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The Genetic Body
Published in Roger Cooter, John Pickstone, Medicine in the Twentieth Century, 2020
After screening comes genetic manipulation. Some concerns are related to current research on somatic gene therapy. This work aims to remedy genetic dysfunction in the non-reproductive cells. More contentious is the possibility of germ-line manipulation, altering the genome of the reproductive cells in order to change future generations. This is plainly a technology for eugenics.
Epidermal Keratins
Published in John P. Sundberg, Handbook of Mouse Mutations with Skin and Hair Abnormalities, 2020
Joseph A. Rothnagel, Dennis R. Roop
To date, transgenic studies have shown that reverse genetic experiments can be powerful tools to study keratin function and have prompted investigations that have led to the discovery of the molecular basis for two dominantly inherited skin diseases, EBS and EHK. We have also exploited the expression characteristics of various epidermal genes to target exogenous gene expression exclusively to the epidermis and hair follicle. In this way, we have observed the effects of overexpression of growth factors, such as TGFα44 and TGFβ,45 and protooncogenes, such as v-ras46 and v-fos.47 These mice exhibit profound epidermal changes and have provided us with the means to identify the contribution of each of these proteins to normal skin function and to the disease state. Moreover, these mice will allow us to assess the viability of somatic gene therapy approaches for the amelioration of human skin diseases. Somatic gene therapy is expected to provide new therapies for many diseases that have been recalcitrant to conventional pharmacological or surgical therapy,48 and given its accessibility and ease of handling, the skin will be an important site of entry for these new technologies.
Challenges Facing the American Healthcare System
Published in Kant Patel, Mark Rushefsky, Healthcare Politics and Policy in America, 2019
There are two basic types of gene therapy. One is somatic gene therapy, where therapeutic genes are transferred into somatic cells of a patient. Here, the modification of the gene and its effects are restricted to the individual patient and will not be inherited by the patient’s offspring or later generations. The second type of gene therapy is germline gene therapy, in which germ cells (sperm or eggs) are modified by introducing a functional gene that is integrated into the patent’s genome. Under this method, changes made are heritable and would be passed on to later generations (Singh et al. 2016).
Perceptions of airway gene therapy for cystic fibrosis
Published in Expert Opinion on Biological Therapy, 2023
Martin Donnelley, David Parsons, Ivanka Prichard
To date, only two studies have specifically examined perceptions of gene therapy for CF. In 2003 Iredale et al. performed a qualitative pilot study to examine the attitudes of people with CF and members of the public [16]. They found support for somatic gene therapy, and hesitancy about germline gene therapy, as well as opposition to gene therapy for enhancement purposes. In 2006 Jaffe et al. performed a short seven question single-center quantitative study that examined parental attitudes toward gene therapy for children with CF in the UK [15]. They found that parents of children with CF reported that gene therapy was an important part of CF research, however safety was still considered a major issue, especially when considering gene therapy for children. While important, these two studies only provide information on the opinions of small populations (N = 22 and N = 80, respectively) from 15+ years ago, well before gene therapies had experienced success across a range of other diseases.
Gene and cell therapy and nanomedicine for the treatment of multiple sclerosis: bibliometric analysis and systematic review of clinical outcomes
Published in Expert Review of Neurotherapeutics, 2021
Javier Caballero-Villarraso, Jamil Sawas, Begoña M. Escribano, Francisco A. Martín-Hersog, Andrea Valverde-Martínez, Isaac Túnez
Gene therapy could be defined as the set of techniques that allow the conveyance of DNA or RNA sequences inside target cells, in order to modulate the expression of certain genes that are altered, thus reversing the biological disorder previously induced [13,14]. Depending on the type of target cell, there are two modalities of gene therapy: germ cell gene therapy and somatic gene therapy. Furthermore, depending on the applied strategy, it can also be classified into in vivo and ex vivo gene therapy. To achieve a certain biological effect in gene therapy, it is necessary to efficiently transfer (introduce) the gene sequence of interest into the target cell and subsequently obtain its expression pattern in the host cell. Physico-chemical gene transfer methods include: microinjection, calcium phosphate precipitation, electroporation, microprojectile bombardment, direct injection of ‘naked’ DNA, DNA-protein conjugates, DNA-adenovirus conjugates, and liposomes [13–15].
Germline Gene Editing for Sickle Cell Disease
Published in The American Journal of Bioethics, 2020
Akshay Sharma, Nickhill Bhakta, Liza-Marie Johnson
However, the main problem with either allogeneic HSCT or somatic gene therapy is that these treatments only help the individual who receives them and not their subsequent generations. Even individuals who are ‘cured’ of their disease can still pass on the affected gene to their children. And if the partner of this individual is a carrier or has sickle cell trait, their children will continue to inherit two copies of the affected genes from their parents 50% of the time, resulting in a clinically significant hemoglobinopathy. If both parents have sickle cell disease, even if they both have undergone an HSCT or gene therapy, all their children will have both copies of the gene affected with the mutation and will develop SCD. For most patients with SCD around the world who cannot even afford basic healthcare, HSCT and somatic gene therapy are untenable from a personal financial basis. But even from a healthcare systems standpoint these interventions may not drastically reduce the overall burden of the disease in the society given that these interventions do not reduce the ongoing rates of heritable transmission that occurs presently. It is doubtful whether any one family will be able to self-finance somatic gene therapy for more than one individual in their household. At the same time, it will become financially unsustainable for public insurance programs over time to repeatedly pay for somatic gene therapy for several members of a family. This will eventually require either a triage system or a distribution mechanism to ‘allot’ gene therapy treatments to patients and families, unless the therapy can provide long term multi-generational eradication of the disease. And this in turn would lead to more inequity and widening of the socio-economic gap in a community that is already ridden with these problems.