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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.
- Neutralizing antibodies to interferon alfa arising during peginterferon therapy of chronic hepatitis B in children and adults: Results from the HBRN trials Wed, 17 Apr 2024 06:00:00 -0400
CONCLUSIONS: The development of anti-IFNα nAbs during PegIFNα treatment diminishes responses to antiviral therapy. Understanding how and why anti-IFNα antibodies develop may allow for optimization of IFN-based therapy, which is critical given its renewed use in HBV-cure strategies. - Expression of LC3A, LC3B and p62/SQSTM1 autophagy proteins in hepatocellular carcinoma (HCC) tissues and the predicted microRNAs involved in the autophagy-related pathway Wed, 17 Apr 2024 06:00:00 -0400
CONCLUSIONS: We conclude that autophagy events are more active in HCC tissues compared to the adjacent non-cancer tissues. We also reported the possible role of several miRNAs in regulating autophagy-related genes in the autophagy pathway in HCC. This may contribute to the development of potential therapeutic targets for improving HCC therapy. Future investigations are warranted to validate the target genes reported in this study using a larger sample size and more targeted molecular technique. - National trends in the treatment of adult diffuse midline gliomas: a rare clinical scenario Wed, 17 Apr 2024 06:00:00 -0400
CONCLUSIONS: Despite being considered ineffective, chemotherapy is still administered in most adult patients diagnosed with DMG. Significant predictors of survival were Charlson-Deyo comorbidity index and race. - New therapeutic target molecules for gastric and gastroesophageal junction cancer Wed, 17 Apr 2024 06:00:00 -0400
Molecularly targeted therapy for receptor tyrosine kinases (RTKs) has faced limitations in gastric and gastroesophageal junction (G/GEJ) cancer except for HER2-targeted agents, possibly due to inappropriate assay selection that has hindered identification of sensitive patients, in addition to coexisting genetic abnormalities as well as intratumoral heterogeneity. Immunohistochemistry of RTKs has, thus, proved largely unsuccessful for patient selection, and detection of RTK gene amplification as... - Comparative performance of scFv-based anti-BCMA CAR formats for improved T cell therapy in multiple myeloma Wed, 17 Apr 2024 06:00:00 -0400
In multiple myeloma (MM), B cell maturation antigen (BCMA)-directed CAR T cells have emerged as a novel therapy with potential for long-term disease control. Anti-BCMA CAR T cells with a CD8-based transmembrane (TM) and CD137 (41BB) as intracellular costimulatory domain are in routine clinical use. As the CAR construct architecture can differentially impact performance and efficacy, the optimal construction of a BCMA-targeting CAR remains to be elucidated. Here, we hypothesized that varying the... - The role of thymic stromal lymphopoietin in cutaneous disorders Wed, 17 Apr 2024 06:00:00 -0400
Thymic Stromal Lymphopoietin (TSLP) is an important cytokine that invokes early immune responses. TSLP, an IL-7-like cytokine encoded by the TSLP gene, activates JAK1 and JAK2 signaling pathways, stimulating dendritic cells to induce inflammatory Th2 cells. This cytokine is associated with pruritus in various cutaneous disorders, particularly atopic dermatitis. Varying levels of the cytokine TSLP have been demonstrated in studies of different cutaneous disorders. Pharmacological treatment... - Orthobiologics in knee osteoarthritis, dream or reality? Wed, 17 Apr 2024 06:00:00 -0400
Cartilage restoration or repair, also known as orthobiologic therapy, is indicated after the failure of conservative or supportive treatment. However, there is paucity in evidence supporting the efficacy of orthobiologic therapy. The blood-derived products, such as platelet-rich plasma (PRP), is one of the commonly used orthobiologic therapy for knee osteoarthritis. Several studies have shown that PRP is superior to other treatments, but the anatomic changes are scarce. Treatment with... - Scoliosis and rare diseases: our experience with the Prader-Willi syndrome Wed, 17 Apr 2024 06:00:00 -0400
INTRODUCTION: Prader-Willi syndrome (PWS) represents a difficult challenge for spine surgeons, due to the association of a structural scoliosis, with a prevalence between 15 and 86%. Conservative therapy is a viable option, but surgery is increasingly becoming the treatment of choice. - Characterization of gene regulatory networks underlying key properties in human hematopoietic stem cell ontogeny Wed, 17 Apr 2024 06:00:00 -0400
Human hematopoiesis starts at early yolk sac and undergoes site- and stage-specific changes over development. The intrinsic mechanism underlying property changes in hematopoiesis ontogeny remains poorly understood. Here, we analyzed single-cell transcriptome of human primary hematopoietic stem/progenitor cells (HSPCs) at different developmental stages, including yolk-sac (YS), AGM, fetal liver (FL), umbilical cord blood (UCB) and adult peripheral blood (PB) mobilized HSPCs. These stage-specific... - Genomic landscape and clinical features of advanced thyroid carcinoma: a national database study in Japan Wed, 17 Apr 2024 06:00:00 -0400
CONCLUSION: Genetic abnormalities with treatment options were found in 62.7% of advanced thyroid carcinomas. TP53 abnormality was an independent poor prognostic factor for overall survival in differentiated thyroid carcinoma. The time to treatment failure for lenvatinib was not different based on genetic profile. - Metagenomic next-generation sequencing for the diagnosis of neurobrucellosis Wed, 17 Apr 2024 06:00:00 -0400
Objective: This study investigates the application of metagenomic next-generation sequencing (mNGS) in the diagnosis of neurobrucellosis (NB). Methods: We retrospectively analyzed patients diagnosed with NB who underwent cerebrospinal fluid (CSF) mNGS testing in Xijing Hospital from 2015 to 2021. Results: Among the 20 individuals included in the study, the serum rose bengal test was positive in 11 out of 16 cases, serum agglutination test was positive in 13 out of 16 cases, CSF culture was... - Reduced Efficacy of Glucagon-Like Peptide-1 Receptor Agonists Therapy in People With Type 1 Diabetes and Genetic Forms of Obesity Wed, 17 Apr 2024 06:00:00 -0400
CONCLUSIONS: The weight loss and glycemic lowering effects of GLP-1 RA therapy may be decreased in people with type 1 diabetes and obesity likely related to genetic causes. Further research is needed to understand GLP-1 RA mechanisms via energy regulating genes. - Gut microbiota plays a significant role in gout Wed, 17 Apr 2024 06:00:00 -0400
With the development of social economy, the incidence of gout is increasing, which is closely related to people's increasingly rich diet. Eating a diet high in purine, fat, sugar and low-fibre for a long time further aggravates gout by affecting uric acid metabolism. The renal metabolism mechanism of uric acid has been thoroughly studied. To find a new treatment method for gout, increasing studies have recently been conducted on the mechanism of intestinal excretion, metabolism and absorption of... - Effectiveness of standard treatments in non-small-cell lung cancer with METexon14 skipping mutation: a real-world study Wed, 17 Apr 2024 06:00:00 -0400
Aim: To assess real-world clinical outcomes with standard therapies for advanced non-small-cell lung cancer (aNSCLC) with METexon14 skipping mutation (METex14). Methods: In an oncologists-led retrospective review of medical records, data were abstracted and analyzed for patients initiating first-line (1L) systemic therapy after 1 January 2017. Results: In total 287 aNSCLC patients with METex14, the real-world best overall response rate was 73.4% for capmatinib (n = 146), 68.8% for immunotherapy... - Understanding and overcoming resistance to immunotherapy in genitourinary cancers Wed, 17 Apr 2024 06:00:00 -0400
The introduction of novel immunotherapies has significantly transformed the treatment landscape of genitourinary (GU) cancers, even becoming the standard of care in some settings. One such type of immunotherapy, immune checkpoint inhibitors (ICIs) like nivolumab, ipilimumab, pembrolizumab, and atezolizumab play a pivotal role by disturbing signaling pathways that limit the immune system's ability to fight tumor cells. Despite the profound impact of these treatments, not all tumors are... - Review on Emerging Therapeutic Strategies for Managing Cardiovascular Disease Wed, 17 Apr 2024 06:00:00 -0400
Cardiovascular disease (CVD) remains a foremost global health concern, necessitating ongoing exploration of innovative therapeutic strategies. This review surveys the latest developments in cardiovascular therapeutics, offering a comprehensive overview of emerging approaches poised to transform disease management. The examination begins by elucidating the current epidemiological landscape of CVD and the economic challenges it poses to healthcare systems. It proceeds to scrutinize the limitations... - Current treatment and novel insights regarding ROS1-targeted therapy in malignant tumors Wed, 17 Apr 2024 06:00:00 -0400
CONCLUSIONS: Further research on next-generation TKIs regarding the localization of ROS1 and its fusion partners, binding sites for targeted drugs, and coadministration with other drugs is required. The correlation between TKIs and chemotherapy or immunotherapy in clinical practice requires further study. - Translational Research and Health Equity: Gene Therapies for Sickle Cell Disease as a Case Study Wed, 17 Apr 2024 06:00:00 -0400
In August of 2023, the National Academies of Science, Engineering, and Medicine published a timely report titled "Toward Equitable Innovation in Health and Medicine: A Framework." Here, we review some of the key contributions of the report, focusing on two dimensions of equity: input equity and deployment equity. We then use the example of new gene therapies to treat sickle cell disease (SCD) as a case study of input and deployment equity in translational research. The SCD case study illustrates... - Promising therapeutic targets for the treatment of urine storage dysfunction: what's the status? Wed, 17 Apr 2024 06:00:00 -0400
INTRODUCTION: Opinions differ on what drugs have both a rationale and a development potential for the treatment of bladder storage dysfunction. - Neoadjuvant chemotherapy is associated with suppression of the B cell-centered immune landscape in pancreatic ductal adenocarcinoma Wed, 17 Apr 2024 06:00:00 -0400
Pancreatic ductal adenocarcinoma (PDAC) is typically diagnosed at advanced stages and associated with early distant metastasis and poor survival. Besides clinical factors, the tumor microenvironment (TME) emerged as a crucial determinant of patient survival and therapy response in many tumors, including PDAC. Thus, the presence of tumor-infiltrating lymphocytes and the formation of tertiary lymphoid structures (TLS) is associated with longer survival in PDAC. Although neoadjuvant therapy (NeoTx)...
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Gene Therapy News
- Tackling Cell and Gene Therapy Challenges - BioProcess Insider
Wed, 17 Apr 2024 21:29:1 GMT - AAN 2024: AskBio's gene therapy seen to ease motor symptoms - Parkinson's News Today
Wed, 17 Apr 2024 18:41:3 GMT - Lexeo wins fast track designation for Friedreich’s ataxia gene therapy - Pharmaceutical Technology
Wed, 17 Apr 2024 18:16:5 GMT - Dr Sarah Bajorek Highlights Integration, Evaluation Strategies for Gene Therapy Implementation - AJMC.com Managed Markets Network
Wed, 17 Apr 2024 17:39:2 GMT - Poseida Therapeutics Hosts Gene Therapy R&D Day Highlighting New Scientific Advancements and Pipeline Focus - BioSpace
Wed, 17 Apr 2024 16:04:0 GMT