Abstract：
:Trials using antisense oligonucleotide technology to lower Huntingtin levels in Huntington's disease (HD) are currently ongoing. This progress, taking place only 27 years after the identification of the Huntingtin gene (HTT) in 1993 reflects the enormous development in genetic engineering in the last decades. It is also the result of passionate basic scientific work and large worldwide registry studies that have advanced the understanding of HD. Increased knowledge of the pathophysiology of this autosomal dominantly inherited CAG-repeat expansion mediated neurodegenerative disease has led to the development of several putative treatment strategies, currently under investigation. These strategies span the whole spectrum of potential targets from genome editing via RNA interference to promoting protein degradation. Yet, recent studies revealed the importance of huntingtin RNA in the pathogenesis of the disease. Therefore, huntingtin-lowering by means of RNA interference appears to be a particular promising strategy. As a matter of fact, these approaches have entered, or are on the verge of entering, the clinical trial period. Here, we provide an overview of huntingtin-lowering approaches via DNA or RNA interference in present clinical trials as well as strategies subject to upcoming therapeutic options. We furthermore discuss putative implications for future treatment of HD patients.
journal_name
Int J Mol Scijournal_title
International journal of molecular sciencesauthors
Marxreiter F,Stemick J,Kohl Zdoi
10.3390/ijms21062146subject
Has Abstractpub_date
2020-03-20 00:00:00issue
6issn
1422-0067pii
ijms21062146journal_volume
21pub_type
杂志文章,评审abstract：:The role of the gut microbiome in human health and disease is the focus of much attention. It has been widely agreed upon that our gut bacteria play a role in host immunity, nutrient absorption, digestion, metabolism, and other key drivers of health. Furthermore, certain microbial signatures and specific taxa have als...
journal_title：International journal of molecular sciences
pub_type： 杂志文章,评审
doi：10.3390/ijms21030924
更新日期：2020-01-30 00:00:00
abstract：:The insulin signaling (IIS) pathway plays an important role in the metabolism, growth, development, reproduction, and longevity of an organism. As a key member of the IIS pathway, insulin-like growth factor binding proteins (IGFBPs) are widely distributed a family in invertebrates and vertebrates that are critical in ...
journal_title：International journal of molecular sciences
pub_type： 杂志文章
doi：10.3390/ijms22031056
更新日期：2021-01-21 00:00:00
abstract：:The nuclear retinoic acid receptors (RARs) function as ligand-dependent transcriptional regulators and include three subtypes (RARα, RARβ and RARγ), which control the expression of specific gene subsets subsequent to ligand binding and to strictly controlled phosphorylation processes. Extracellular matrix (ECM) accumu...
journal_title：International journal of molecular sciences
pub_type： 杂志文章
doi：10.3390/ijms131114073
更新日期：2012-10-31 00:00:00
abstract：:Metastatic melanoma is the most deadly type of skin cancer. Despite the success of immunotherapy and targeted agents, the majority of patients experience disease recurrence upon treatment and die due to their disease. Long non-coding RNAs (lncRNAs) are a new subclass of non-protein coding RNAs involved in (epigenetic)...
journal_title：International journal of molecular sciences
pub_type： 杂志文章,评审
doi：10.3390/ijms18040715
更新日期：2017-03-28 00:00:00
abstract：:Tacrolimus is widely used as an immunosuppressant in liver transplantation, and tacrolimus-induced acute kidney injury (AKI) is a serious complication. The urinary neutrophil gelatinase-associated lipocalin (NGAL) level has been linked to tacrolimus-induced AKI in patients starting tacrolimus treatment the morning aft...
journal_title：International journal of molecular sciences
pub_type： 杂志文章
doi：10.3390/ijms20123103
更新日期：2019-06-25 00:00:00
abstract：:Mast cells are pluripotent leukocytes that reside in the mucosa and connective tissue. Recent studies show an increased prevalence of cardiovascular disease among patients with mastocytosis, which is a hematological disease that is characterized by the accumulation of mast cells due to clonal proliferation. This assoc...
journal_title：International journal of molecular sciences
pub_type： 杂志文章,评审
doi：10.3390/ijms20143395
更新日期：2019-07-10 00:00:00
abstract：:As a conserved actin-regulating protein, CAP (adenylyl Cyclase-Associated Protein) functions to facilitate the rearrangement of the actin cytoskeleton. The ubiquitously expressed isoform CAP1 drives mammalian cell migration, and accordingly, most studies on the involvement of CAP1 in human cancers have largely been ba...
journal_title：International journal of molecular sciences
pub_type： 杂志文章,评审
doi：10.3390/ijms20112653
更新日期：2019-05-30 00:00:00
abstract：:Forkhead box P1 (FOXP1) belongs to a family of winged-helix transcription factors that are involved in the processes of cellular proliferation, differentiation, metabolism, and longevity. FOXP1 can affect cell proliferation and migratory ability in hepatocellular carcinoma (HCC) in vitro. However, little is known abou...
journal_title：International journal of molecular sciences
pub_type： 杂志文章
doi：10.3390/ijms17091501
更新日期：2016-09-08 00:00:00
abstract：:Marine environments harbor a wide range of microorganisms from the three domains of life. These microorganisms have great potential to enable discovery of new enzymes and bioactive compounds for industrial use. However, only ~1% of microorganisms from the environment can currently be identified through cultured isolat...
journal_title：International journal of molecular sciences
pub_type： 杂志文章
doi：10.3390/ijms161226101
更新日期：2015-11-27 00:00:00
abstract：:Retinoids exert antitumor effects through the retinoic acid receptor α (RARα). In the present study, we sought to identify the factors involved in the RARα-mediated transcriptional regulation of the tumor suppressor gene and the tissue factor pathway inhibitor 2 (TFPI2) in hepatocellular carcinoma (H...
journal_title：International journal of molecular sciences
pub_type： 杂志文章
doi：10.3390/ijms19051450
更新日期：2018-05-13 00:00:00
abstract：:The analgesic effect of venlafaxine (VLX), which is a selective serotonin and noradrenaline reuptake inhibitor (SNRI), has been observed on oxaliplatin-induced neuropathic pain in mice. Significant allodynia was shown after oxaliplatin treatment (6 mg/kg, i.p.); acetone and von Frey hair tests were used to assess cold...
journal_title：International journal of molecular sciences
pub_type： 杂志文章
doi：10.3390/ijms20071652
更新日期：2019-04-03 00:00:00
abstract：:Neurodegenerative diseases (NDDs) such as Alzheimer's disease, Parkinson's disease and Huntington's disease (HD), amyotrophic lateral sclerosis, and prion diseases are all characterized by the accumulation of protein aggregates (amyloids) into inclusions and/or plaques. The ubiquitous presence of amyloids in NDDs sugg...
journal_title：International journal of molecular sciences
pub_type： 杂志文章,评审
doi：10.3390/ijms19010325
更新日期：2018-01-22 00:00:00
abstract：:Hypertension is asymptomatic and a well-known "silent killer", which can cause various concomitant diseases in human population after years of adherence. Although there are varieties of synthetic antihypertensive drugs available in current market, their relatively low efficacies and major application in only single dr...
journal_title：International journal of molecular sciences
pub_type： 杂志文章,评审
doi：10.3390/ijms19010120
更新日期：2018-01-02 00:00:00
abstract：:(1) Background: A high incidence of intervening sequence (IVS)4+919 G>A mutation with later-onset cardiac phenotype have been reported in a majority of Taiwan Fabry cohorts. Some evidence indicated that conventional biomarkers failed to predict the long-term progression and therapeutic outcome; (2) Methods: In this st...
journal_title：International journal of molecular sciences
pub_type： 杂志文章
doi：10.3390/ijms19051480
更新日期：2018-05-16 00:00:00
abstract：:The basic structural and functional unit of a living organism is a single cell. To understand the variability and to improve the biomedical requirement of a single cell, its analysis has become a key technique in biological and biomedical research. With a physical boundary of microchannels and microstructures, single ...
journal_title：International journal of molecular sciences
pub_type： 杂志文章,评审
doi：10.3390/ijms160922319
更新日期：2015-09-15 00:00:00
abstract：:Tricyclic antidepressants and serotonin noradrenaline reuptake inhibitors are used to treat chronic pain, such as neuropathic pain. Why antidepressants are effective for treatment of neuropathic pain and the precise mechanisms underlying their effects, however, remain unclear. The inhibitory effects of these antidepre...
journal_title：International journal of molecular sciences
pub_type： 杂志文章,评审
doi：10.3390/ijms18112483
更新日期：2017-11-21 00:00:00
abstract：:The aim of this study was to evaluate the in cellulo inhibition of hydrogen-peroxide-induced oxidative stress in skin fibroblasts using different low-molecular-weight polysaccharides (LMPS) prepared from agar (LMAG), chitosan (LMCH) and starch (LMST), which contain various different functional groups (i.e., sulfate, a...
journal_title：International journal of molecular sciences
pub_type： 杂志文章
doi：10.3390/ijms141019399
更新日期：2013-09-25 00:00:00
abstract：:In this study, new composite bisphosphonate delivery systems were obtained from polyurethanes (PUs) and nanocrystalline hydroxyapatite (HA). The biodegradable PUs were first synthesized from poly(ε-caprolactone) diols (PCL diols), poly(ethylene adipate) diol, 1,6-hexamethylene diisocyanate, 1,4-butanediol and HA. More...
journal_title：International journal of molecular sciences
pub_type： 杂志文章
doi：10.3390/ijms150916831
更新日期：2014-09-22 00:00:00
abstract：:Mitochondria in neurons contribute to energy supply, the regulation of synaptic transmission, Ca2+ homeostasis, neuronal excitability, and stress adaptation. In recent years, several studies have highlighted that the neurotransmitter serotonin (5-HT) plays an important role in mitochondrial biogenesis in cortical neur...
journal_title：International journal of molecular sciences
pub_type： 杂志文章
doi：10.3390/ijms21249629
更新日期：2020-12-17 00:00:00
abstract：BACKGROUND:Autologous fat transfer in the form of lipoaspirates for the reconstruction of the breast after breast cancer surgery is a commonly used procedure in plastic surgery. However, concerns regarding the oncologic risk of nutrient-rich fat tissue are widely debated. Previous studies have primarily focused on stud...
journal_title：International journal of molecular sciences
pub_type： 杂志文章
doi：10.3390/ijms21239171
更新日期：2020-12-01 00:00:00
abstract：:For possible future use in Photodynamic Therapy (PDT) and/or Photothermal Therapy (PTT) of cancer and screening of cancer cells a new type of ionic liquid photosensitizer -Cholinium-Purpurin-18 (Chol-Pu-18) - was synthesized and small gold (Au) nanoparticles, stabilized by this photosensitizer were prepared without ad...
journal_title：International journal of molecular sciences
pub_type： 杂志文章
doi：10.3390/ijms9050864
更新日期：2008-05-01 00:00:00
abstract：:Tight regulation of inflammation is very important to guarantee a balanced immune response without developing chronic inflammation. One of the major mediators of the resolution of inflammation is the transcription factor: the nuclear factor erythroid 2-like 2 (Nrf2). Stabilized following oxidative stress, Nrf2 induces...
journal_title：International journal of molecular sciences
pub_type： 杂志文章,评审
doi：10.3390/ijms18122772
更新日期：2017-12-20 00:00:00
abstract：:Pterostilbene (PTE) is a natural sterbenoid contained in blueberries that has an antioxidant effect. In contrast, PTE also generates oxidative stress in cancer cells and provides an antitumor effect. Here, we examined the potential mechanism of this contrasting effect of PTE using three gastrointestinal cancer cell li...
journal_title：International journal of molecular sciences
pub_type： 杂志文章
doi：10.3390/ijms21249347
更新日期：2020-12-08 00:00:00
abstract：:Gaucher disease (GD) is an autosomal recessive lysosomal storage disorder caused by mutations in the acid β-glucosidase gene (GBA1). Besides causing GD, GBA1 mutations constitute the main genetic risk factor for developing Parkinson's disease. The molecular basis of neurological manifestations in GD remain elusive. Ho...
journal_title：International journal of molecular sciences
pub_type： 杂志文章
doi：10.3390/ijms21093268
更新日期：2020-05-05 00:00:00
abstract：:Growing importance has been attributed to interactions between tumors, the stromal microenvironment and adult mesenchymal stem cells. Adipose-derived stem cells (ASCs) are routinely employed in regenerative medicine and in autologous fat transfer procedures. To date, clinical trials have failed to demonstrate the pote...
journal_title：International journal of molecular sciences
pub_type： 杂志文章,评审
doi：10.3390/ijms20133296
更新日期：2019-07-04 00:00:00
abstract：:Immune response plays a fundamental role in protecting the organism from infections; however, dysregulation often occurs and can be detrimental for the organism, leading to a variety of immune-mediated diseases. Recently our understanding of the molecular and cellular networks regulating the immune response, and, in p...
journal_title：International journal of molecular sciences
pub_type： 杂志文章,评审
doi：10.3390/ijms140917347
更新日期：2013-08-22 00:00:00
abstract：:Abstract: Cancer heterogeneity and progression are subject to complex interactions between neoplastic cells and their microenvironment, including the immune system. Although glioblastomas (GBMs) are classified as 'cold tumours' with very little lymphocyte infiltration, they can contain up to 30-40% of tumour-associate...
journal_title：International journal of molecular sciences
pub_type： 杂志文章,评审
doi：10.3390/ijms21030689
更新日期：2020-01-21 00:00:00
abstract：:Plant regeneration and somatic embryogenesis through interspecific hybridization among different Carica species were studied for the development of a papaya ringspot virus-resistant variety. The maximum fruit sets were recorded from the cross of the native variety C. papaya cv. Shahi with the wild species C. cauliflor...
journal_title：International journal of molecular sciences
pub_type： 杂志文章
doi：10.3390/ijms131217065
更新日期：2012-12-12 00:00:00
abstract：:The sialic acid-based molecular mimicry in pathogens and malignant cells is a regulatory mechanism that leads to cross-reactivity with host antigens resulting in suppression and tolerance in the immune system. The interplay between sialoglycans and immunoregulatory Siglec receptors promotes foreign antigens hiding and...
journal_title：International journal of molecular sciences
pub_type： 杂志文章,评审
doi：10.3390/ijms21124361
更新日期：2020-06-19 00:00:00
abstract：:The use of gentamicin for the treatment of bacterial infection has always been an interesting and highly speculated issue for the scientific community. Conversely, its effect on cancer cells has been very little investigated. We studied the effect of high doses of gentamicin on non-Hodgkin's T-cell human lymphoblastic...
journal_title：International journal of molecular sciences
pub_type： 杂志文章
doi：10.3390/ijms16022307
更新日期：2015-01-22 00:00:00
