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Current approaches in CRISPR-Cas systems for hereditary diseases.
Prog Mol Biol Transl Sci
January 2025
School of Health Sciences & Technology, UPES, Dehradun, Uttarakhand, India. Electronic address:
CRISPR-Cas technologies have drastically revolutionized genetic engineering and also dramatically changed the potential for treating inherited disorders. The potential to correct genetic mutations responsible for numerous hereditary disorders from single-gene disorders to complex polygenic diseases through precise DNA editing is feasible. The tactic now employed in CRISPR-Cas systems for treating inherited disorders is the usage of particular guide RNAs to target and edit disease-causing mutations in the patient's genome.
View Article and Find Full Text PDFIn vitro ILC differentiation from human HSCs.
Methods Cell Biol
January 2025
Innate Lymphoid Cells Unit, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
The Innate Lymphoid Cells (ILCs) are a family of innate immune cells composed by the Natural Killer (NK) cells and the helper ILCs (hILCs) (ILC1, ILC2, ILC3), both developing from a common ILC precursor (ILCP) derived from hematopoietic stem cells (HSCs). A correct ILC reconstitution is crucial, particularly in patients receiving HSC transplantation (HSCT), the only therapeutic option for many adult and pediatric high-risk hematological malignancies. Indeed, mainly thanks to their cytotoxic activity, NK cells have a strong Graft-versus-Leukemia (GvL) effect.
View Article and Find Full Text PDFSynaptic protein expression in bipolar disorder patient-derived neurons implicates PSD-95 as a marker of lithium response.
Neuropharmacology
January 2025
Department of Psychiatry and Center for Circadian Biology, University of California San Diego, La Jolla, CA, USA; VA San Diego Healthcare System, San Diego, CA, USA. Electronic address:
Bipolar disorder (BD) is a severe mental illness characterized by recurrent episodes of depression and mania. Lithium is the gold standard pharmacotherapy for BD, but outcomes are variable, and the relevant therapeutic mechanisms underlying successful treatment response remain uncertain. To identify synaptic markers of BD and lithium response, we measured the effects of lithium on induced pluripotent stem cell-derived neurons from BD patients and controls.
View Article and Find Full Text PDFBimodal In Situ Analyzer for Circular RNA in Extracellular Vesicles Combined with Machine Learning for Accurate Gastric Cancer Detection.
Adv Sci (Weinh)
January 2025
Department of Laboratory Medicine, Guangdong Provincial Key Laboratory of Precision Medical Diagnostics， Guangdong Engineering and Technology Research Center for Rapid Diagnostic Biosensors， Guangdong Provincial Key Laboratory of Single Cell Technology and Application, School of Laboratory Medicine and Biotechnology, Nanfang Hospital, Southern Medical University, Guangzhou, Guangdong, 510515, P. R. China.
Circular RNAs in extracellular vesicles (EV-circRNAs) are gaining recognition as potential biomarkers for the diagnosis of gastric cancer (GC). Most current research is focused on identifying new biomarkers and their functional significance in disease regulation. However, the practical application of EV-circRNAs in the early diagnosis of GC is yet to be thoroughly explored due to the low accuracy of EV-circRNAs analysis.
View Article and Find Full Text PDFCorrection: Astaxanthin and DHA Supplementation Modulates the Maternal Undernutrition-induced Impairment of Cognitive Behavior and Synaptic Plasticity in Adult Life of Offspring's -Exploring the Molecular Mechanism.
Mol Neurobiol
January 2025
Nitte (Deemed to be University), Department of Physiology, KS Hegde Medical Academy, Deralakatte, Mangalore, Karnataka, 575018, India.
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