p16.3), which codes the huntingtin protein (mHTT). The common symptoms of HD include motor and cognitive impairment of psychiatric functions. Patients exhibit a representative phenotype of involuntary movement (chorea) of limbs, impaired cognition, and severe psychiatric disturbances (mood swings, depression, and personality changes). A variety of symptomatic treatments (which target glutamate and dopamine pathways, caspases, inhibition of aggregation, mitochondrial dysfunction, transcriptional dysregulation, and fetal neural transplants, etc.) are available and some are in the pipeline. Advancement in novel therapeutic approaches include targeting the mutant huntingtin (mHTT) protein and the HTT gene. New gene editing techniques will reduce the CAG repeats. More appropriate and readily tractable treatment goals, coupled with advances in analytical tools will help to assess the clinical outcomes of HD treatments. This will not only improve the quality of life and life span of HD patients, but it will also provide a beneficial role in other inherited and neurological disorders. In this review, we aim to discuss current therapeutic research approaches and their possible uses for HD.
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页数:20
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