Cells naturally sense and actively response to their environment. Cell-therapy has long been studied and shown therapeutic effects in various diseases. However, several hurdles should be overcome to improve cell-based therapy. Gene delivery-mediated cellular modification has shown improvement of cell function by obstacle gene silencing and therapeutic gene expression. Especially, CRISPR/Cas9-mediated genome editing is a very promising method for gene modification. In this review, we describe the recent advances in genetic modification for cell therapy. Stem cells are still promising source of cell therapy due to their self-renewal character and differentiation potential. Immune cells regulate the inflammatory response and immunization, which inspired various cell therapy using immune-regulatory cells. Conclusively, we emphasize the need to develop gene-modification-based cell therapy as potent future treatment.
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http://dx.doi.org/10.1007/s40005-017-0381-1 | DOI Listing |
Aging Dis
February 2025
International Center for Neuroscience Research, Institute for Intelligent Research, Tbilisi, Georgia.
This paper provides a thorough examination of aging-related diseases, exploring into the intricate molecular mechanisms that underline their development and progression. It explores cutting-edge therapeutic interventions aimed at addressing these conditions, with a particular focus on non-pharmacological approaches such as personalized lifestyle modifications, cognitive enhancement strategies, and robust social engagement initiatives. Additionally, it highlights emerging modalities including gene therapy and precision medicine as promising avenues for mitigating the challenges associated with age-related ailments.
View Article and Find Full Text PDFSmall
March 2025
State Key Laboratory of Advanced Medical Materials and Devices, Medical College, Tianjin University, Tianjin, 300072, China.
Irreversible electroporation (IRE) is a minimally invasive, non-thermal tumor ablation technique that induces nanoscale membrane perforation, leading to immunogenic cell death (ICD). However, IRE alone is limited by uneven electric field attenuation, incomplete tumor ablation, and the immunosuppressive nature of the tumor microenvironment. To address these challenges, a multifunctional nanomaterial, vermiculite nanosheets/calcium peroxide nanosheets (VMT/CaO NSs), is developed to enhance the efficacy of IRE.
View Article and Find Full Text PDFEpileptic Disord
March 2025
Division of Child Neurology, Department of Pediatrics, Ege University Medical Faculty, Izmir, Turkey.
Objective: To evaluate the significance of genetic testing in neonatal- and infantile-onset genetic epilepsies (NIGEP) for enhanced molecular diagnosis with management implications.
Methods: A single-center cohort of 128 patients with NIGEP (aged 0-36 months) from 2010 to 2022 was retrospectively assessed. The diagnostic utility of genetic testing, including next-generation sequencing (NGS) and chromosome-based approaches, was surveyed to determine their impact on antiseizure medication adjustments and precision medicine.
Adv Mater
March 2025
Centre for Craniofacial and Regenerative Biology, King's College London, London, SE1 1UL, UK.
Base editing, a CRISPR-based genome editing technology, enables precise correction of single-nucleotide variants, promising resolutive treatment for monogenic genetic disorders like recessive dystrophic epidermolysis bullosa (RDEB). However, the application of base editors in cell manufacturing is hindered by inconsistent efficiency and high costs, contributed by suboptimal delivery methods. Nanoneedles have emerged as an effective delivery approach, enabling highly efficient, non-perturbing gene therapies both in vitro and in vivo.
View Article and Find Full Text PDFCells
March 2025
SAMRC Precision Oncology Research Unit (PORU), DSI/NRF SARChI Chair in Precision Oncology and Cancer Prevention (POCP) Pan African Research Institute (PACRI), University of Pretoria, Hartfield, Pretoria 0028, South Africa.
The peremptory need to circumvent challenges associated with poorly differentiated epithelial endometrial cancers (PDEECs), also known as Type II endometrial cancers (ECs), has prompted therapeutic interrogation of the prototypically intractable and most prevalent gynecological malignancy. PDEECs account for most endometrial cancer-related mortalities due to their aggressive nature, late-stage detection, and poor response to standard therapies. PDEECs are characterized by heterogeneous histopathological features and distinct molecular profiles, and they pose significant clinical challenges due to their propensity for rapid progression.
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