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TOPICAL COLLECTIONS

Gene-Based Therapeutics: Pioneering a New Era in Precision Medicine
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Guest Editors

Manish Dwivedi

Manish Dwivedi

Amity University Lucknow

<p>Dr. Manish Dwivedi is an associate professor at the Institute of Biotechnology, Amity University Uttar Pradesh, Lucknow. He earned his PhD in biotechnology from the University of Allahabad, India (2014), and holds an MSc and MPhil in Biotechnology. Dr. Dwivedi has gained extensive research experience at leading institutes, including the Weizmann Institute of Science and the Hebrew University in Israel, and the Max Planck Institute in Germany.</p><p><br></p><p>His research focuses on the biophysical and structural studies of membrane proteins associated with human diseases, as well as proteomics, immunoinformatics, gene polymorphism, and protein biochemistry. He is a recipient of several prestigious awards, including the DST-INSPIRE Faculty Award (Government of India), PBC International Postdoctoral Fellowship (Israel), and EMBO Fellowships (Germany, Greece, Spain, Italy).</p><p><br></p><p>Dr. Dwivedi has led several funded research projects on gallbladder cancer and has published over 60 papers in reputed journals such as <em>Nature Communications</em>, <em>Journal of Biological Chemistry</em>, and <em>Scientific Reports</em>. He has also contributed multiple book chapters and serves as an Ambassador for Bentham Science, as well as on the editorial and review boards of several international journals and research communities.</p>

Swadha Singh

Swadha Singh

Department of Psychiatry, Icahn School of Medicine at Mount Sinai

<p>Dr. Swadha Singh is an assistant scientist in the Department of Psychiatry at the Icahn School of Medicine at Mount Sinai, New York. She earned her PhD and master’s in quantitative and systems biology from the University of California, Merced (2017–2022), and her MSc (2012–2014) and BSc (2009–2012) degrees in bioinformatics and applied science from the University of Allahabad, India. Her research integrates computational biology, single-cell transcriptomics, and epigenomics to uncover molecular mechanisms underlying neuropsychiatric disorders such as schizophrenia and bipolar disorder. She developed the first large-scale cell-type-specific genomic and 3D chromatin architecture resource from human midbrain dopaminergic neurons. In parallel, she has developed an integrative multi-omic framework combining CAGE-seq promoter entropy, ATAC-seq, and RNA-seq to dissect promoter activity, chromatin accessibility, and transcriptional regulation in the human brain, providing insight into disease-associated promoter switching and transcription factor dynamics. She has authored multiple research publications spanning computational genomics, chromatin biology, and transcriptomic regulation. Dr. Singh is a member of the PsychENCODE Consortium, the Society for Neuroscience, and the American Society of Human Genetics.</p>

Collection Overview

Gene-based therapeutic strategies represent a transformative approach in modern medicine, targeting the root causes of diseases at the genetic level rather than merely managing symptoms. By correcting, silencing, or replacing defective genes, these therapies offer the potential for long-term or even permanent cures for genetic disorders, cancers, and infectious diseases.


Techniques such as CRISPR-Cas9 genome editing, RNA interference, and viral vector-mediated gene delivery have revolutionized precision medicine, enabling targeted interventions with minimal off-target effects. Gene therapies also hold promise for personalized treatment, allowing therapies to be tailored to an individual’s genetic makeup.


Beyond monogenic disorders, they are being explored for treating complex diseases like cancer, cardiovascular disorders, and neurodegeneration. Despite challenges in delivery efficiency, immune responses, and cost, ongoing advancements in vector design and regulatory oversight continue to enhance their safety and effectiveness, making gene-based therapies a cornerstone of next-generation medicine.


This collection highlights innovative techniques and experimental approaches that advance gene therapy research and clinical translation. It focuses on cutting-edge methods for gene delivery, genome editing, vector engineering, and functional validation of therapeutic genes.


By providing standardized, reproducible, and accessible methodologies, the collection aims to accelerate the development of safer and more effective gene-based treatments. It serves as a practical resource for researchers, offering validated protocols that enhance experimental rigor and support the translation of gene therapy from bench to bedside.

Articles

Genome Editing in Primary Mammalian Cells via Electroporation of Editor RNA
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Genome Editing in Primary Mammalian Cells via Electroporation of Editor RNA

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