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A Phase I, Open-label, Multicenter, Dose-Escalating Study to Evaluate the Safety and Tolerability of KH658 Gene Therapy in Participants with Neovascular Age-related Macular Degeneration
13 February 2025
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A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients with Type I Gaucher Disease
13 February 2025
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Latest Articles on Gene Therapy
Overview of latest articles and publications on gene therapy in PubMed, including Human Gene Therapy, Journal of Molecular Medicine and Journal of Gene Medicine. PubMed is a service of the US National Library of Medicine that includes over 18 million citations from MEDLINE and other life science journals.
- Pathophysiological mechanisms of ARDS: a narrative review from molecular to organ-level perspectives Thu, 13 Feb 2025 06:00:00 -0500
CONCLUSIONS: This narrative review provides fresh perspectives for future research, ultimately enhancing patient outcomes and optimizing management approaches in ARDS. - Clinical efficacy and therapy response prediction of neoadjuvant dalpiciclib plus letrozole in postmenopausal patients with HR+/HER2- stage II-III breast cancer (DARLING 01): a single-arm, open-label, exploratory study Thu, 13 Feb 2025 06:00:00 -0500
CONCLUSIONS: Dalpiciclib combined with letrozole demonstrated promising antitumor activity and an acceptable safety profile in postmenopausal patients with HR+/HER2- breast cancer. The identification of TFRC, SCUBE2, and MMP11A as predictive biomarkers provides insights into the potential for personalized neoadjuvant treatment strategies. - Investigator-initiated phase I trial of an oligonucleotide therapeutic targeting long noncoding RNA TUG 1 for recurrent glioblastoma Thu, 13 Feb 2025 06:00:00 -0500
BACKGROUND: Glioblastoma (GB) is the most common and aggressive primary malignant brain tumor in adults. To date, no effective treatment has been reported for recurrent GB (rGB). Long noncoding RNA taurine upregulated gene 1 (TUG1), which is highly expressed in GB, resolves the formation of R-loops, thereby maintaining tumor growth. TUG1-targeting antisense oligonucleotide (ASO) (TUG1ASO) is a nucleotide therapeutic with drug delivery system that targets TUG1, demonstrating efficacy against GB... - The haematopoietic cell transplantation comorbidity index predicts clinical outcomes for severe aplastic anaemia patients after haploidentical haematopoietic stem cell transplantation Thu, 13 Feb 2025 06:00:00 -0500
To validate the ability of the haematopoietic cell transplantation comorbidity index (HCT-CI) to predict the outcomes of patients with severe aplastic anaemia (SAA) receiving haploidentical haematopoietic stem cell transplantation (haplo-HSCT), we conducted a retrospective study including 530 SAA patients. Patients were stratified based on their HCT-CI scores into three distinct risk categories: low-risk (HCT-CI scores of 0, n = 343), intermediate-risk (HCT-CI scores of 1, n = 126), and... - Full-length transcriptome atlas of gallbladder cancer reveals trastuzumab resistance conferred by ERBB2 alternative splicing Thu, 13 Feb 2025 06:00:00 -0500
Aberrant RNA alternative splicing in cancer generates varied novel isoforms and protein variants that facilitate cancer progression. Here, we employed the advanced long-read full-length transcriptome sequencing on gallbladder normal tissues, tumors, and cell lines to establish a comprehensive full-length gallbladder transcriptomic atlas. It is of note that receptor tyrosine kinases were one of the most dynamic components with highly variable transcript, with Erb-B2 receptor tyrosine kinase 2... - Genomic Signatures of Recurrence After Resection of Early-Stage Node-Negative Colon Cancer Thu, 13 Feb 2025 06:00:00 -0500
CONCLUSION: This study identified novel genomic signatures that may improve risk stratification in early-stage node-negative CC, potentially guiding the selection of high-risk patients for adjuvant therapy. - De Novo MET-amplified NSCLC treated with savolitinib achieved remarkable tumor regression: a case report and review of literature Thu, 13 Feb 2025 06:00:00 -0500
Primary MET amplification is an infrequent tumorigenic driver gene alteration identified in pulmonary neoplasms. Data on the effectiveness of MET-tyrosine kinase inhibitor (TKI) therapy in de novo MET amplification are relatively scarce, and there remains a dearth of empirical evidence supporting the use of precision therapy as first-line treatment for advanced non-small cell lung cancer (NSCLC) with primary MET amplification. We present a case of advanced lung adenocarcinoma in an elderly... - Disulfidptosis links the pathophysiology of ulcerative colitis and immune infiltration in colon adenocarcinoma Thu, 13 Feb 2025 06:00:00 -0500
Ulcerative colitis (UC), a chronic inflammatory bowel disease, significantly increases the risk of colon adenocarcinoma (COAD). Disulfidptosis, a novel form of programmed cell death, has been implicated in various diseases, including UC. This study investigates the expression of disulfidptosis-related genes, particularly CD2AP and MYH10, in UC and COAD. Through analysis of public datasets, we found MYH10 significantly upregulated and CD2AP downregulated in UC compared to healthy controls, with... - Characterizing circulating biomarkers for childhood dementia disorders: A scoping review of clinical trials Thu, 13 Feb 2025 06:00:00 -0500
Childhood dementias, a group of neurological disorders are characterised by neurocognitive decline, with physical and psychosocial impacts for individuals. With therapy available for <5 % of childhood dementias, there is a high level of unmet need. Integration of biomarkers in clinical trials are important to characterize distinctive biological activities and interrogate targets for therapeutic development. This study reviewed four clinical trial registries to examine circulating biomarkers in... - Detection of Overlooked Rare EGFR Mutations in Non-small Cell Lung Cancer Using Multigene Testing Thu, 13 Feb 2025 06:00:00 -0500
CONCLUSIONS: ODxTT failed to identify 10 actionable EGFR mutations, accounting for 12.2% (10/82) of the cases initially reported as not carrying actionable mutations. Therefore, comprehensive genomic profiling should be actively performed early in cases with high clinical suspicion of EGFR mutations. - Oligogenic structure of amyotrophic lateral sclerosis has genetic testing, counselling and therapeutic implications Thu, 13 Feb 2025 06:00:00 -0500
CONCLUSIONS: Our findings represent the first large-scale, case-control assessment of oligogenicity in ALS and show that oligogenic events involving known ALS risk genes are relevant for disease risk in ~6% of ALS but not necessarily for disease onset and survival. This must be considered in genetic counselling and testing by ensuring to use comprehensive gene panels even when a pathogenic variant has already been identified. Moreover, in the age of stratified medication and gene therapy, it... - The Huntington's disease drug pipeline: a review of small molecules and their therapeutic targets Thu, 13 Feb 2025 06:00:00 -0500
Huntington's disease (HD) is a progressive neurodegenerative condition resulting from a CAG repeat expansion in the huntingtin gene (HTT). Recent advancements in understanding HD's cellular and molecular pathways have paved the way for identifying various effective small-molecule candidates to treat the disorder. Two small molecules, Tetrabenazine and Deutetrabenazine, are approved for managing chorea associated with HD, and several others are under clinical trials. Notably, the field of... - How 'miracle' weight-loss semaglutide promises to change medicine but can we afford the expense? Thu, 13 Feb 2025 06:00:00 -0500
Obesity is a complex and growing global concern, affecting one in eight individuals and compromising health, quality of life and life expectancy. It carries significant metabolic, cardiovascular, oncological, hepatorenal, skeletal and psychiatric risks, imposing substantial costs on health-care systems. Traditional treatments have often been ineffective or have led to relapse after lifestyle changes. Whereas bariatric surgery is effective, it also involves risks such as mortality and... - Central Hypothyroidism: Advances in Etiology, Diagnostic Challenges, Therapeutic Targets, and Associated Risks Thu, 13 Feb 2025 06:00:00 -0500
Central hypothyroidism is a rare condition characterized by insufficient production of thyroid hormones by the thyroid gland caused by defects in TSH secretion from the pituitary or TRH secretion from the hypothalamus. Its etiology includes genetic and acquired causes, mainly associated with tumors in the hypothalamic-pituitary region or treatments such as surgery and radiotherapy. This review discusses advances in its etiology, diagnosis, and treatment, with a focus on diagnostic and... - A review on pectin-based nanostructures for drug and gene delivery systems Thu, 13 Feb 2025 06:00:00 -0500
Pectin, a widely available and cost-effective polysaccharide, has garnered considerable attention in biomedical applications, particularly as a matrix for drug and gene delivery systems. Pectin possesses prominent properties such as biocompatibility, biodegradability, and low toxicity, which are desired features for delivery systems. Various pectin-based nanostructures have been applied to encapsulate therapeutic agents, drugs, and genetic materials with the potential of protecting them until... - Gut microbiota variation and diversity and gut graft-versus-host disease (GVHD) in pediatrics: A systematic review Thu, 13 Feb 2025 06:00:00 -0500
CONCLUSION: As a result, our findings indicated a decrease in diversity as well as a change in intestinal microbiota in children with GVHD under HSCT in most of the studies. - Cell membrane-camouflaged nanocarriers: A cutting-edge biomimetic technology to develop cancer immunotherapy Thu, 13 Feb 2025 06:00:00 -0500
The development and growth of many diseases are significantly influenced by immune dysregulation. Similarly, uncontrolled tumor growth occurs in cancer because the immune system is unable to identify and eradicate cancer cells. Therefore, to address this issue, cancer immunotherapy plays a crucial role in detecting tumors and inhibiting their growth. This immune-oncotherapy has gained significant interest over the last decade because of its relevant success in biomedical applications. The... - Sequential allogeneic HSCT after CAR-T therapy for relapsed/refractory acute lymphoblastic leukemia patients: A long-term follow-up result Thu, 13 Feb 2025 06:00:00 -0500
CONCLUSION: Our study exhibits favorable long-term outcomes consistent with those reported in clinical trials, with sustained, durable responses observed at the 4-year follow-up. However, these benefits are less pronounced in older patients and those with poor-risk disease characteristics. - Mechanisms of congenital hearing loss caused by GJB2 gene mutations and current progress in gene therapy Thu, 13 Feb 2025 06:00:00 -0500
GJB2 gene is a common pathogenic gene for non-syndromic hearing loss, located on chromosome 13q12.11, and primarily encodes connexin 26 (Cx26). Cx26, a member of the gap-junction protein family, is mainly expressed in the supporting cells of the cochlea, where it is responsible for intercellular material transfer and signal exchange. Gene therapy, a treatment method that repairs or reconstructs genetic material, has emerged as the most effective approach for hereditary hearing loss. During the... - Therapeutic PCSK9 targeting: Inside versus outside the hepatocyte? Thu, 13 Feb 2025 06:00:00 -0500
As a major regulator of LDL receptor (LDLR) activity and thus of LDL-cholesterol (LDL-C) levels, proprotein convertase subtilisin/kexin type 9 (PCSK9) represents an obvious therapeutic target for lipid lowering. The PCSK9 inhibitors, alirocumab and evolocumab, are human monoclonal antibodies (mAbs) that act outside the cell by complexing circulating PCSK9 and thus preventing its binding to the LDLR. In contrast, inclisiran, a small interfering RNA (siRNA), inhibits hepatic synthesis of PCSK9,...
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Gene Therapy News
- Reena Sharma, MD, on Assessing Spur Therapeutics’ Gene Therapy FLT201 in Gaucher Disease - CGTLive™
Fri, 14 Feb 2025 00:52:1 GMT - Japan researchers target degenerative eye disease with gene therapy - Nikkei Asia
Thu, 13 Feb 2025 22:33:0 GMT - Thyroid Cancer Market Size to Reach USD 1,892.4 Million by 2035, Impelled by Advancements in Gene Therapy and Personalized Medicine - BioSpace
Thu, 13 Feb 2025 19:53:0 GMT - Genprex's New Study Highlights NPRL2 Gene Therapy's Antitumor Activity In Lung Cancer - Nasdaq
Thu, 13 Feb 2025 18:59:0 GMT - Sanfilippo Syndrome Market Size to Reach USD 2.7 Billion by 2035, Impelled by Advancements in Gene Therapy - BioSpace
Thu, 13 Feb 2025 18:54:5 GMT