Chemistry
Gene Therapy Process
Gene therapy is a process that involves introducing genetic material into a patient's cells to treat or prevent disease. This can be achieved by using viral vectors or other delivery methods to transfer the therapeutic genes into the target cells. The goal is to correct genetic mutations or provide new functions to the cells, offering potential treatments for various genetic disorders.
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6 Key excerpts on "Gene Therapy Process"
eBook - PDF- Joseph D. Bronzino, Donald R. Peterson(Authors)
- 2018(Publication Date)
- CRC Press(Publisher)
56 -1 56.1 Introduction The area of gene therapy is considered to have its roots in the early 1960s with the birth of genetic trans-formation of eukaryotic cells in vitro (Friedmann, 1992), although it could be argued that it was the trans-formation of pneumococcal cells in the 1940s that really inspired the concept (Avery et al., 1944). Another critical leap was made in the early 1980s with the work of Spradling and Rubin in Drosophilia, where exogeneous DNA sequences were introduced into germ line cells in order to correct a genetic defect (Rubin and Spradling, 1982, Spradling and Rubin, 1982). In the subsequent years, gene therapy has been proposed for a variety of genetic diseases (Friedmann, 1989) as well as other, more organ-specific pathologies. Gene therapy broadly encompasses any technique used to regulate eukaryotic protein expres-sion by manipulation of the genetic machinery. This includes everything from delivery of DNA sequences to miRNA interference of mRNA translation to delivery of cells with altered genomes. This can take the form of permanently inserting a gene into a nonspecific location on a chromosome in order to replace a nonfunctional gene, regulating a specific gene, temporarily placing a gene in the nucleus to be expressed for a short period, replacing an original, impaired gene or gene promoter with a functioning sequence using homologous recombination, or repairing an impaired gene using selective reverse mutation to return a gene to normal function. Unlike drug delivery, a cell transfected by gene delivery to produce a specific protein can continuously release the bioactive chemical. Epigenetic promoters can even be used so that expression will only occur under certain conditions. Gene vectors can be tailored to preferentially transfect specific cells. In contrast, local drug injections expose the drug to the surrounding area, possibly causing unnecessary side effects.
eBook - PDFTissue Engineering
Principles and Practices
- John P. Fisher, Antonios G. Mikos, Joseph D. Bronzino, Donald R. Peterson, John P. Fisher, Antonios G. Mikos, Joseph D. Bronzino, Donald R. Peterson(Authors)
- 2012(Publication Date)
- CRC Press(Publisher)
18 -1 18.1 Introduction The area of gene therapy is considered to have its roots in the early 1960s with the birth of genetic trans-formation of eukaryotic cells in vitro (Friedmann, 1992), although it could be argued that it was the trans-formation of pneumococcal cells in the 1940s that really inspired the concept (Avery et al., 1944). Another critical leap was made in the early 1980s with the work of Spradling and Rubin in Drosophilia, where exogeneous DNA sequences were introduced into germ line cells in order to correct a genetic defect (Rubin and Spradling, 1982, Spradling and Rubin, 1982). In the subsequent years, gene therapy has been proposed for a variety of genetic diseases (Friedmann, 1989) as well as other, more organ-specific pathologies. Gene therapy broadly encompasses any technique used to regulate eukaryotic protein expres-sion by manipulation of the genetic machinery. This includes everything from delivery of DNA sequences to miRNA interference of mRNA translation to delivery of cells with altered genomes. This can take the form of permanently inserting a gene into a nonspecific location on a chromosome in order to replace a nonfunctional gene, regulating a specific gene, temporarily placing a gene in the nucleus to be expressed for a short period, replacing an original, impaired gene or gene promoter with a functioning sequence using homologous recombination, or repairing an impaired gene using selective reverse mutation to return a gene to normal function. Unlike drug delivery, a cell transfected by gene delivery to produce a specific protein can continuously release the bioactive chemical. Epigenetic promoters can even be used so that expression will only occur under certain conditions. Gene vectors can be tailored to preferentially transfect specific cells. In contrast, local drug injections expose the drug to the surrounding area, possibly causing unnecessary side effects.
eBook - PDF- Danilo D. Lasic(Author)
- 2019(Publication Date)
- CRC Press(Publisher)
GENE THERAPY Medicine has passed in its history through several breakthroughs, from Galen and Pasteur to the introduction of anesthesia, surgery, vaccines, antibiotics, novel imaging techniques, lasers, and remote operating devices. Direct treatment on the molecular level of diseases themselves and not their symptoms may be the next important development in human therapy. Gene therapy is emerging as a new modality as well as a technology in medical practice. It offers the potential to cure disease on its most basic level and has therefore captured the imagination of scientific and popular media. The therapy requires the insertion of a functional gene or other molecule with an information sequence into a cell to achieve a therapeutic effect, and the gene therefore serves as a drug (Anderson, 1992; Miller, 1992; Mulligan, 1993; Goldspiel et al., 1993). All the organisms of a particular species have an identical number and type of genes. Obviously, because we are not absolutely identical, small variations among genes exist within the species. While most of these polymorphisms have no effect on the protein function and some of them bring only innocuous variations in physical appearance, such as the color of eyes or hair, some of them produce serious inherited disorders. These disorders can be apparent immediately after birth or can develop only later in the life span; they can also make some individuals more susceptible to environmental factors. It is anticipated that gene therapy can handle or eliminate such problems. ■ ■ STRATEGIES IN GENE DELIVERY Several thousand diseases can be traced to defective or missing functional genes, and it is hoped that by delivering the appropriate gene into the appropriate cells, the mutated or missing proteins can be synthesized and the signs of the disease alleviated. In the majority of cases the mutation is in the coding region of the gene.
eBook - PDFAdvanced Textbook On Gene Transfer, Gene Therapy And Genetic Pharmacology: Principles, Delivery And Pharmacological And Biomedical Applications Of Nucleotide-based Therapies
Principles, Delivery and Pharmacological and Biomedical Applications of Nucleotide-Based Therapies
- Daniel Scherman(Author)
- 2013(Publication Date)
- ICP(Publisher)
8 Advanced Textbook on Gene Transfer, Gene Therapy and Genetic Pharmacology effect, or which encodes a missing protein or any protein or peptide allowing a therapeutic or vaccination effect. The distinction between genetic pharmacology and gene therapy (Fig. 2.1) will be preferentially used in the present textbook. However, other authors employ the generic expression “gene therapy” to designate all approaches implying the use of any natural or modified DNA or RNA nucleotide molecule. Gene therapy might be used, in a non-exhaustive list: • to compensate for a missing protein in a genetic disease, • to inhibit the production of a given protein (by generating an antisense mRNA), for instance to render cells resistant to viral infection, • to express a trophic factor or an anti-inflammatory cytokine, • to introduce a suicide gene for the treatment of cancer, Examples of potential applications of gene therapy are just too many to be listed here. Several genetic diseases have attracted large interest for application of a gene-replacement strategy (Table 2.1). Small-drug molecules generally target proteins Gene Therapy delivers a gene flanked by a eukaryotic promoter and a transcription termination signal Genetic Pharmacology : Small oligonucleotides target either DNA or RNA Genomic DNA mRNA Protein Function FIGURE 2.1 While chemical drugs classically target proteins (except in the case of anticancer cytotoxic compounds), small-chemical oligonucleotides used in genetic pharmacology generally target DNA and/or RNA, with the exception of aptamers (Chapter 4). Gene therapy consists of administering a gene-expressing cassette (promoter — gene — polyadenylation signal) to the cells.
eBook - PDF- Ming Wei, David Good, Ming Wei, David Good(Authors)
- 2013(Publication Date)
- IntechOpen(Publisher)
Section 3 Gene Therapy for Cancer Chapter 10 Challenges in Advancing the Field of Cancer Gene Therapy: An Overview of the Multi-Functional Nanocarriers Azam Bolhassani and Tayebeh Saleh Additional information is available at the end of the chapter http://dx.doi.org/10.5772/54862 1. Introduction Recent developments in molecular biology and cell biology have led to the discovery of novel genes and proteins having therapeutic potentials for various diseases including cancers. Based on these findings, novel categories of therapeutic biomacromolecules in‐ cluding genes, small interfering RNA (siRNAs), antisense oligonucleic acids, bioactive proteins and peptides have been developed. These macromolecules can be more advanta‐ geous than small-molecular-weight therapeutic agents in terms of their specificity and high potency to the target molecules [Nakase et al., 2010]. Gene therapy is the newest therapeutic strategy for treating human diseases. The basic idea of gene therapy is a gene or gene product that can be selectively delivered to a specific cell/tissue with mini‐ mal toxicity. This product can inhibit the expression of a specific defective gene or ex‐ press a normal gene. Efficient and safe delivery is one of the key issues for the clinical application of nucleic acids as therapeutic agents [Du et al., 2010]. The goal of the Phar‐ maceutical Industry is to have a gene therapy medical product that can be delivered sys‐ temically. In vivo gene therapies have focused on viral vectors for gene delivery and have had marginal clinical successes. Major disadvantage of these delivery systems is the integration of some viral vectors into human chromosomes of normal tissue.
eBook - PDF- Yongping You(Author)
- 2011(Publication Date)
- IntechOpen(Publisher)
Part 1 Target Strategy in Gene Therapy 1 Choosing Targets for Gene Therapy Karina J. Matissek, Ruben R. Bender, James R. Davis and Carol S. Lim University of Utah USA 1. Introduction Gene therapy is often attempted in fatal diseases with no known cure, or after standard therapies have failed. Targeting gene defects includes addressing a single mutation, multiple mutations in several genes, or even addressing missing or extra copies in a particular disease. A defect in one specific gene may impair normal function of the corresponding expressed protein. For example, in X-linked severe combined immunodeficiency (X-SCID), there is a mutation in the IL2 receptor γ gene. Another classic example occurs in thalassemia propagated by a defect in the β -globulin gene. Some diseases are caused by multiple mutations in several genes. For example, some cardiovascular diseases may manifest due to mutations in different chromosomes which are a result of inherited or environmental factors. Before approaching a disease using gene therapy, the key protein(s) and pathways involved in the disease should first be identified. However, in some cases an abnormal gene is formed that results in disease; such is the case for the Bcr-Abl gene. The oncogenic Bcr-Abl protein is the causative agent of chronic myelogenous leukemia (CML) which could be blocked for CML treatment. Genomic sequencing information, microarrays, and biochemical assays can be used to determine up- or down-regulated proteins involved in disease, and will help determine the function of these proteins. In the case of some cancers, the signal transduction pathways for oncogenesis have been mapped out, allowing hub proteins to be identified. Hub proteins are essential proteins that interact with multiple other proteins in signaling cascades. If selected properly, adding back a tumor-suppressing hub protein (such as p53), or blocking an oncogenic hub protein (such as survivin) could halt cancer or alter disease progression.
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