Advancing gene editing therapeutics: Clinical trials and innovative delivery systems across diverse diseases
Mozhghan Raigani, Zohre Eftekhari, Ahmad Adeli, Fatemeh Kazemi‐Lomedasht
Abstract
modification of T cells and hematopoietic stem cells in autoimmune and infectious diseases. Despite encouraging results, challenges remain in optimizing delivery specificity, minimizing off-target effects, and ensuring long-term safety and efficacy. Ongoing and upcoming trials continue to refine these delivery technologies and expand the therapeutic reach of gene editing.
Topics & Concepts
Genome editingClinical trialGene deliveryMedicineGenetic enhancementComputational biologyDelivery systemBiologyGenePharmacologyInternal medicineCRISPRGeneticsCRISPR and Genetic EngineeringVirus-based gene therapy researchRNA Interference and Gene Delivery