Rheumatoid arthritis (RA) is a systemic inflammatory joint disease affecting about 1% of the population worldwide. Current treatment approaches do not ensure a cure for every patient. Moreover, classical regimens are based on nontargeted systemic immune suppression and have significant side effects. Biological treatment has advanced considerably but efficacy and specificity issues remain. Gene therapy is one of the potential future directions for RA therapy, which is rapidly developing. Several gene therapy trials done so far have been of moderate success, but experimental and genetics studies have yielded novel targets. As a result, the arsenal of gene therapy tools keeps growing. Currently, both viral and nonviral delivery systems are used for RA therapy. Herein, we review recent approaches for RA gene therapy.
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http://dx.doi.org/10.3390/biomedicines8010009 | DOI Listing |
Ann Surg Oncol
January 2025
Department of Otolaryngology, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Background: Anaplastic thyroid cancer (ATC) is a highly lethal disease, often diagnosed with advanced locoregional and distant metastases, resulting in a median survival of just 3-5 months. This study determines the stratified effectiveness of baseline treatments in all combinations, enabling precise prognoses prediction and establishing benchmarks for advanced therapeutic options.
Methods: The study extracted a cohort of pathologically confirmed ATC patients from the Surveillance, Epidemiology, and End Results program.
Support Care Cancer
January 2025
Fudan University School of Nursing, Shanghai, China and Fudan University Centre for Evidence-Based Nursing: A Joanna Briggs Institute Centre of Excellence, 305 Fenglin Rd, Shanghai, 200032, China.
Purpose: Aromatase inhibitor-associated musculoskeletal symptoms (AIMSS) are the most common adverse effects experienced by breast cancer patients. This scoping review aimed to systematically synthesize the predictors/risk factors and outcomes of AIMSS in patients with early-stage breast cancer.
Methods: A systematic search was conducted in PubMed, Web of Science, EMBASE, CINAHL, and the China National Knowledge Internet (CNKI) from inception to December 2024 following the scoping review framework proposed by Arksey and O'Malley (2005).
Commun Med (Lond)
January 2025
Rare Disease Translational Center, The Jackson Laboratory, Bar Harbor, ME, USA.
Background: Multiple Sulfatase Deficiency (MSD) is a rare inherited lysosomal storage disorder characterized by loss of function mutations in the SUMF1 gene that manifests as a severe pediatric neurological disease. There are no available targeted therapies for MSD.
Methods: We engineered a viral vector (AAV9/SUMF1) to deliver working copies of the SUMF1 gene and tested the vector in Sumf1 knock out mice that generally display a median lifespan of 10 days.
NPJ Vaccines
January 2025
Jiangzhong Cancer Research Center, Jiangxi University of Chinese Medicine, Nanchang, Jiangxi, China.
Tumor-derived exosomes (TDEs) mediate oncogenic communication, which modifies target cells to reinforce a tumor-promoting microenvironment. TDEs support cancer progression by suppressing anti-tumor immune responses, promoting metastasis, and conferring drug resistance. Thus, targeting TDEs could improve the efficacy of anti-cancer treatments and control metastasis.
View Article and Find Full Text PDFJ Pathol Clin Res
January 2025
State Key Laboratory of Holistic Integrative Management of Gastrointestinal Cancers and National Clinical Research Center for Digestive Diseases, Xijing Hospital of Digestive Diseases, Fourth Military Medical University, Xi'an, PR China.
CXC chemokine receptor 4 (CXCR4) and programmed cell death-ligand 1 (PD-L1) are two critical molecules involved in the tumor immune microenvironment. However, the impact of platinum drugs, such as cisplatin, on CXCR4 or PD-L1 expression and the underlying mechanisms in gastric cancer (GC) remain unknown. Moreover, the correlation between their expression levels in GC remains elusive.
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