Alzheimer’s disease (AD) remains a significant challenge due to its complex pathology and limited effectiveness of current treatments. Recent advances in gene therapy offer promising avenues for addressing AD by targeting its root causes. Studies highlight the role of genetic mutations, such as those in the PSEN-1, PSEN-2, and APP genes, in familial AD, as well as mutations in the tau and APO-E genes, which contribute to amyloid accumulation, tau hyperphosphorylation, and neurodegeneration. Gene-based interventions, focusing on correcting these genetic abnormalities, have shown potential in animal models and early clinical trials to mitigate cognitive decline and neuronal loss, paving the way for innovative treatments.

Gene therapy, combined with multimodal strategies like lifestyle interventions and advanced drug delivery mechanisms, represents a shift toward more comprehensive treatment approaches. By leveraging genetic insights and animal models, researchers aim to identify novel therapeutic targets and enhance the precision of gene delivery techniques. The integration of gene-based strategies with traditional and non-pharmacological methods underscores a transformative direction in AD treatment, offering hope for improved outcomes in this progressive neurodegenerative disease.

Reference: Ataei B, Hokmabadi M, Asadi S, et al. A review of the advances, insights, and prospects of gene therapy for Alzheimer's disease: A novel target for therapeutic medicine. Gene. 2024 Jun 20;912:148368. doi: 10.1016/j.gene.2024.148368. Epub 2024 Mar 12. PMID: 38485038.

Alzheimer's disease (AD) is a prevalent neurodegenerative disorder, primarily affecting the elderly, and is the leading cause of dementia, contributing to 50–75% of cases globally. MicroRNAs (miRNAs) have emerged as crucial regulators in AD progression, influencing amyloid-beta production, tau phosphorylation, synaptic function, and neuroinflammation. Recent research highlights miRNAs as potential biomarkers for early diagnosis and therapeutic targets, offering new avenues to understand and address AD's molecular complexity.

MiRNAs play a central role in modulating gene expression and are implicated in synaptic dysfunction, mitochondrial impairment, and inflammatory responses in AD. Dysregulated miRNAs contribute to neurodegenerative processes by impacting oxidative stress, mitochondrial dynamics, and neuronal signaling pathways. Studies on miR-132, for instance, reveal its neuroprotective role in reducing tau pathology and enhancing cognitive functions. The integration of miRNA research with genomic and molecular network studies holds promise for uncovering novel therapeutic strategies.

Reference: Li YB, Fu Q, Guo M, et al. MicroRNAs: pioneering regulators in Alzheimer's disease pathogenesis, diagnosis, and therapy.Transl Psychiatry. 2024 Sep 10;14(1):367. doi: 10.1038/s41398-024-03075-8. PMID: 39256358; PMCID: PMC11387755.

Alzheimer's disease (AD) is a prevalent neurodegenerative disorder affecting millions worldwide. Emerging research highlights the gut microbiome's potential role in AD pathogenesis, mediated through the gut-brain axis. Alterations in the gut microbiome, including reduced short-chain fatty acids (SCFAs) and increased lipopolysaccharides (LPSs), can lead to systemic inflammation, blood-brain barrier dysfunction, and heightened amyloid-beta deposition, contributing to AD progression.

Studies reveal significant differences in gut microbiota composition between patients with AD and healthy controls, including shifts in bacterial genera and reduced SCFA-producing strains. Potential treatments targeting the gut microbiome, including prebiotics, probiotics, synbiotics, antibiotics, and fecal microbiota transplantation (FMT), have shown promise in reducing amyloid-beta levels and improving cognitive function. These interventions have also demonstrated potential in modulating inflammation in animal models and small clinical trials. Understanding the gut-brain connection may open new avenues for innovative therapies to combat this debilitating disease.

Reference: Sait AM, Day PJR. Interconnections between the Gut Microbiome and Alzheimer's Disease: Mechanisms and Therapeutic Potential. Int J Mol Sci. 2024 Aug 7;25(16):8619. doi: 10.3390/ijms25168619. PMID: 39201303; PMCID: PMC11354889.