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.

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• Local manufacturing processes contribute to variability in human mesenchymal stromal cell expansion while growth media supplements contribute to variability in gene expression and cell function: a Biomedical Excellence for Safer Transfusion (BEST) collaborative study Sun, 03 Dec 2023 06:00:00 -0500
  CONCLUSIONS: Local manufacturing process plays a critical role in MSC expansion while growth media may influence function and gene expression. All HPL and FBS products supported cell growth.


• Human CD4+CD25+ T cells expressing a chimeric antigen receptor against aberrant superoxide dismutase 1 trigger antigen-specific immunomodulation Sun, 03 Dec 2023 06:00:00 -0500
  CONCLUSIONS: Taken together, this study demonstrates that gene-modified Tregs can be developed to target an aggregated ALS-relevant protein to elicit CAR-mediated Treg effector functions and provides an approach for generating Treg therapies for ALS with the goal of enhanced disease site-specific immunomodulation.


• Continued improvement in disease manifestations of acid sphingomyelinase deficiency for adults with up to 2 years of olipudase alfa treatment: open-label extension of the ASCEND trial Sat, 02 Dec 2023 06:00:00 -0500
  CONCLUSION: Treatment with olipudase alfa is well tolerated and reduces manifestations of chronic ASMD with sustained efficacy. Trial registration NCT02004691 registered 9 December 2013, https://clinicaltrials.gov/ct2/show/NCT02004691.


• IGF2 prevents dopaminergic neuronal loss and decreases intracellular alpha-synuclein accumulation in Parkinson's disease models Sat, 02 Dec 2023 06:00:00 -0500
  Parkinson's disease (PD) is the second most common late-onset neurodegenerative disease and the predominant cause of movement problems. PD is characterized by motor control impairment by extensive loss of dopaminergic neurons in the substantia nigra pars compacta (SNpc). This selective dopaminergic neuronal loss is in part triggered by intracellular protein inclusions called Lewy bodies, which are composed mainly of misfolded alpha-synuclein (α-syn) protein. We previously reported insulin-like...


• Photodynamic Therapy in Treating a Subset of Basal Cell Carcinoma: Strengths, Shortcomings, Comparisons with Surgical Modalities, and Potential Role as Adjunctive Therapy Sat, 02 Dec 2023 06:00:00 -0500
  Basal cell carcinoma (BCC) is the most common skin cancer, for which there are multiple treatment options, including the gold standard Mohs micrographic surgery (MMS), surgical excision, electrodesiccation and curettage, radiation therapy, cryosurgery, and photodynamic therapy (PDT). While PDT is currently approved for treating actinic keratosis, it has been used off-label to treat BCC patients who may not tolerate surgery or other treatment modalities. We present a review of the efficacy of...


• Efficacy of radial shock wave therapy on rat models of adjuvant arthritis Sat, 02 Dec 2023 06:00:00 -0500
  CONCLUSIONS: RPW improved the mechanical hypersensitivity between days 7 and 14 after application. Like FSW, RPW also induced the degeneration of sensory nerve fibers in the skin in the early period after irradiation, and reinnervation occurred between 14 and 28 days. Thus, our results demonstrate one of the pain relief mechanisms after RPW application.


• FGF12: biology and function Sat, 02 Dec 2023 06:00:00 -0500
  Fibroblast growth factor 12 (FGF12) belongs to the fibroblast growth factor homologous factors (FHF) subfamily, which is also known as the FGF11 subfamily. The human FGF12 gene is located on chromosome 3 and consists of four introns and five coding exons. Their alternative splicing results in two FGF12 isoforms - the shorter 'b' isoform and the longer 'a' isoform. Structurally, the core domain of FGF12, is highly homologous to that of the other FGF proteins, providing the classical tertiary...


• Circulating Biomarkers and Risk of Hypertension: A Two-Sample Mendelian Randomisation Study Sat, 02 Dec 2023 06:00:00 -0500
  CONCLUSIONS: This two-sample Mendelian randomisation study found that SMOC1 and TIE1 are causally linked to hypertension, making them a promising target for therapy.


• Stepwise Introduction of Elexacaftor-Tezacaftor-Ivacaftor in Patients With Cystic Fibrosis and Liver Cirrhosis Child-Pugh A or B Using Clinical and Therapeutic Drug Monitoring: A Case Series Sat, 02 Dec 2023 06:00:00 -0500
  PURPOSE: Cystic fibrosis (CF) is a monogenetic disease caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein and affecting multiple organs, including the lungs and liver. Almost 90% of people affected carry at least 1 Phe508del CFTR mutation. Medical treatment with the CFTR-modulating drug elexacaftor-tezacaftor-ivacaftor (ETI) has been proven to be efficacious in carriers of at least 1 Phe508del CFTR mutation. Use of ETI in patients with...


• Recent approaches in regenerative medicine in the fight against neurodegenerative disease Sat, 02 Dec 2023 06:00:00 -0500
  Neurodegenerative diseases arise due to slow and gradual loss of structure and/or function of neurons and glial cells and cause different degrees of loss of cognition abilities and sensation. The little success in developing effective treatments imposes a high and regressive economic impact on society, patients and their families. In recent years, regenerative medicine has provided a great opportunity to research new innovative strategies with strong potential to treatleva these diseases. These...


• Supramolecular self-assembled peptide-engineered nanofibers: A propitious proposition for cancer therapy Sat, 02 Dec 2023 06:00:00 -0500
  Cancer is a devastating disease that causes a substantial number of deaths worldwide. Current therapeutic interventions for cancer include chemotherapy, radiation therapy, or surgery. These conventional therapeutic approaches are associated with disadvantages such as multidrug resistance, destruction of healthy tissues, and tissue toxicity. Therefore, there is a paradigm shift in cancer management wherein nanomedicine-based novel therapeutic interventions are being explored to overcome the...


• LINC00319: Unraveling the Spectrum from Gene Regulation to Clinical Applications in Cancer Progression Sat, 02 Dec 2023 06:00:00 -0500
  LncRNAs are RNA transcripts that exceed 200 nucleotides in length and do not encode proteins. LINC00319 is a type of lncRNA that is highly expressed in various cancers and is regulated by CCL18 and MYC. High levels of LINC00319 are associated with poorer prognosis and more malignant clinical features in cancer patients. LINC00319 can regulate the expression of downstream genes, including 2 protein-coding genes and 11 miRNAs. It participates in controlling three signaling pathways and various...


• Detecting EGFR gene amplification using a fluorescence in situ hybridization platform based on digital microfluidics Sat, 02 Dec 2023 06:00:00 -0500
  Signal transduction mediated by epidermal growth factor receptor (EGFR) gene affects the proliferation, invasion, metastasis, and angiogenesis of tumor cells. In particular, non-small cell lung cancer (NSCLC) patients with increased in copy number of EGFR gene are often sensitive to tyrosine kinase inhibitors. Despite being the standard for detecting EGFR amplification in the clinic, fluorescence in situ hybridization (FISH) traditionally involves repetitive and complex benchtop procedures that...


• Regulatory role of RGMb in lung injury promoted by the combination of carbon ion irradiation and anti-PD-1 antibody through Erk1/2 and p38 MAPK pathways Sat, 02 Dec 2023 06:00:00 -0500
  The combination of carbon ion radiotherapy and anti-PD-1 antibody represents a new approach to treating thoracic tumors. However, the lung damage caused by this combination therapy may limit its use, and the potential mechanisms for this are worthy of investigation. The objective of this research was to examine the potential involvement of repulsive guidance molecule b (RGMb) in lung damage promoted by the utilization of carbon ion irradiation combined with an anti-PD-1 antibody. The C57BL/6...


• Stem cell therapy with CRISPR/Cas9-mediated MALAT1 delivery modulates miR-142 and rescues wound healing in rats with age-associated diabetic foot ulcers Sat, 02 Dec 2023 06:00:00 -0500
  CONCLUSIONS: MALAT1 in human umbilical cord mesenchymal stem cells expedited foot ulcer healing in diabetic rats, particularly in age-associated diabetes, through miR-142 sponge activity. These findings offer insights for novel therapeutic strategies targeting elderly diabetic foot ulcers, emphasizing exogenous stem cell transplantation's potential in effective DFU treatment for the elderly.


• Radioiodine-refractory differentiated thyroid cancer: Molecular mechanisms and therapeutic strategies for radioiodine resistance Sat, 02 Dec 2023 06:00:00 -0500
  Radioiodine-refractory differentiated thyroid cancer (RAIR-DTC) is difficult to treat with radioactive iodine because of the absence of the sodium iodide transporter in the basement membrane of thyroid follicular cells for iodine uptake. This is usually due to the mutation or rearrangement of genes and the aberrant activation of signal pathways, which result in abnormal expression of thyroid-specific genes, leading to resistance of differentiated thyroid cancer cells to radioiodine therapy....


• VEGF-C-expressing TAMs rewire the metastatic fate of breast cancer cells Sat, 02 Dec 2023 06:00:00 -0500
  The expression of pro-lymphangiogenic VEGF-C in primary tumors is associated with sentinel lymph node metastasis in most solid cancer types. However, the impact of VEGF-C on distant organ metastasis remains unclear. Perivascular tumor-associated macrophages (TAMs) play a crucial role in guiding hematogenous spread of cancer cells by establishing metastatic pathways within the tumor microenvironment. This process supports breast cancer cell intravasation and metastatic dissemination. We show here...


• Analyzing safety and effectiveness of Mavacamten in comparison with placebo for managing hypertrophic cardiomyopathy: a systemic review and meta-analysis Sat, 02 Dec 2023 06:00:00 -0500
  CONCLUSIONS: Our study suggests that Mavacamten may have therapeutic benefits for HCM patients, as indicated by its positive impact on certain endpoints. Further research with larger samples, longer follow-up, and comprehensive analysis is needed to understand Mavacamten's safety and efficacy in HCM patients.


• Multidisciplinary approach in cardiomyopathies: From genetics to advanced imaging Sat, 02 Dec 2023 06:00:00 -0500
  Cardiomyopathies are myocardial diseases characterized by mechanical and electrical dysfunction of the heart muscle which could lead to heart failure and life-threatening arrhythmias. Certainly, an accurate anamnesis, a meticulous physical examination, and an ECG are cornerstones in raising the diagnostic suspicion. However, cardiovascular imaging techniques are indispensable to diagnose a specific cardiomyopathy, to stratify the risk related to the disease and even to track the response to the...


• DNA on the move: mechanisms, functions and applications of transposable elements Sat, 02 Dec 2023 06:00:00 -0500
  Transposons are mobile genetic elements that have invaded all domains of life by moving between and within their host genomes. Due to their mobility (or transposition), transposons facilitate horizontal gene transfer in bacteria and foster the evolution of new molecular functions in prokaryotes and eukaryotes. As transposition can lead to detrimental genomic rearrangements, organisms have evolved a multitude of molecular strategies to control transposons, including genome defence mechanisms...