Abstracts for 6th Central European Symposium on Pharmaceutical Technology and Biotechnology (original) (raw)
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Targeted delivery of DNA for gene therapy via receptors
Trends in Biotechnology, 1993
The general concept of targeted delivery of therapeutic agents was first recognized by Paul Ehrlich at the turn of the century I. Only recently has this strategy been applied to the delivery of DNA-protein complexes to cells 2. Most of the research on DNA delivery by this approach has been performed by targeting the liver-specific asialoglycoprotein receptor (ASGPr) (Refs 3-10) and the relatively ubiquitous transferrin receptor,1 19. DNA delivery to hepatocytes has also been effected using insulin-polylysine conjugates 2°, and to antigen-bearing cells using an antibody 21. The majority of these experiments use a covalently crosslinked receptor ligand-polycation conjugate to bind DNA in an electrostatic complex 5 . Receptormediated endocytosis of the ligand carries the bound DNA into a cell, allowing subsequent expression of the foreign DNA ). This technique, which has been demonstrated in animals using the ASGPr (Refs 5-8), is promising for the delivery of therapeutic DNAs as well as antisense oligonucleotides2L
Delivery systems for gene therapy
The structure of DNA was unraveled by Watson and Crick in 1953, and two decades later Arber, Nathans and Smith discovered DNA restriction enzymes, which led to the rapid growth in the field of recombinant DNA technology. From expressing cloned genes in bacteria to expressing foreign DNA in transgenic animals, DNA is now slated to be used as a therapeutic agent to replace defective genes in patients suffering from genetic disorders or to kill tumor cells in cancer patients. 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. 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.
ChemInform Abstract: Gene Delivery System: A Developing Arena of Study for the New Era of Medicine
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Gene therapy concept has been being overcome massive challenges from 1972 in ethical, socio-economical and developmental issues. In this review, we have attempted to go through almost all the arenas and described in a methodical way that reflects not only the initial ethical and scientific thoughts but also adorned a solid depiction of gene therapy related physico-chemical barriers, approaches and strategies till to date.
Gene delivery system: a developing arena of study for the new era of medicine
Recent patents on DNA & gene sequences, 2012
Gene therapy concept has been being overcome massive challenges from 1972 in ethical, socio-economical and developmental issues. In this review, we have attempted to go through almost all the arenas and described in a methodical way that reflects not only the initial ethical and scientific thoughts but also adorned a solid depiction of gene therapy related physico-chemical barriers, approaches and strategies till to date.
DNA as therapeutics; an update
Indian Journal of Pharmaceutical Sciences, 2009
Saraswat, et al.: DNA as Therapeutics Human gene therapy is the introduction of new genetic material into the cells of an individual with the intention of producing a therapeutic benefi t for the patient. Deoxyribonucleic acid and ribonucleic acid are used in gene therapy. Over time and with proper oversight, human gene therapy might become an effective weapon in modern medicine's arsenal to help fi ght diseases such as cancer, acquired immunodefi ciency syndrome, diabetes, high blood pressure, coronary heart disease, peripheral vascular disease, neurodegenerative diseases, cystic fi brosis, hemophilia and other genetic disorders. Gene therapy trials in humans are of two types, somatic and germ line gene therapy. There are many ethical, social, and commercial issues raised by the prospects of treating patients whose consent is impossible to obtain. This review summarizes deoxyribonucleic acid-based therapeutics and gene transfer technologies for the diseases that are known to be genetic in origin. Deoxyribonucleic acid-based therapeutics includes plasmids, oligonucleotides for antisense and antigene applications, deoxyribonucleic acid aptamers and deoxyribonucleic acidzymes. This review also includes current status of gene therapy and recent developments in gene therapy research.
DNA nanoparticles for gene delivery to cells and tissue
International Journal of Nanotechnology, 2006
The development over the past decade of methods for delivering genes to mammalian cells has stimulated great interest in the possibility of treating human disease by gene-based therapies. The major aim of gene therapy is to effectively deliver the genetic materials into cells, genetically modifying and repairing cell functions, which may induce therapeutic healing of disease conditions. This review provides a critical view of gene therapy with a major focus on advanced DNA nanoparticles technologies to control the in vivo location and function of administered genes.
Recent trends in non‐viral vector‐mediated gene delivery
Biotechnology journal, 2009
Nucleic acids-based next generation biopharmaceuticals (i.e., pDNA, oligonucleotides, short interfering RNA) are potential pioneering materials to cope with various incurable diseases. However, several biological barriers present a challenge for efficient gene delivery. On the other hand, developments in nanotechnology now offer numerous non-viral vectors that have been fabricated and found capable of transmitting the biopharmaceuticals into the cell and even into specific subcellular compartments like mitochondria. This overview illustrates cellular barriers and current status of non-viral gene vectors, i.e., lipoplexes, liposomes, polyplexes, and nanoparticles, to relocate therapeutic DNA-based nanomedicine into the target cell. Despite the awesome impact of physical methods (i.e., ultrasound, electroporation), chemical methods have been shown to accomplish high-level and safe transgene expression. Further comprehension of barriers and the mechanism of cellular uptake will facilitate development of nucleic acids-based nanotherapy for alleviation of various disorders.
The twentieth century is considered for the technological revolution in different fields such as Industry, Research and Medicine. Scientific inventions have improved Research and Industrial output while medicine one step ahead. Numerous inventions have made a revolution in the management of life threatening diseases which were incurable decades ago. Due to these revelations, health care system is growing exponentially and especially, Gene Therapy is known as one of the most advanced approaches for the treatment of diseases associated with abnormal functioning of the genome. Gene therapy offers management of diseases/disorders through manipulation at genetic level either by replacement of abnormal gene(s) and /or repairs. Gene therapy essentially requires targeted and efficient gene delivery to tissue or cell. There are numerous methods available to carry out gene delivery either In-Vivo or Ex-Vivo for particular diseases. Both viral and nonviral vehicles used for delivery of exogenous genes have shown tremendous benefits in numerous clinical trials carried out over last few decades. Both the options, viral and non-viral tools, for gene delivery with remarkable significance are often linked with numerous complications. To surpass these complications, novel tools like Nano-constructs designed for nanomaterials are in practice and have shown promising results in delivering candidate drugs and biomolecules. Here in this review, we have summarized the potential of new generation delivery vehicles and their advantages over viral and other available non-viral vehicles. Also, current information with respect to the design and functioning of Nano-constructs implemented in clinical study for management of many diseases is provided.