Somatic gene therapy based on nonviral and viral vectors is an attractive approach for treatment of human diseases. It has developed from preliminary animal experiments to clinical trials. Vectors and target genes used in gene therapy are mainly focused on viral, nonviral vector and single target gene or monogene and each vector system has a series of advantages and limitations. However, the application of gene therapy to human disease is currently hampered by potential hazards of methods of gene delivery, the relatively low efficiency and intracellular stability of target gene. Safety, efficacy and stability are three problems that people often met and also three principles that people should be followed in gene therapy.
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Clin Cancer Res
January 2025
Stanford University, Palo Alto, CA, United States.
Purpose: After failing primary and secondary hormonal therapy, castration-resistant and neuroendocrine prostate cancer metastatic to the bone is invariably lethal, although treatment with docetaxel and carboplatin can modestly improve survival. Therefore, agents targeting biologically relevant pathways in PCa and potentially synergizing with docetaxel and carboplatin in inhibiting bone metastasis growth are urgently needed.
Experimental Design: Phosphorylated (activated) AXL expression in human prostate cancer bone metastases was assessed by immunohistochemical staining.
Sci Transl Med
January 2025
Department of Cell Biology and Physiology, Washington University School of Medicine, Saint Louis, MO 63110, USA.
Sci Transl Med
January 2025
Graduate Program in Human Genetics, University of Miami Miller School of Medicine, 1501 NW 10th Avenue (M-860), Miami, FL 33136, USA.
Primary mitochondrial disorders are most often caused by deleterious mutations in the mitochondrial DNA (mtDNA). Here, we used a mitochondrial DddA-derived cytosine base editor (DdCBE) to introduce a compensatory edit in a mouse model that carries the pathological mutation in the mitochondrial transfer RNA (tRNA) alanine (mt-tRNA) gene. Because the original m.
View Article and Find Full Text PDFJ Med Chem
January 2025
Molecular Imaging Branch, National Institute of Mental Health, National Institutes of Health, Bethesda, Maryland, 20892 United States.
PLoS One
January 2025
Faculty of Sciences and Technology (FAST), Laboratory of Biology and Molecular Typing in Microbiology (LBTMM), University of Abomey-Calavi, Atlantic, Benin.
Background: Antiretroviral treatment increases the risk of accumulation of resistance mutations that negatively impact the possibilities of future treatment. This study aimed to present the frequency of HIV-1 antiretroviral resistance mutations and the genetic diversity among children with virological failure in five pediatric care facilities in Benin.
Methods: A cross-sectional study was carried out from November 20, 2020, to November 30, 2022, in children under 15 years of age who failed ongoing antiretroviral treatment at five facilities care in Benin (VL > 3log10 on two consecutive realizations three months apart).
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