The Aims of Operative Surgery
Hutan Ashrafian in Surgical Philosophy, 2015
Sometimes a diseased body can be tricked into accepting an inherent cure. Gene therapy works on this principle whereby a gene or deoxyribonucleic acid (DNA) is the therapeutic agent used in combating disease. Two forms exist: (i) somatic gene therapy in which a therapeutic piece of DNA will be expressed in an individual who has a disease resulting from lack of or a defective segment of inherent DNA (this treatment will only last in the individual receiving the extraneous gene therapy), and (ii) germ-line gene therapy, in which DNA can be placed in germ lines, so that any defective or lacking genes will be negated by extraneous gene therapy for all subsequent replicative generations. Several vectors can be used in this genetic ‘war on disease’ through the application of both viral and nonviral vectors.
The new genetics and health promotion
Robin Bunton, Gordon Macdonald in Health Promotion, 2003
Gene therapy involves the manipulation of somatic cells, i.e. those not involved in reproduction (germ line gene therapy is not allowed currently and involves manipulation of the germ line, thus affecting future generations). Usually some kind of vector, such as a virus, is used to carry a new copy of a gene into affected cells in order to alter the course of disease. So far, trials have had limited or no success, and it seems unlikely that gene therapy will provide a rapid solution to the problem of human disease. However, wider developments in pharmaceuticals, resulting from developments in genetics, pharmacogenomics, are likely to be more rapid. Drug therapy is likely to become more tailored to individuals, taking into account their specific responses to medication, which their specific genotype will indicate (Zimmern and Cook 2000).
Genetic engineering: Past and present as prelude to the future
Mark J. Cherry, Ana Smith Iltis, Roberta M. Berry in The Ethics of Genetic Engineering, 2013
In-principle objections to germ-line genetic engineering were increasingly assailed beginning in the 1980s. Commentators urged that germ-line genetic engineering aimed at a therapeutic purpose should be assessed according to its risks and benefits, as with gene therapy and other medical interventions. They also urged the importance of the ethical and legal principle of autonomy and free parental choice.135 Commentators argued that holding the line certainly did not seem worth it if it meant that our descendents would suffer disease or disability that we could have prevented, especially if abortion or forgone pregnancies were the only other “preventive” options in some cases.136 And if genetic engineering did introduce undesirable consequences, it was argued, we could always re-engineer future generations to eliminate these problems.137
The current status of gene therapy in bladder cancer
Published in Expert Review of Anticancer Therapy, 2023
Côme Tholomier, Alberto Martini, Sharada Mokkapati, Colin P. Dinney
Adeno- and adeno-associated-viral (AAVs) vectors are promising vectors for gene therapy as they provide relatively long-term transgene expression with minimal risks of unwanted integration into the host’s genome, reducing the risk of insertional mutagenesis [18,19]. Although adenoviruses are usually considered immunogenic, multiple reports with long-term follow-up have confirmed an adequate safety profile [20,21]. Concerns that patients might have natural antibodies to adenovirus from previous flu exposures that would blunt efficacy have not been borne out by clinical experience. Finally, there is concern for the possibility that gene therapy could induce germ-line alterations which could lead to the unwanted transfer genes to the offspring. Current trials are designed to minimize this risk [22].
Gene therapy for inherited retinal diseases: progress and possibilities
Published in Clinical and Experimental Optometry, 2021
Monica L Hu, Thomas L Edwards, Fleur O’Hare, Doron G Hickey, Jiang-Hui Wang, Zhengyang Liu, Lauren N Ayton
Gene therapy refers to the use of genetic material (DNA or RNA) to modify gene expression, in order to treat disease. Since its conceptualisation several decades ago, diverse techniques have been developed. These aim to accomplish the major therapeutic principles of facilitating restoration of gene function in the case where a mutation has inactivated it, or inactivating a mutated gene with aberrant function, such as a dominant negative effect as seen in autosomal dominant RP (adRP) caused by mutations in the rhodopsin (RHO) gene. The genetic material can be delivered to the target cells by either direct administration to the target tissue, for instance the retina (an in vivo approach), or to cells extracted from a patient for subsequent reintroduction to the body (an ex vivo approach).11 The genetic payload can be given in the form of naked genetic material,12 or can be packaged into a specialised vector (vehicle used to deliver the transgene) to be delivered into target cells.
Leading edge: emerging drug, cell, and gene therapies for junctional epidermolysis bullosa
Published in Expert Opinion on Biological Therapy, 2020
Allison R. Keith, Kirk Twaroski, Christen L. Ebens, Jakub Tolar
Gene therapy is the process of correcting genetic abnormalities to treat or prevent disease. In the case of JEB, this can range from the correction of specific mutations to the total replacement of nonfunctional alleles. Loss-of-function mutations leading to generalized severe JEB are most commonly found within the β3 subunit of heterotrimer LM332. These are associated with some of the most severe clinical manifestations of the disease [90]. Much of the JEB-specific gene-editing research to date has focused on the restoration of β3-subunit (LAMB3 gene) function. As such, corrective therapies have been limited to ex vivo genetic manipulation and subsequent autologous transplant [10–12]. A variety of viral and nonviral methods have been employed to aid in the correction of JEB-causing gene mutations.
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