01 April 2005 : Case report
Vectors and delivery systems in gene therapy
Roman Gardlík, Roland Pálffy, Július Hodosy, Ján Lukács, Ján Turna, Peter CelecMed Sci Monit 2005; 11(4): RA110-121 :: ID: 15907
Abstract
Gene therapy provides modern medicine with new perspectives that were unthinkable two decades ago. Progress in molecular biology and, especially, molecular medicine is now changing the basics of clinical medicine. Reports of long-term side effects of the first successful human gene therapy study have slowed the penetration of DNA usage into clinical routine. However, the main safety problem lies in the secure and efficient delivery of genes into target cells and tissues. A number of older and more recently discovered techniques have been developed for therapeutic gene transfer. A variety of viral and non-viral possibilities are available for basic and clinical research. This review summarizes the delivery routes and methods for gene transfer used in gene therapy. The best known are viral vectors derived from adenoviruses and adeno-associated viruses. Specific properties of lentiviruses and retroviruses ensure their status in current gene therapy procedures. Recent advances in the preparation of lipoplexes and greater safety support the use of naked DNA transfer. The latest approach, using bacteria as vectors for gene therapy or as in situ producers of therapeutic proteins (alternative gene therapy), must undergo critical evaluation in further experiments.
Keywords: Viruses - classification, Viruses - genetics, Electroporation, Gene Expression Regulation, Gene Transfer Techniques, Genetic Therapy - methods, Genetic Vectors, Oligodeoxyribonucleotides, Viruses - genetics
Editorial
01 May 2024 : Editorial
Editorial: First Regulatory Approval for Adoptive Cell Therapy with Autologous Tumor-Infiltrating Lymphocytes (TILs) – Lifileucel (Amtagvi)DOI: 10.12659/MSM.944927
Med Sci Monit 2024; 30:e944927
In Press
12 Mar 2024 : Clinical Research
Comparing Neuromuscular Blockade Measurement Between Upper Arm (TOF Cuff®) and Eyelid (TOF Scan®) Using Miv...Med Sci Monit In Press; DOI: 10.12659/MSM.943630
11 Mar 2024 : Clinical Research
Enhancement of Frozen-Thawed Human Sperm Quality with Zinc as a Cryoprotective AdditiveMed Sci Monit In Press; DOI: 10.12659/MSM.942946
12 Mar 2024 : Database Analysis
Risk Factors of Age-Related Macular Degeneration in a Population-Based Study: Results from SHIP-TREND-1 (St...Med Sci Monit In Press; DOI: 10.12659/MSM.943140
12 Mar 2024 : Clinical Research
Preoperative Blood Transfusion Requirements for Hemorrhoidal Severe Anemia: A Retrospective Study of 128 Pa...Med Sci Monit In Press; DOI: 10.12659/MSM.943126
Most Viewed Current Articles
17 Jan 2024 : Review article
Vaccination Guidelines for Pregnant Women: Addressing COVID-19 and the Omicron VariantDOI :10.12659/MSM.942799
Med Sci Monit 2024; 30:e942799
14 Dec 2022 : Clinical Research
Prevalence and Variability of Allergen-Specific Immunoglobulin E in Patients with Elevated Tryptase LevelsDOI :10.12659/MSM.937990
Med Sci Monit 2022; 28:e937990
16 May 2023 : Clinical Research
Electrophysiological Testing for an Auditory Processing Disorder and Reading Performance in 54 School Stude...DOI :10.12659/MSM.940387
Med Sci Monit 2023; 29:e940387
01 Jan 2022 : Editorial
Editorial: Current Status of Oral Antiviral Drug Treatments for SARS-CoV-2 Infection in Non-Hospitalized Pa...DOI :10.12659/MSM.935952
Med Sci Monit 2022; 28:e935952