Browsing by Author "Aakriti Sharma"

Now showing 1 - 1 of 1

Gene Therapy, OMICS Approaches, and Translational Neurodegenerative Models: Recent Advancements and Future Perspectives
(CRC Press, 2024) Aakriti Sharma; Shashank Shekhar Mishra; Sandeep Singh
Gene therapy, OMICS approaches, and translational neurodegenerative models collectively represent a cutting-edge triumvirate at the forefront of addressing neurodegenerative diseases. Gene therapy, through gene replacement, silencing, and editing, aims to correct genetic defects at their source, offering hope for conditions like Alzheimer’s and Parkinson’s. OMICS technologies-including genomics, transcriptomics, proteomics, and metabolomics-provide a comprehensive view of the molecular intricacies of these diseases, identifying genetic variants, gene expression changes, and protein profiles critical for understanding and developing targeted interventions. Translational neurodegenerative models bridge the gap between bench research and clinical applications, utilising in vitro cell cultures, induced pluripotent stem cells, and organoids to replicate disease complexities observed in humans. These models enhance the reliability and relevance of patient-specific studies, paving the way for personalised treatments. Understanding the molecular basis of neurodegeneration is imperative for advancing therapeutic interventions, offering insights into genetic mutations, abnormal protein aggregations, and disrupted signaling pathways. This knowledge facilitates the development of precise diagnostics, biomarkers, and targeted therapies, potentially halting or reversing disease progression. Gene therapy’s evolution, marked by key milestones such as the first clinical trial for Parkinson’s disease and the FDA approval for spinal muscular atrophy treatment, underscores its transformative potential. Techniques like CRISPR-Cas9 have further revolutionised the field, enabling precise genetic modifications. This integrated approach, combining gene therapy, OMICS, and translational models, holds promise for innovative and personalised medical interventions, aiming to improve outcomes and quality of life for individuals with neurodegenerative diseases. © 2025 selection and editorial matter, Sachchida Nand Rai, Sandeep Singh, Santosh Kumar Singh.