Current Gene Therapy[JOURNAL]
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200 papers
Exploring Gene Therapy: The Next Generation of Colorectal Cancer Treatment.
Kamal R, Paul P, Diksha … +1 more , Awasthi A
Curr Gene Ther · 2025 · PMID 40351072 · Publisher ↗
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Advances in Gene Therapy for X-Linked Myotubular Myopathy: Current Progress and Future Challenges.
Hussain MS, Khan G, Gupta G
Curr Gene Ther · 2026 · PMID 40329721 · Publisher ↗
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Identification and Validation of Amino Acid Metabolism-Related Biomarkers and Investigation of their Potential Mechanisms in Lung Adenocarcinoma.
Zhu X, Zhang Y, Pan P … +10 more , Liu X, Zhang J, Du X, Wang T, Teng Y, Fan C, Li J, Wu J, Zeng Z, Yang S
Curr Gene Ther · 2025 Apr · PMID 40257028 · Publisher ↗
BACKGROUND: In lung adenocarcinoma (LUAD), the metabolism of amino acids (AAs) plays a crucial role in the growth, infiltration, and metastasis of tumor cells. Nevertheless, the potential of AA metabolism-associated gene...
BACKGROUND: In lung adenocarcinoma (LUAD), the metabolism of amino acids (AAs) plays a crucial role in the growth, infiltration, and metastasis of tumor cells. Nevertheless, the potential of AA metabolism-associated genes (AAMRGs) to serve as prognostic indicators in LUAD remains ambiguous. Thus, this study sought to evaluate the prognostic value of AAMRGs in LUAD patients. METHODS: Herein, we extracted LUAD transcriptomic information from two key repositories, namely The Cancer Genome Atlas Program (TCGA) and Gene Expression Omnibus. The non-negative matrix factorization (NMF) clustering technique was used to categorize the LUAD cases based on their AAM profiles before assessing the survival rates and composition of immune cells. Using limma software, shared dysregulated transcripts were identified across subgroups before functional annotation via DAVID, which comprised exploration of gene ontology and the Kyoto Encyclopedia of Genes and Genomes pathway. The prognostic framework was developed using five prognostic indicators through TCGA-derived LUAD specimens. We performed the analysis using singlevariable Cox, least absolute shrinkage and selection operator regression, and multi-factorial Cox regression. Molecular pathways between cohorts were compared with gene set enrichment analysis (GSEA). Real-time quantitative polymerase chain reaction (RT-qPCR) and immunohistochemical (IHC) analysis were utilized to validate the key genetic components of the model. RESULTS: NMF clustering analysis was performed to categorize 497 LUAD patients into three distinct subgroups with obvious variations in the survival rates. The subtypes exhibited substantial disparities in immune cell populations, particularly in monocytes and mast cells. Analysis of 176 shared differentially expressed genes (DEGs) revealed enrichment in T lymphocyte stimulation, immunological reactions, and extra immune-related processes within the subgroups. The prognostic framework was constructed using biomarkers, such as ERO1LB, HPGDS, LOXL2, TMPRSS11E, and SLC34A2. Moreover, GSEA demonstrated a correlation between elevated risk and cell cycle processes, but lower risk was linked with arachidonic acid metabolic pathways. Analysis of 1128 DEGs revealed enrichment in various physiological processes, including cellular division, p53 signaling cascades, immunological responses, and additional pathways upon the comparison of high and low-risk cohorts. The RT-qPCR analysis confirmed elevated expression levels of ERO1LB and TMPRSS11E in LUAD specimens. Consistent with RT-qPCR analysis, the IHC results affirmed that the expression levels of ERO1LB and TMPRSS11E were increased in LUAD specimens. CONCLUSION: The five identified AAMRGs in LUAD were validated and appropriately utilized to construct a risk assessment model that could potentially act as prognostic biomarkers for LUAD patients.
Imaged Capillary Isoelectric Focusing (icIEF) Platform for Characterization of Charge Variants of Adeno-Associated Virus (AAV) Capsids and Impact on their Transduction Efficiency.
Hoyle B, Bello D, Hill J … +2 more , Das S, Kim J
Curr Gene Ther · 2026 · PMID 40237057 · Publisher ↗
OBJECTIVE: Adeno-Associated Virus (AAV) vectors are comprised of a capsid protein encapsulating a Deoxyribonucleic Acid (DNA) transgene that has been used in the gene therapy field showing potential to treat a range of g...
OBJECTIVE: Adeno-Associated Virus (AAV) vectors are comprised of a capsid protein encapsulating a Deoxyribonucleic Acid (DNA) transgene that has been used in the gene therapy field showing potential to treat a range of genetic diseases. Methods in the field of gene therapy should be optimized or enhanced to deepen understanding of AAVs, specifically around charge heterogeneity of capsid species. METHODS: In this study, a versatile approach was presented for investigating the charge heterogeneity of AAV capsid proteins of a variety of serotypes. This method employs Imaged Capillary Isoelectric Focusing (icIEF) coupled with native fluorescence imaging detection and has undergone exhaustive validation. RESULTS: Demonstrating its platform nature, this method analyzed seven different AAV serotypes from multiple manufacturing platforms. The distinctive profiles generated for each AAV serotype serve as valuable indicators for both identity confirmation and stability assessment. It was shown that thermal stress and pH conditions play a role in increasing acidic charged variants over time, affecting the charge heterogeneity of AAVs, which can be serotype-specific. Reverse phase LC-MS was used to identify and confirm the increased presence of Post-Translational Modifications (PTMs) that are linked to increasing acidic species variants relative to non-stressed AAVs. CONCLUSION: These PTMs have biological consequences reflected in the diminished expression of the protein of interest in vitro. This cIEF method successfully analyzed a variety of AAV serotypes, and increasing trends of acidic variants led to reduced potency.
Non-coding RNAs-based Therapy and Angiogenesis: A New Era for the Management of Gliomas.
Beylerli O, Gareev I, Musaev E … +3 more , Ilyasova T, Roumiantsev S, Chekhonin V
Curr Gene Ther · 2026 · PMID 40231533 · Publisher ↗
The relentless pursuit of understanding and combating glioblastoma (GBM), one of the most formidable foes in the realm of cancer, requires a deeper exploration of its intricate dynamics. Gliomas, particularly GBM, are kn...
The relentless pursuit of understanding and combating glioblastoma (GBM), one of the most formidable foes in the realm of cancer, requires a deeper exploration of its intricate dynamics. Gliomas, particularly GBM, are known for their lethal nature, and a significant aspect of their pathogenesis lies in their ability to manipulate the blood vessels that sustain them. This complex relationship is governed by a multitude of molecular mechanisms involving a diverse array of cell types within the tumor microenvironment. Central to this intricate web of regulation are non-coding RNAs (ncRNAs), enigmatic molecules that have recently emerged as key players in cancer biology. These ncRNAs wield a remarkable influence on gene expression, often via epigenetic modifications and intricate control over angiogenesis-related molecules. Their role in GBM angiogenesis adds another layer of complexity to our understanding of this disease. In the realm of cancer therapeutics, targeting angiogenesis has become a prominent strategy. However, the efficacy of current antiangiogenic treatments against GBM is often transient, as these tumors can rapidly develop resistance, becoming even more aggressive. GBM employs a diverse set of strategies to foster its abnormal vasculature, which, in turn, holds the key to understanding why anti-angiogenic therapies often fall short of expectations. This review aims to shed light on potential strategies and novel perspectives to overcome GBM 's resistance to anti-angiogenic therapy. By exploring innovative approaches, including those centered on ncRNAs, we strive to chart a course toward more effective treatments. This journey into the depths of GBM 's complexities offers not only hope but also a blueprint for future research and therapeutic development. As we uncover the intricate mechanisms at play, we inch closer to the day when GBM is no longer an insurmountable adversary in the fight against cancer.
Next-Generation Whole-Exome Pattern: Advanced Methods and Clinical Significance.
Ashique S, Islam A, Kaur Sandhu N … +3 more , Sharma B, Pathak R, Sharma H
Curr Gene Ther · 2026 · PMID 40231504 · Publisher ↗
NGS (Next-generation sequencing) has emerged as the primary approach for gene finding in uncommon hereditary disorders. Targeted gene panels, whole genome sequencing (WGS), and whole exome sequencing (WES) are uses of ne...
NGS (Next-generation sequencing) has emerged as the primary approach for gene finding in uncommon hereditary disorders. Targeted gene panels, whole genome sequencing (WGS), and whole exome sequencing (WES) are uses of next-generation sequencing and other related technologies. It is possible to explain personal or individual genome sequencing using NGS technology, as well as to detect disease-causing mutations using NGS findings. NGS, deep sequencing or massively parallel are similar words that describe a method of DNA sequencing leading to revolutionary change in genomic research. Due to its cost-effectiveness, Whole-Exome sequencing (WES) using Next-Generation Sequencing (NGS) is becoming increasingly popular in the field of human genetics. As a diagnostic tool, this technology can reduce the duration of the diagnostic process for several patients and has mostly made a significant contribution to the identification of new genes responsible for causing diseases. Considering the diverse range of phenotypic presentations of the diagnosis, NGS has the potential to uncover causative mutations, including de novo, new, and familial variants, related to epileptic syndromes and significantly enhance molecular diagnosis. The present study centres on the potential applications of next-generation exome sequencing in clinical diagnostics and the challenges encountered in the data processing of such data.
CRISPR/Cas9 Technology for Modifying Immune Checkpoint in CAR-T Cell Therapy for Hematopoietic Malignancies.
Shams F, Sharif E, Abbasi-Kenarsari H … +8 more , Hashemi N, Hosseini MS, Heidari N, Noori E, Amini AH, Bazrgar M, Rouhani M, Teng Y
Curr Gene Ther · 2026 · PMID 40231503 · Publisher ↗
Hematologic malignancies, which arise from dysregulation of hematopoiesis, are a group of cancers originating in cells with diminished capacity to differentiate into mature progeny and accumulating immature cells in bloo...
Hematologic malignancies, which arise from dysregulation of hematopoiesis, are a group of cancers originating in cells with diminished capacity to differentiate into mature progeny and accumulating immature cells in blood-forming tissues such as lymph nodes and bone marrow. Immune- targeted therapies, such as Immune Checkpoint Blockade (ICB), chimeric antigen receptor T (CAR-T) cell therapy, and the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) system, a precise, popular, and versatile genome engineering tool, has opened new avenues for the treatment of malignancies. Targeting immune checkpoints has revolutionized FDA approval in cytotoxic T-lymphocyte-associated protein 4 (CTLA-4), PD-1 (programmed death-1), and PDL1. According to the ICB and CAR techniques, the production of efficient CAR-T cells depends on the successful genetic modification of T cells, making them less susceptible to immune escape and suppression by cancer cells, which results in reduced off-target toxicity. Therefore, CRISPR/Cas9 has revolutionized the immune checkpoint-based approach for CAR-T cell therapy of hematologic malignancy. Continued research and clinical trials will undoubtedly pave the way for further advances in this field, ultimately benefiting patients and improving outcomes.
Transforming RNA-Based Gene Therapy with Innovative Nanocarriers for siRNA and miRNA Delivery.
Chaudhary JS, Chanchal DK, Singh K … +5 more , Gupta JK, Jain D, Dubey A, Pandey M, Khan S
Curr Gene Ther · 2026 · PMID 40231502 · Publisher ↗
The cells have been given precise instructions proprio to the regulation of gene expression by the main genesis of Ryan-based gene therapy, which has revived cancer treatment and other disorders. The difficulty of delive...
The cells have been given precise instructions proprio to the regulation of gene expression by the main genesis of Ryan-based gene therapy, which has revived cancer treatment and other disorders. The difficulty of delivering small interfering RNA (siRNA) and microRNA (miRNA) to a target cell is an enormous task and is often faced by researchers due to characteristic instabilities of these carriers and their poor uptake by the cell membrane. The new developments from nanocarrier technologies offer opportunities for better effectiveness of RNA therapy for its delivery and the effectiveness of the treatment regimen. The objective of this article is to provide an overview of the existing as well as the newest developments in nanocarrier technology, particularly as related to microRNA and small interfering RNA (siRNA) delivery. Their modes of operation and their uses in gene therapies are also examined as principles of their design. We focus on several nanocarrier technologies, which have shown proof of concept in multiple disciplines such as stability, controlled release profiles, and delivery. Lipid-based nanoparticles, polymeric systems, and hybrid nanocarriers are some of the platforms that fall under this category; however, this list is not exhaustive. We also study the idea that certain nanocarriers could have multiple functionalities, which would make it possible to improve cancer treatment by simultaneously carrying chemotherapy and genes. We aim to shed light on the future of RNA-based gene therapy by providing a thorough overview of recent research in the field. This will help us understand how novel nanocarrier technologies can tackle the delivery issues.
Expression of miR-92a in Green Tea EGCG Preconditioned Adiposederived Stem Cells Ameliorates Inflammatory Response and Increases Cartilage Regeneration in RA Rats through KLF4/IL-17/MMP-2 Axis Modulation.
Chen TS, Kuo WW, Huang CY
Curr Gene Ther · 2025 · PMID 40183268 · Publisher ↗
BACKGROUND: The global prevalence of rheumatoid arthritis (RA) is on the rise. Numerous studies have demonstrated the potential of stem cell-based therapies in RA treatment. Experimental evidence suggests that preconditi...
BACKGROUND: The global prevalence of rheumatoid arthritis (RA) is on the rise. Numerous studies have demonstrated the potential of stem cell-based therapies in RA treatment. Experimental evidence suggests that preconditioning enhances the regenerative capabilities of stem cells compared to their unconditioned counterparts. OBJECTIVE: This study aimed to evaluate whether adipose-derived stem cells (ADSCs) preconditioned with green tea epigallocatechin gallate (EGCG) and miR-92a exhibit superior therapeutic effects in RA compared to unconditioned ADSCs. METHODS: Both in vitro and in vivo models were employed. In the cellular model, ADSCs were preconditioned with EGCG and miR-92a. In the animal model, male Wistar rats were used, and RA was induced using the collagen-induced arthritis (CIA) model. Following RA induction, the animals were divided into six groups: Sham (healthy rats), RA (RA-induced rats), RA+ADSC (RA-induced rats receiving unconditioned ADSCs), RA+E-ADSC (RA-induced rats receiving EGCGpreconditioned ADSCs), RA+mic-ADSC (RA-induced rats receiving miR-92a mimicpreconditioned ADSCs), and RA+inh-ADSC (RA-induced rats receiving miR-92a inhibitorpreconditioned ADSCs). RESULTS: In the cellular model, preconditioning with EGCG and miR-92a activated the CXCR4/p- Akt signaling pathway, thereby enhancing ADSC viability. In the animal model, RA induction caused several joint pathologies, including hind paw swelling, disrupted bone metabolism, immune cell infiltration, increased expression of IL-17, KLF4, and IL-6, as well as cartilage degradation. While transplantation of unconditioned ADSCs modestly improved these pathological features, the administration of E-ADSCs and mic-ADSCs significantly ameliorated these conditions in RA rats. Conversely, the therapeutic effects of E-ADSCs and mic-ADSCs were attenuated by the transplantation of inh-ADSCs. CONCLUSION: The therapeutic effects of E-ADSCs and mic-ADSCs in RA were strongly associated with the modulation of the KLF4/IL-17/MMP-2 axis. These findings suggest that ADSCs preconditioned with EGCG and miR-92a hold considerable clinical promise for the treatment of RA.
Riding the Wave of Progress: Examining the Current Landscape and Future Potential of MicroRNAs in Cancer Gene Therapy.
Ahmad S, Khan H, Muthoni NI … +7 more , Alissa M, Safhi AY, Sabei FY, Abdulaziz O, Khan KH, Ibrahim EH, Khan SU
Curr Gene Ther · 2026 · PMID 40129155 · Publisher ↗
MicroRNAs, commonly referred to as miRNAs, exert a significant impact on cellular processes by coordinating post-transcriptional gene regulation. These non-coding RNAs, which are only 22 nucleotides long, form a part of...
MicroRNAs, commonly referred to as miRNAs, exert a significant impact on cellular processes by coordinating post-transcriptional gene regulation. These non-coding RNAs, which are only 22 nucleotides long, form a part of the RNA-induced silencing complex (RISC) and play a crucial role in regulating gene expression. Their complex participation in cell proliferation, differentiation, and death highlights their crucial role in maintaining cellular balance. MicroRNAs have become significant contributors in the complex field of cancer biology, operating beyond the usual tasks of cells. Their dysregulation is closely intertwined with cancer initiation and development. miRNAs act as cellular regulators and regulate complex processes of gene expression. Disruption of this regulation can result in tumor development. This review article explores the intricate process of miRNA biosynthesis and its mechanisms, providing insights into its complex interactions with cancer. It also discusses the exciting field of miRNA-based cancer treatment. Exploring the therapeutic possibilities of these small RNA molecules presents opportunities for precision medicine, introducing a new age where miRNAs can be utilized to create targeted therapeutic interventions that mainly address the abnormal genetic characteristics that cause tumor formation. miRNAs provide a harmonious balance between understanding their biology and utilizing their therapeutic potential in cancer treatment. However, they also serve as conductors and possible therapeutic instruments in the symphony of molecular biology for gene therapy.
CRISPR-Cas9: Transforming Functional Genomics, Precision Medicine, and Drug Development - Opportunities, Challenges, and Future Directions.
Khan Z, Mumtaz, Gupta S … +7 more , Mehan S, Sharma T, Kumar M, Maurya PK, Sharma AK, Gupta GD, Narula AS
Curr Gene Ther · 2026 · PMID 40129147 · Publisher ↗
CRISPR-Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats) is a groundbreaking gene-editing technology that enables scientists to make precise changes to the DNA of living organisms. It was first discovered...
CRISPR-Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats) is a groundbreaking gene-editing technology that enables scientists to make precise changes to the DNA of living organisms. It was first discovered in Escherichia coli and emerged as a breakthrough tool in molecular biology. This technique is essential because of its adaptability, affordability, and ease of use. It uses the adaptive immune response of bacteria and archaea to repel viral invasions. It significantly influences drug discovery, functional genomics, disease models, and pharmaceutical research. CRISPR-Cas9 is a better and more accurate way to change genes than other methods, such as zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs). This technology promotes the generation of double-strand breaks in DNA, allowing for precise genetic alterations required for therapeutic target identification and confirmation. Functional genomics enables high-throughput screening (HTS) to identify gene functions, disease causes, and therapeutic targets. CRISPR-Cas9 increases drug development by enabling Cas9 to create novel antimicrobial drugs and cancer therapies. It has also helped to generate disease models, advance our understanding of neurodegenerative and other diseases, test a variety of chemicals, and facilitate precise genetic changes. Despite its promise, ethical considerations and the possibility of off-target effects require careful evaluation to ensure its safe and effective clinical application. This study investigates the current and future possibilities of CRISPR-Cas9 in drug development, focusing on its transformational influence and addressing the challenges and limitations of its therapeutic application.
Polysaccharide-Based Delivery Systems for CRISPR/Cas Gene Therapy: Overcoming Challenges and Advancing Pharmaceutical Solutions.
Manchanda D, Kumar S, Makhija M
Curr Gene Ther · 2026 · PMID 40070071 · Publisher ↗
The advent of CRISPR/Cas gene-editing technology has revolutionized molecular biology, offering unprecedented precision and potential in treating genetic disorders, cancers, and other complex diseases. However, for CRISP...
The advent of CRISPR/Cas gene-editing technology has revolutionized molecular biology, offering unprecedented precision and potential in treating genetic disorders, cancers, and other complex diseases. However, for CRISPR/Cas to be truly effective in clinical settings, one of the most significant challenges lies in the delivery of the CRISPR components, including guide RNA (gRNA) and Cas protein, into specific cells or tissues. Safe, targeted, and efficient delivery remains a critical bottleneck. Viral vectors, lipid nanoparticles, and synthetic polymers have been explored, but they come with limitations, such as immunogenicity, toxicity, and limited delivery capacity. Polysaccharide-based delivery systems, with their natural origin, biocompatibility, and versatile chemical properties, offer a promising alternative that could address these delivery challenges while advancing the pharmaceutical applications of CRISPR/Cas gene therapy.
The Antimicrobial Peptide Pipeline: A Bacteria-Centric AMP Predictor.
Veldsman WP, Yuanjie Z, Zhang Q … +2 more , Zhao Q, Zhang L
Curr Gene Ther · 2025 · PMID 40012382 · Publisher ↗
INTRODUCTION: Antimicrobial peptides (AMPs), unlike antibiotics, are encoded in genomes. AMPs are exported from the cell after expression and translation. In the case of bacteria, the exported peptides target other micro...
INTRODUCTION: Antimicrobial peptides (AMPs), unlike antibiotics, are encoded in genomes. AMPs are exported from the cell after expression and translation. In the case of bacteria, the exported peptides target other microbes to give the producing bacterium a competitive edge. While AMPs are sought after for their similar antimicrobial activity to traditional antibiotics, it is difficult to predict which combinations of amino acids will confer antimicrobial activity. Many computer algorithms have been designed to predict whether a sequence of amino acids will exhibit antimicrobial activity, but the vast majority of validated AMPs in databases are still of eukaryotic origin. This defies common sense since the vast majority of life on Earth is prokaryotic. METHODS: The antimicrobial peptide pipeline, presented here, is a bacteria-centric AMP predictor that predicts AMPs by taking design inspiration from the sequence properties of bacterial genomes with the intention to improve the detection of naturally occurring bacterial AMPs. The pipeline integrates multiple concepts of comparative biology to search for candidate AMPs at the primary, secondary, and tertiary peptide structure levels. RESULTS: Results showed that the antimicrobial peptide pipeline identifies known AMPs that are missed by state-of-the-art AMP predictors and that the pipeline yields more AMP candidates from real bacterial genomes than from fake genomes, with the rate of AMP detection being significantly higher in the genomes of six nosocomial pathogens than in the fake genomes. CONCLUSION: This bacteria-centric AMP pipeline enhances the detection of bacterial AMPs by incorporating sequence properties unique to bacterial genomes. It complements existing tools, addressing gaps in AMP detection and providing a promising avenue for discovering novel antimicrobial peptides.
Targeting of YKL-40 as a Protumor in Personalized Medicine: A New Dimension in Disease Understanding.
Moustafa HAM, Sohaib AU, Saleem I … +1 more , Ullah A
Curr Gene Ther · 2026 · PMID 39976089 · Publisher ↗
BACKGROUND: Individualization of the therapeutic plan for cancer patients is the essence of modern clinical practice. Standard cancer diagnostic and prognostic factors are invasive, and their value for the stratification...
BACKGROUND: Individualization of the therapeutic plan for cancer patients is the essence of modern clinical practice. Standard cancer diagnostic and prognostic factors are invasive, and their value for the stratification of cancer patients with a higher risk of local or distant recurrence is limited. YKL-40 is a protumor glycoprotein linked to the immunosuppressive tumor in a microenvironment and an important biomarker of cell activation, proliferation, and migration. OBJECTIVE: The objective is to update the review, and molecular and clinical research should investigate novel modalities of targeting this glycoprotein for cancer treatment. METHODS: Relevant studies published in the English language were identified by searching PubMed, Google Scholar, and MEDLINE from January 2000 to December 2023. Published studies that specifically elicited the role of YKL-40 as a biomarker in different types of tumors were included. RESULTS: YKL-40 cancer prognostic effect was reported in various cancer types. CONCLUSION: Since antibodies against YKL-40 can inhibit tumor angiogenesis and cancer progression, it can be suggested as an attractive candidate for chemical cancer therapy and immunomodulation.
Gene Therapy and Gene Editing: Current Trends and Future Prospects of Molecular Medicine.
Sen A, Singh V, Dwivedi S … +4 more , Agrawal R, Bansal A, Shekhar S, Kushwaha SS
Curr Gene Ther · 2026 · PMID 39976035 · Publisher ↗
Gene therapy and genome editing have emerged as transformative approaches in the management of a diverse range of genetic and acquired diseases. This evaluation offers a thorough examination of the present state and pros...
Gene therapy and genome editing have emerged as transformative approaches in the management of a diverse range of genetic and acquired diseases. This evaluation offers a thorough examination of the present state and prospects of these innovative technologies. Gene therapy is a prospective approach to the treatment and prevention of a variety of conditions, including complex cancers and inherited genetic disorders, which entail the introduction, removal, or modification of genetic material within a patient's cells. Genome editing, particularly through techniques such as CRISPR-Cas9, enables targeted corrections of genetic defects and opens new possibilities for personalized medicine by allowing for precise modifications at the DNA level. The review addresses the ethical implications, clinical applications, and significant advancements of these technologies. This article endeavors to underscore the substantial influence of gene therapy and genome editing on contemporary medicine by assessing the most recent research and clinical trials, thereby emphasizing their potential to revolutionize disease treatment and management.
Parkinson's Disease: From Bench to Bedside-Advancements in Diagnosis and Therapeutics using Pharmacogenomic Approach.
Suhas M, Kadiri SK
Curr Gene Ther · 2026 · PMID 39945257 · Publisher ↗
This study provides a detailed overview of Parkinson's disease (PD), a neurodegenerative ailment mostly known for movement difficulties such tremor, stiffness, and bradykinesia, which affects approximately 1% of persons...
This study provides a detailed overview of Parkinson's disease (PD), a neurodegenerative ailment mostly known for movement difficulties such tremor, stiffness, and bradykinesia, which affects approximately 1% of persons over the age of 60. Although the precise cause of PD is still unknown, various factors such as pesticide exposure, genetics, and lifestyle choices like smoking and caffeine consumption are thought to play a role in its development. The presence of Lewy bodies characterizes the disease, the aggregation of alpha-synuclein, the loss of dopaminergic neurons in the substantia nigra, and disruptions in basal ganglia circuitry, resulting in both motor and nonmotor symptoms. This review is structured into several key sections, beginning with an exploration of the pathophysiological mechanisms behind PD, including how genetic mutations can lead to deficits in the Ubiquitin Proteasome System and mitochondrial function, which are linked to familial cases of the disease. Following this, the review explores diagnostic methods, such as the UK Brain Bank Criteria, advanced imaging techniques, olfactory testing, and innovative technologies like machine learning, all of which support early detection and accurate diagnosis of PD. Treatment strategies are also comprehensively reviewed, focusing on traditional pharmacological options like levodopa and dopamine agonists, as well as surgical interventions such as deep brain stimulation. Additionally, the review discusses promising new therapies, including immunotherapy aimed at neuroinflammation and gene therapy for disease modification. The impact of lifestyle changes such as exercise and diet on reducing PD risk and enhancing symptom management are also considered. In conclusion, this review highlights the complex nature of Parkinson's disease and underscores the need for a holistic approach that combines pharmacotherapy, advanced treatments, and lifestyle adjustments. By addressing both symptom management and disease modification, these strategies provide hope for improving quality of life.
Gene Therapy: Transforming the Battle Against Pancreatic Cancer.
Sharma R, Kumar S, Ghosh R … +2 more , Komal K, Kumar M
Curr Gene Ther · 2026 · PMID 39936422 · Publisher ↗
Pancreatic cancer remains one of the most aggressive and lethal malignancies, with a dismal prognosis despite advancements in conventional treatment modalities. Gene therapy has emerged as a promising approach to combat...
Pancreatic cancer remains one of the most aggressive and lethal malignancies, with a dismal prognosis despite advancements in conventional treatment modalities. Gene therapy has emerged as a promising approach to combat pancreatic cancer by targeting the underlying genetic alterations and harnessing the power of the immune system. This review explores the current landscape of gene therapy strategies for pancreatic cancer, including gene replacement therapy, gene silencing, immunotherapy enhancement, and oncolytic virotherapy. Gene replacement therapy aims to restore the function of tumor suppressor genes, such as TP53, while gene silencing targets oncogenes like KRAS (Kirsten rat sarcoma viral oncogene homolog) to inhibit tumor growth. Immunotherapy enhancement, particularly through chimeric antigen receptor (CAR) T-cell therapy, has shown potential in overcoming the immunosuppressive tumor microenvironment. Oncolytic viruses, engineered to replicate in and destroy cancer cells selectively, have demonstrated efficacy in preclinical models and are being evaluated in clinical trials. Recent advances, including the successful treatment of a patient with advanced pancreatic cancer using neoantigen T-cell receptor gene therapy, highlight the potential of personalized gene therapy approaches. However, challenges such as precise gene delivery, tumor heterogeneity, and ethical considerations must be addressed to realize the potential of gene therapy for pancreatic cancer fully. Ongoing research and clinical trials are expected to facilitate the way for the development of safe and effective gene therapies, offering hope for improved outcomes in pancreatic cancer.
CD1B Expression in Triple-Negative Breast Cancer: Its Implications for Prognosis and Immunotherapy Outcomes.
Jin H, Wan M, Pan S … +4 more , Wang Z, Wang W, Zhang J, Han X
Curr Gene Ther · 2025 Feb · PMID 39936421 · Publisher ↗
INTRODUCTION: The absence of the estrogen receptor (ER), progesterone receptor (PR), and human epidermal growth factor receptor 2 (HER2) is a hallmark of triple-negative breast cancer (TNBC), which results in fewer treat...
INTRODUCTION: The absence of the estrogen receptor (ER), progesterone receptor (PR), and human epidermal growth factor receptor 2 (HER2) is a hallmark of triple-negative breast cancer (TNBC), which results in fewer treatment options and inferior clinical outcomes. The major histocompatibility complex family includes CD1B. By exposing T cells to lipid antigens, it alters immunological responses. Although the function of CD1B has been investigated in a number of malignancies, its relevance in TNBC has not been fully investigated. METHOD: In this study, immunohistochemistry (IHC) analysis of tissue samples and public databases was carried out to examine the expression of CD1B and its implications for prognosis in TNBC. RESULT: Compared to normal tissues, TNBC tissues demonstrated significantly higher levels of CD1B expression. Better overall survival, including survival without distant metastases and survival without recurrence, was found to be associated with higher levels. Additionally, more immune cells, primarily memory B cells and regulatory T cells, entering the TNBC region were found to be associated with greater levels of CD1B. It was found that the immunological microenvironment of TNBC was significantly affected by CD1B. The association between CD1B and immune-related pathways was also identified by examining functional enrichment. Drug sensitivity can be used to identify potential CD1B-targeting therapies. According to these results, CD1B might be a useful prognostic indicator and a possible target for treatment in TNBC. CONCLUSION: Nevertheless, additional experimental verification is required to verify the clinical significance of CD1B.
Neurotrophins as Potential Gene Therapy Targets for Huntington's Disease.
Roy SK, Barman A, Sarkar KL … +2 more , Richard SA, Lacmane BA
Curr Gene Ther · 2025 · PMID 39934995 · Publisher ↗
"Huntington's disease" (HD) is an autosomal dominant hereditary neurodegenerative disease characterized by defects in efferent striatal neurons, cortical neurons, and the basal ganglia. The pathogenesis of HD is still un...
"Huntington's disease" (HD) is an autosomal dominant hereditary neurodegenerative disease characterized by defects in efferent striatal neurons, cortical neurons, and the basal ganglia. The pathogenesis of HD is still unclear, and there is currently no curative therapy for this disorder. This review emphasizes the potential beneficial effects of various neurotrophic factors in HD. PubMed, Web of Science, Embase, and google scholar databases were used to search for all studies on the efficacy of neurotrophic factors in HD. Several gene therapy strategies have been employed to treat HD, including gene therapy with a variety of neuroprotective factors. Moreover, a wide variability of gene therapy approaches such as a neurotrophin, has shown promising results for both prevention and neuroprotection in HD, which may be due to their potential to prevent neuronal cell death or decrease neurodegeneration, thereby promoting the growth of innovative axons, dendrites, and synapses leading to improvement of HD. Neurotrophic factors may be suitable as neuroprotective therapy agents in HD. Therefore, substantial research on gene therapy should be conducted to provide better treatment options for HD in the future.
Targeting Undruggable Proteins: The siRNA Revolution Beyond Small Molecules - Advances, Challenges, and Future Prospects in Therapeutic Innovation.
Jan SA, Debnath A, Singh RK … +3 more , Tyagi PK, Singh S, Singh AK
Curr Gene Ther · 2026 · PMID 39917924 · Publisher ↗
The field of drug discovery has long been challenged by the existence of "undruggable" proteins - targets that have resisted traditional small molecule approaches due to their structural or functional characteristics. Th...
The field of drug discovery has long been challenged by the existence of "undruggable" proteins - targets that have resisted traditional small molecule approaches due to their structural or functional characteristics. This review explores the revolutionary potential of small interfering RNA (siRNA) technology in addressing these elusive targets, marking a paradigm shift in therapeutic development. We discuss the historical development of siRNA technology and its unique mechanism of action, which allows for the silencing of virtually any gene, including those coding for proteins previously deemed undruggable. The review provides a comprehensive analysis of the challenges in targeting undruggable proteins and how siRNA approaches are overcoming these obstacles. We examine several case studies of undruggable targets being successfully addressed by siRNA, including oncogenic proteins like KRAS and c-Myc, transcription factors such as NF-κB and STAT3, and proteins involved in complex protein-protein interactions. The article delves into the latest advances in siRNA design, delivery systems, and targeting strategies, highlighting innovations that enhance specificity and reduce off-target effects. We also discuss the challenges facing siRNA therapeutics, including delivery obstacles, potential immune responses, and regulatory considerations. The review concludes with an exploration of future directions, including combination therapies, personalized medicine approaches, and emerging technologies that complement siRNA strategies. By providing a thorough examination of the advances, challenges, and prospects of using siRNA to target undruggable proteins, this review underscores the transformative potential of this technology in expanding the landscape of therapeutic targets and ushering in a new era of precision medicine.
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