Therapeutic Strategies in Huntington’s Disease: From Genetic Defect to Gene Therapy DOI Creative Commons
Anamaria Jurcău, Maria Carolina Jurcău

Biomedicines, Journal Year: 2022, Volume and Issue: 10(8), P. 1895 - 1895

Published: Aug. 5, 2022

Despite the identification of an expanded CAG repeat on exon 1 huntingtin gene located chromosome as genetic defect causing Huntington’s disease almost 30 years ago, currently approved therapies provide only limited symptomatic relief and do not influence age onset or progression rate. Research has identified various intricate pathogenic cascades which lead to neuronal degeneration, but interfering with these mechanisms have been marked by many failures remain be validated. Exciting new opportunities are opened emerging techniques target mutant protein DNA RNA, allowing for “gene editing”. Although some issues relating “off-target” effects immune-mediated side need solved, strategies, combined stem cell more traditional approaches targeting specific cascades, such excitotoxicity bioavailability neurotrophic factors, could significant improvement outcomes treated patients.

Language: Английский

The Use of Stem Cells as a Potential Treatment Method for Selected Neurodegenerative Diseases: Review DOI Creative Commons
Elżbieta Cecerska‐Heryć,

Maja Pękała,

Natalia Serwin

et al.

Cellular and Molecular Neurobiology, Journal Year: 2023, Volume and Issue: 43(6), P. 2643 - 2673

Published: April 7, 2023

Stem cells have been the subject of research for years due to their enormous therapeutic potential. Most neurological diseases such as multiple sclerosis (MS), amyotrophic lateral (ALS), Alzheimer's disease (AD), Parkinson's (PD), and Huntington's (HD) are incurable or very difficult treat. Therefore new therapies sought in which autologous stem used. They often patient's only hope recovery slowing down progress symptoms. The most important conclusions arise after analyzing literature on use neurodegenerative diseases. effectiveness MSC cell therapy has confirmed ALS HD therapy. slow progression show early promising signs efficacy. In HD, they reduced huntingtin (Htt) aggregation stimulation endogenous neurogenesis. MS with hematopoietic (HSCs) inducted significant recalibration pro-inflammatory immunoregulatory components immune system. iPSC allow accurate PD modeling. patient-specific therefore minimize risk rejection and, long-term observation, did not form any tumors brain. Extracellular vesicles derived from bone marrow mesenchymal stromal (BM-MSC-EVs) Human adipose-derived stromal/stem (hASCs) widely used treat AD. Due reduction Aβ42 deposits increasing survival neurons, improve memory learning abilities. Despite many animal models clinical trial studies, still needs be refined increase its human body.

Language: Английский

Citations

36

Advances in nanotechnology versus stem cell therapy for the theranostics of Huntington's disease DOI Creative Commons
Ghulam Mustafa Channa, Dilawar Hassan, Mahira Zeeshan

et al.

Journal of Drug Delivery Science and Technology, Journal Year: 2023, Volume and Issue: 87, P. 104774 - 104774

Published: July 20, 2023

Huntington's disease (HD) is a neurological disorder characterized by reduction in medium-spiny neurons the brain. Currently, there no cure for HD and treatment relies on symptomatic therapy. The generation of stem cells, including neural, mesenchymal, pluripotent, through conventional strategies or direct cell reprogramming has revolutionized therapy research. Due to their unique ability differentiate into variety self-renew, grow, cells have become an area interest treating various complex unresolved neurodegenerative disorders. Nanotechnology emerged as novel approach with great potential reduced side effects. Nanoparticles (NPs) can act nanovehicles delivering therapeutic agents, siRNAs, neurotrophic factors, different drugs. Additionally, NPs be used alternative based antioxidant reactive oxygen species (ROS)-scavenging properties that protect neuronal cells. Some even exhibit interfere protein aggregation mutant Huntingtin proteins during processes. This review focuses most studied HD, polymeric, lipid-based, liposomes, solid lipid metal/metal oxide. combination diagnosis treatment. been manage cellular microenvironment, improve efficiency drug delivery brain enhance transplant survival. Understanding characteristics essential applying them purposes. In this study, biology well benefits drawbacks using its are discussed.

Language: Английский

Citations

34

Induction Mechanism of Ferroptosis, Necroptosis, and Pyroptosis: A Novel Therapeutic Target in Nervous System Diseases DOI Open Access
Lu Tang, Sitong Liu, Shiwei Li

et al.

International Journal of Molecular Sciences, Journal Year: 2023, Volume and Issue: 24(12), P. 10127 - 10127

Published: June 14, 2023

In recent years, three emerging cell deaths, ferroptosis, necroptosis and pyroptosis, have gradually attracted everyone’s attention, they also play an important role in the occurrence development of various diseases. Ferroptosis is idiographic iron-dependent form regulated death with hallmark accumulation intracellular reactive oxygen species (ROS). Necroptosis a necrotic mediated by receptor-interacting protein kinase 1(RIPK1) 3RIPK3. Pyroptosis, known as inflammatory necrosis, programmed necrosis Gasdermin D (GSDMD). It manifested continuous swelling cells until membrane ruptures, resulting release contents activation strong response. Neurological disorders remain clinical challenge patients do not respond well to conventional treatments. Nerve can aggravate neurological This article reviews specific mechanisms these types their relationship diseases evidence for diseases; understanding pathways helpful treatment

Language: Английский

Citations

30

Gut microbiota dysbiosis and Huntington's disease: Exploring the gut-brain axis and novel microbiota-based interventions DOI

Garvita Sharma,

Shristi Saroj Biswas,

Jayapriya Mishra

et al.

Life Sciences, Journal Year: 2023, Volume and Issue: 328, P. 121882 - 121882

Published: June 24, 2023

Language: Английский

Citations

25

C1q and central nervous system disorders DOI Creative Commons
Wenjie Zhang, Yuan Chen,

Hui Pei

et al.

Frontiers in Immunology, Journal Year: 2023, Volume and Issue: 14

Published: March 23, 2023

C1q is a crucial component of the complement system, which activated through classical pathway to perform non-specific immune functions, serving as first line defense against pathogens. can also bind specific receptors carry out and other playing vital role in maintaining homeostasis normal physiological functions. In developing central nervous system (CNS), functions synapse formation pruning, key player development neuronal networks CNS. has close relationship with microglia astrocytes, under their influence, may contribute CNS disorders. Furthermore, have independent effects on neurological disorders, producing either beneficial or detrimental outcomes. Most evidence for these comes from animal models, some human specimen studies. now emerging promising target treatment variety diseases, clinical trials are already underway This article highlights offering new directions diagnosis conditions.

Language: Английский

Citations

23

Role of histone modifications in neurogenesis and neurodegenerative disease development DOI
Anqi Zhao,

Wenhong Xu,

Rui Han

et al.

Ageing Research Reviews, Journal Year: 2024, Volume and Issue: 98, P. 102324 - 102324

Published: May 16, 2024

Language: Английский

Citations

13

The Role of the Gut Microbiota in Neurodegenerative Diseases DOI Creative Commons

Arshilin Philip Mani,

Balamuralikrishnan Balasubramanian,

Linsha A. Mali

et al.

Microbiology Research, Journal Year: 2024, Volume and Issue: 15(2), P. 489 - 507

Published: April 5, 2024

The human gut has a rich and dynamic microbial population that plays an important role in many physiological activities. This review explores the complex interaction between microbiota health, with emphasis on its effect neurodegenerative illnesses. makeup of microbiome impact brain function through gut–brain axis is highlighted. Dysbiosis, characterized by changes microbiota’s composition, been linked to development diseases such as Alzheimer’s, Parkinson’s, Huntington’s, amyotrophic lateral sclerosis. A Bidirectional communication stomach takes place via variety channels, including neurotransmitters metabolites generated bacteria. We investigate processes which dysbiosis causes neuroinflammation, oxidative stress, neuronal damage, drive disease development. Potential therapeutic approaches focus microbiota, antibiotics, probiotics, prebiotics, fecal transplantation, are reviewed, promising preclinical clinical findings. Overall, this study emphasizes relevance illnesses, well need understand target gut-brain for future treatment options.

Language: Английский

Citations

10

Targeting selective autophagy and beyond: From underlying mechanisms to potential therapies DOI Creative Commons
Wei Ma, Yingying Lü, Xin Jin

et al.

Journal of Advanced Research, Journal Year: 2024, Volume and Issue: 65, P. 297 - 327

Published: May 14, 2024

Autophagy is an evolutionarily conserved turnover process for intracellular substances in eukaryotes, relying on lysosomal (in animals) or vacuolar yeast and plants) mechanisms. In the past two decades, emerging evidence suggests that, under specific conditions, autophagy can target particular macromolecules organelles degradation, a termed selective autophagy. Recently, accumulating studies have demonstrated that abnormality of closely associated with occurrence progression many human diseases, including neurodegenerative cancers, metabolic cardiovascular diseases. This review aims at systematically comprehensively introducing its role various while unravelling molecular mechanisms By providing theoretical basis development related small-molecule drugs as well treating this seeks to contribute understanding therapeutic potential. review, we introduce dissect major categories been discovered. We also focus recent advances underlying both classical non-classical Moreover, current situation targeting different types further summarized, valuable insights into discovery more candidate future. On other hand, reveal clinically relevant implementations are potentially autophagy, such predictive approaches treatments tailored individual patients.

Language: Английский

Citations

10

Therapeutic role of neural stem cells in neurological diseases DOI Creative Commons
Ling Yang, Sicheng Liu, Yiyi Liu

et al.

Frontiers in Bioengineering and Biotechnology, Journal Year: 2024, Volume and Issue: 12

Published: March 7, 2024

The failure of endogenous repair is the main feature neurological diseases that cannot recover damaged tissue and resulting dysfunction. Currently, range treatment options for limited, approved drugs are used to treat diseases, but therapeutic effect still not ideal. In recent years, different studies have revealed neural stem cells (NSCs) made exciting achievements in diseases. NSCs potential self-renewal differentiation, which shows great foreground as replacement therapy broadens a new way cell therapy. biological functions nerve injury include neuroprotection, promoting axonal regeneration remyelination, secretion neurotrophic factors, immune regulation, improve inflammatory microenvironment injury. All these reveal play an important role improving progression Therefore, it significance better understand functional view this, we comprehensively discussed application value well existing problems challenges.

Language: Английский

Citations

9

Exploring molecular mechanisms, therapeutic strategies, and clinical manifestations of Huntington’s disease DOI
Alaa Shafie, Amal Adnan Ashour,

Saleha Anwar

et al.

Archives of Pharmacal Research, Journal Year: 2024, Volume and Issue: 47(6), P. 571 - 595

Published: May 19, 2024

Language: Английский

Citations

9