2022
DOI: 10.14802/jmd.21006
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Gene Therapy for Huntington’s Disease: The Final Strategy for a Cure?

Abstract: Huntington’s disease (HD) has become a target of the first clinical trials for gene therapy among movement disorders with a genetic origin. More than 100 clinical trials regarding HD have been tried, but all failed, although there were some improvements limited to symptomatic support. Compared to other neurogenetic disorders, HD is known to have a single genetic target. Thus, this is an advantage and its cure is more feasible than any other movement disorder with heterogeneous genetic causes. In this review pa… Show more

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Cited by 15 publications
(9 citation statements)
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“…37 Thus, a second trial (GENERATION-HD2) with lower doses of the medication started recruiting patients aged 25-50 years with prodromal or early-manifest HD to assess dose finding and possible efficacy in this population. 37,38 The single-gene nature of HD makes the prospect of "gene silencing" to decrease the amount of huntingtin produced, an attractive therapeutic option. In addition to antisense oligonucleotides therapy, a number of other genetic techniques have been studied in humans, including RNA interference (RNAi) gene therapy 39 and brain penetrant mRNA small molecule splicing modifiers, 40 among many others.…”
Section: Finding the Genementioning
confidence: 99%
See 1 more Smart Citation
“…37 Thus, a second trial (GENERATION-HD2) with lower doses of the medication started recruiting patients aged 25-50 years with prodromal or early-manifest HD to assess dose finding and possible efficacy in this population. 37,38 The single-gene nature of HD makes the prospect of "gene silencing" to decrease the amount of huntingtin produced, an attractive therapeutic option. In addition to antisense oligonucleotides therapy, a number of other genetic techniques have been studied in humans, including RNA interference (RNAi) gene therapy 39 and brain penetrant mRNA small molecule splicing modifiers, 40 among many others.…”
Section: Finding the Genementioning
confidence: 99%
“…In addition to antisense oligonucleotides therapy, a number of other genetic techniques have been studied in humans, including RNA interference (RNAi) gene therapy 39 and brain penetrant mRNA small molecule splicing modifiers, 40 among many others. Despite more than 100 clinical trials in HD, 38 only symptomatic medications for the disease have been approved in the past three decades, such as Vmat2 inhibitors 41 . Nevertheless, now, we have a better understanding of the pathophysiology of the disease, we have expanded our knowledge of the genetic and epigenetic factors involved, and the curve of ongoing clinical trials is growing exponentially.…”
Section: The Modern Heritage Of Gene Discoverymentioning
confidence: 99%
“…279 CAG repeats in the HTT gene lead to the appearance of repeated segments of glutamine in the translated protein Huntingtin (Htt). 280 This makes the protein more susceptible to misfolding, resulting in protein aggrega-tion. Consequently, the protein undergoes a loss of its basic functionality.…”
Section: Biomedical Applications Of Bioinspired Gene Delivery Systems...mentioning
confidence: 99%
“…DNA repair pathways have been recognized by genome-wide association studies as moderators of somatic instability and disease progression in HD and other repeat expansion diseases . CAG repeats in the HTT gene lead to the appearance of repeated segments of glutamine in the translated protein Huntingtin (Htt) . This makes the protein more susceptible to misfolding, resulting in protein aggregation.…”
Section: Biomedical Applications Of Bioinspired Gene Delivery Systems...mentioning
confidence: 99%
“…Drug-based therapy involves symptomatic treatment through various therapeutic drugs [ 41 ]. HD therapeutic development and advancement that modulates the m HTT level through genetic transfer is one of the most important strategies to ameliorate the disease [ 42 ].…”
Section: Introductionmentioning
confidence: 99%