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Recombinant DNA Technology and Gene Therapy Using Viruses
Published in Patricia G. Melloy, Viruses and Society, 2023
After the development of recombinant DNA technology, scientists began investigating how to use cloned genes to treat disease. Gene therapy involves using some type of gene transfer method, usually a virus, to deliver a gene for expression in a person’s cells to treat a disease (Colavito 2007; Kurreck and Stein 2016). The therapy may be designed so that the new gene substitutes for a defective gene (a type of gene editing), or that the new gene inactivates another gene. In addition, gene therapy can work by adding another gene to the patient’s genome to treat a disease, rather than replacing or inactivating a gene (FDA 2022b; Anguela and High 2019). Gene therapy was first explored as a type of disease treatment for individuals with a monogenic disease, meaning that the disease or disorder was caused by a change in a single gene (Colavito 2007).
Genetic Limitations to Athletic Performance
Published in Peter M. Tiidus, Rebecca E. K. MacPherson, Paul J. LeBlanc, Andrea R. Josse, The Routledge Handbook on Biochemistry of Exercise, 2020
So far, we have concentrated on how natural genetic variation contributes to sporting performance. However, the knowledge gained from studying the genetics of sporting performance could ultimately be used to enhance an individual's ability to perform. Whilst “gene doping” is not currently believed to be possible, in 2003 it was added to the World Antidoping Agency (WADA) prohibited list (80). Gene doping is defined as “the non-therapeutic use of genes, genetic elements and/or cells that have the capacity to enhance athletic performance.” More simply put, gene doping is gene therapy in people who have no medical need for it. Gene therapy is a medical technique that involves either transferring modified DNA into an individual or modifying the DNA of an individual to treat a medical condition. In its simplest form, this would be to provide a functional version of a missing or damaged protein.
Clinical Progresses in Regenerative Dentistry and Dental Tissue Engineering
Published in Vincenzo Guarino, Marco Antonio Alvarez-Pérez, Current Advances in Oral and Craniofacial Tissue Engineering, 2020
Gene therapy is a term that has appeared with increasing frequency in both the popular and the scientific literature. It is commonly used in reference to any clinical application of the transfer of a foreign gene Human gene therapy is defined as the treatment of disorder or disease through the transfer of engineered genetic material into human cells, often by viral transduction (Scheller and Krebsbach 2009).
Virus-like particle-based nanocarriers as an emerging platform for drug delivery
Published in Journal of Drug Targeting, 2023
Bingchuan Yuan, Yang Liu, Meilin Lv, Yilei Sui, Shenghua Hou, Tinghui Yang, Zakia Belhadj, Yulong Zhou, Naidan Chang, Yachao Ren, Changhao Sun
Gene therapy has a wide range of applications, such as gene replacement, genetic defect correction, gene knockdown and gene augmentation, which have many therapeutic applications for different diseases. VLPs are excellent nucleic acid carriers, as it is common for viral structural proteins to bind to nucleic acids. Nucleic acids can be encapsulated into VLPs mainly by noncovalent methods utilising the inherent affinities of VLPs. A viral capsid protein can load a gene onto the inner surface of VLPs by adjusting the pH, osmotic shock or direct interaction. Many negatively charged substrates are also amenable to be loaded on the inner surface of the viral capsid, as the inner surface is generally positively charged. As shown in Table 2, CCMV [91], HBV [27,72–75], JC [83,84] and P22 [23] VLPs could load DNA or RNA by electrostatic interactions between the nucleic acid and the viral coat proteins. These nucleic acid-loaded VLPs have been explored for various uses, such as vaccines and delivery systems. DNA and RNA are easy to load and retain in VLPs, as they are similar to viral genomes.
Comprehensive analysis and prediction of long-term durability of factor IX activity following etranacogene dezaparvovec gene therapy in the treatment of hemophilia B
Published in Current Medical Research and Opinion, 2023
Jinesh Shah, Hongseok Kim, Krupa Sivamurthy, Paul E. Monahan, Michael Fries
Gene therapy provides a possible cure for people who suffer from diseases associated with faulty or missing genes. Ideally, those gene therapies target the root causes of the disease with a single (or limited) administration of treatment dose. For chronic diseases, gene therapy may be able to replace a lifetime of expensive maintenance therapies, which may lead to decreased treatment burden and to cost savings in the longer term. Yet, the uncertainty around the long-term durability of gene therapy is of concern to patients, treating physicians, regulators and payers. To address these concerns specifically related to etranacogene dezaparvovec, we analyzed 55 participants from the Phase 2 b and Phase 3 studies who responded to treatment, to predict the long-term durability of the selected outcome – etranacogene dezaparvovec produced factor IX activity levels as measured by central laboratory one-stage aPTT assay. More specifically, Bayesian and Frequentist linear mixed models were deployed to predict the factor IX activity level up to 25.5 years post-etranacogene dezaparvovec infusion at both the individual and population level.
Onasemnogene abeparvovec for the treatment of spinal muscular atrophy
Published in Expert Opinion on Biological Therapy, 2022
Hugh J. McMillan, Crystal M. Proud, Michelle A. Farrar, Ian E. Alexander, Francesco Muntoni, Laurent Servais
The therapeutic landscape for SMA has changed dramatically over the past few years. Although most cases were essentially untreatable and fatal within the first years of life, disease-modifying treatments, including onasemnogene abeparvovec, are improving survival and permitting many patients to thrive. Prognoses for most patients with SMA are greatly improved, and the importance of ongoing multidisciplinary care remains undiminished. While onasemnogene abeparvovec for SMA represents a significant milestone in human gene therapy, this field is still in its infancy, and challenges and uncertainties, such as patient and disease selection, need to be clarified. The safety and efficacy of gene therapy may be affected by many factors, including patient age, weight, and disease severity, as well as delivery mechanisms and targets of the gene therapy vector.