Genetic Phenotypes of Alzheimer’s Disease: Mechanisms and Potential Therapy

Phenomics, Journal Year: 2023, Volume and Issue: 3(4), P. 333 - 349

Published: April 3, 2023

Abstract Years of intensive research has brought us extensive knowledge on the genetic and molecular factors involved in Alzheimer's disease (AD). In addition to mutations three main causative genes familial AD (FAD) including presenilins amyloid precursor protein genes, studies have identified several as most plausible for onset progression FAD, such triggering receptor expressed myeloid cells 2 , sortilin-related 1 adenosine triphosphate-binding cassette transporter subfamily A member 7 . The apolipoprotein E ε4 allele is reported be strongest risk factor sporadic (SAD), it also plays an important role FAD. Here, we reviewed recent developments that contributed understanding phenotypes FAD compared them with SAD. We further advancements gene therapy discussed future perspectives based phenotypes.

Language: Английский

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CRISPR/Cas9 genome editing for neurodegenerative diseases.

PubMed, Journal Year: 2023, Volume and Issue: 22, P. 567 - 582

Published: Jan. 1, 2023

Gene therapy has emerged as a promising therapeutic strategy for various conditions, including blood disorders, ocular disease, cancer, and nervous system disorders. The advent of gene editing techniques facilitated the ability researchers to specifically target modify eukaryotic cell genome, making it valuable tool therapy. This can be performed through either in vivo or ex approaches. tools, such zinc finger nucleases, transcription activator-like effector CRISPR-Cas-associated employed purposes. Among these CRISPR-Cas-based stands out because its introduce heritable genome changes by designing short guide RNAs. review aims provide an overview CRISPR-Cas technology summarizes latest research on application CRISPR/Cas9 treatment most prevalent neurodegenerative diseases Alzheimer's Parkinson's Huntington's Amyotrophic lateral sclerosis, Spinocerebellar ataxia.

Language: Английский

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Nano biomaterials based strategies for enhanced brain targeting in the treatment of neurodegenerative diseases: an up-to-date perspective

Journal of Nanobiotechnology, Journal Year: 2023, Volume and Issue: 21(1)

Published: Dec. 13, 2023

Neurons and their connecting axons gradually degenerate in neurodegenerative diseases (NDs), leading to dysfunctionality of the neuronal cells eventually death. Drug delivery for treatment effected nervous system is notoriously complicated because presence natural barriers, i.e., blood-brain barrier blood cerebrospinal fluid barrier. Palliative care currently standard many diseases. Therefore, programs that target disease's origin rather than its symptoms are recommended. Nanotechnology-based drug platforms offer an innovative way circumvent these obstacles deliver medications directly central system, thereby enabling several common neurological problems, Alzheimer's, Parkinson's, Huntington's, amyotrophic lateral sclerosis. Interestingly, combination nanomedicine gene therapy enables targeting selective mutant genes responsible progression NDs, which may provide a much-needed boost struggle against Herein, we discussed various obstacles, followed by detailed insight into recently developed techniques restore function via differentiation neural stem cells. Moreover, comprehensive background on role controlling neurogenesis explained. Additionally, numerous phytoconstituents with neuroprotective properties molecular targets identification management NDs also deliberated. Furthermore, ongoing clinical trials marketed products provided this manuscript.

Language: Английский

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The role of neurogenomics in personalized neurosurgical interventions: a new frontier in precision medicine

Neurosurgical Review, Journal Year: 2024, Volume and Issue: 47(1)

Published: Aug. 27, 2024

Language: Английский

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Targeting Cytokine-Mediated Inflammation in Brain Disorders: Developing New Treatment Strategies

Pharmaceuticals, Journal Year: 2025, Volume and Issue: 18(1), P. 104 - 104

Published: Jan. 15, 2025

Cytokine-mediated inflammation is increasingly recognized for playing a vital role in the pathophysiology of wide range brain disorders, including neurodegenerative, psychiatric, and neurodevelopmental problems. Pro-inflammatory cytokines such as interleukin-1 (IL-1), tumor necrosis factor-alpha (TNF-α), interleukin-6 (IL-6) cause neuroinflammation, alter function, accelerate disease development. Despite progress understanding these pathways, effective medicines targeting are still limited. Traditional anti-inflammatory immunomodulatory drugs peripheral inflammatory illnesses. Still, they face substantial hurdles when applied to central nervous system (CNS), blood-brain barrier (BBB) unwanted systemic effects. This review highlights developing treatment techniques modifying cytokine-driven focusing on advances that selectively target critical involved pathology. Novel approaches, cytokine-specific inhibitors, antibody-based therapeutics, gene- RNA-based interventions, sophisticated drug delivery systems like nanoparticles, show promise with respect lowering neuroinflammation greater specificity safety. Furthermore, developments biomarker discoveries neuroimaging improving our ability monitor responses, allowing more accurate personalized regimens. Preclinical clinical trial data demonstrate therapeutic potential tailored techniques. However, significant challenges remain, across BBB reducing off-target As research advances, creation personalized, cytokine-centered therapeutics has therapy landscape illnesses, giving patients hope better results higher quality life.

Language: Английский

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The role of fusion proteins as biomarkers and therapeutic agents for Alzheimer's disease: A narrative review

NeuroMarkers., Journal Year: 2025, Volume and Issue: 2(2), P. 100041 - 100041

Published: March 8, 2025

Language: Английский

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Development of bromo- and fluoro-based α, β-unsaturated ketones as highly potent MAO-B inhibitors for the treatment of Parkinson's disease

Journal of Molecular Structure, Journal Year: 2022, Volume and Issue: 1266, P. 133545 - 133545

Published: June 17, 2022

Language: Английский

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Molecular mechanisms of topiramate and its clinical value in epilepsy

Seizure, Journal Year: 2022, Volume and Issue: 98, P. 51 - 56

Published: April 4, 2022

Language: Английский

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Biomaterials-mediated CRISPR/Cas9 delivery: recent challenges and opportunities in gene therapy

Frontiers in Chemistry, Journal Year: 2023, Volume and Issue: 11

Published: Sept. 28, 2023

The use of biomaterials in delivering CRISPR/Cas9 for gene therapy infectious diseases holds tremendous potential. This innovative approach combines the advantages with protective properties biomaterials, enabling accurate and efficient editing while enhancing safety. Biomaterials play a vital role shielding components, such as lipid nanoparticles or viral vectors, from immunological processes degradation, extending their effectiveness. By utilizing flexibility tailored systems can be designed to address specific genetic diseases, paving way personalized therapeutics. Furthermore, this delivery method offers promising avenues combating illnesses by precisely modifying pathogen genomes, reducing pathogenicity. facilitate site-specific modifications, ensuring effective infected cells minimizing off-target effects. However, challenges remain, including optimizing efficiency, effects, long-term safety, establishing scalable production techniques. Thorough research, pre-clinical investigations, rigorous safety evaluations are imperative successful translation laboratory clinical applications. In review, we discussed how using revolutionizes disease treatment, offering precise safe capabilities potential significantly improve human health quality life.

Language: Английский

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Small Extracellular Vesicles’ miRNAs: Biomarkers and Therapeutics for Neurodegenerative Diseases

Pharmaceutics, Journal Year: 2023, Volume and Issue: 15(4), P. 1216 - 1216

Published: April 11, 2023

Neurodegenerative diseases are critical in the healthcare system as patients suffer from progressive despite currently available drug management. Indeed, growing ageing population will burden country’s and caretakers. Thus, there is a need for new management that could stop or reverse progression of neurodegenerative diseases. Stem cells possess remarkable regenerative potential has long been investigated to resolve these issues. Some breakthroughs have achieved thus far replace damaged brain cells; however, procedure’s invasiveness prompted scientists investigate using stem-cell small extracellular vesicles (sEVs) non-invasive cell-free therapy address limitations cell therapy. With advancement technology understand molecular changes diseases, efforts made enrich stem cells’ sEVs with miRNAs increase therapeutic efficacy sEVs. In this article, pathophysiology various highlighted. The role biomarkers treatments also discussed. Lastly, applications delivery their miRNA-enriched treating emphasised reviewed.

Language: Английский

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