RECENT DEVELOPMENT IN GENE THERAPY-VECTORS AND DELIVERY SYSTEM
Miss. Suchita S. Lathi , Shruti Pramod Shinde , Tushar S. Sawant , Sudarshana R. Shelke, Prajakta B. Shinde, Isha R. Suryavanshi
Raosaheb Patil Danve College of Pharmacy , Badnapur, Jalna, Maharashtra
Abstract
Gene therapy is the transfer of a genetic material to treat a disease, or at least to improve the clinical status of a patient.viruses into genetic vectors carrying the gene of interest to the target cells is the aim of gene therapy. Based on the genome’s nature, these vectors are divided into RNA-based or DNA-based viral vectors .[1] Gene therapy has recently witnessed accelerated progress as a new therapeutic strategy with the potential to treat a range of inherited and acquired diseases. The process of gene therapy involves converting viruses into genetic vectors that transport the desired gene to the specific cells. These vectors are categorized as either RNA-based or DNA-based viral vectors depending on the genome's composition. Recently, there has been rapid advancement in gene therapy, which is emerging as a new method to potentially treat various inherited and acquired illnesses. Billions of dollars have been dedicated to basic and clinical research in gene medicine, with current clinical trials targeting cancer, monogenic diseases, cardiovascular diseases, and other difficult-to-treat illnesses. Gene therapy shows great potential as a molecular method to treat uncommon genetic disorders. Gene therapy works by restoring, replacing, inhibiting, and editing genes to correct the disease phenotype. Recent studies indicate that a growing percentage of gene therapy clinical trials are opting for viral vectors (64.2%) over non-viral vectors. Gene therapy of human genetic diseases essentially requires gene delivery systems. Gene therapy is a special technique that can use modified genes to treat any illness. Gene therapy shows great potential as a treatment for various conditions like genetic diseases, viral infections, and cancer. The efficacy of gene delivery systems is determined by the flexibility of targeting gene delivery systems. In recent times, several effective gene delivery systems have been successfully developed for the practical use of gene therapy. Gene therapy can permanently fix diseases by introducing normal genes to replace mutant gene deficiencies, suppressing mRNA of mutant alleles, and triggering cell death in cancer cells with transgenes that encode apoptosis-inducing proteins. Encouraging findings from clinical trials for eye disease and Parkinson's disease indicate that gene-based neurotherapeutics hold significant promise.
Keywords: Gene therapy, viral vector ,non viral vector, delivery system
Journal Name :
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EPRA International Journal of Multidisciplinary Research (IJMR)
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Published on : 2024-12-12
Vol | : | 10 |
Issue | : | 12 |
Month | : | December |
Year | : | 2024 |