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Dendrimers in Gene Delivery
Published in Neelesh Kumar Mehra, Keerti Jain, Dendrimers in Nanomedicine, 2021
Dnyaneshwar Baswar, Ankita Devi, Awanish Mishra
Somatic and germline therapy are types of gene therapy. Somatic gene therapy is considered a much safer and most common therapy, which contains gene vectors, such as viral vectors and non-viral vectors (Nayerossadat et al. 2012). Vectors are basically transporters that may deliver the therapeutic gene to the infected cells. The general concept of gene therapy is inserting the exogenous gene into somatic cells that develop organs to produce a therapeutic effect. Germline gene therapy has the potential to manipulate reproductive sperm cells and eggs to create heritable changes (Misra 2013). Some therapeutic agents contain nucleic acid materials, which upon administration results in cellular adaptation, repairing, insertion or deletion of a gene sequence. Genetic materials such as DNA, mRNA, miRNA, siRNA and antisense oligonucleotides are commonly used in deficient target cells or tissue to re-establish the specific gene function for disease management (Chen et al. 2018). On this basis, gene therapy may appear as a promising pharmacotherapy tool (Klug et al. 2012; Ingusci et al. 2019).
Nanotechnology in Gene Therapy
Published in Paula V. Messina, Luciano A. Benedini, Damián Placente, Tomorrow’s Healthcare by Nano-sized Approaches, 2020
Paula V. Messina, Luciano A. Benedini, Damián Placente
The treatment based on gene therapy can be divided into categories depending on the type of target cells involved: Germline gene therapy (Okita et al. 2007) and somatic line gene therapy (Bank 1996). The election of one or another type depends on the type of the disease and on the kind of strategy used for treating the disease. Germline gene therapy is based on the transfection of genes from a vector to reproductive cells such as zygote or sperm. Thus, the integration of these genes within the guest genome will produce heritable phenotypic characteristics (Stribley et al. 2002). Somatic gene therapy is based on the transference of genes to somatic cells and, therefore, they are devoid of reproductive capacity. This concept means that only an individual cell or group of cells can express the phenotype, and this change only affects one-generation cells. In other words, this new phenotypic characteristic is not heritable.
Genome Editing and Gene Therapies: Complex and Expensive Drugs
Published in Peter Grunwald, Pharmaceutical Biocatalysis, 2020
As outlined in the previous subchapters, genome editing can be used in the following three ways (see also, e.g., Rossant, 2018): Research tool for use in human cells or embryos to explore human disease models, and develop new treatment approaches.Editing of somatic cells either ex vivo or in vivo to treat/ prevent diseases.Genome editing in gametes or embryos, also known as germline editing
Gene Editing: A View Through the Prism of Inherited Metabolic Disorders
Published in The New Bioethics, 2018
Ethically though, is there any difference between selecting embryos such that only those without the condition are born, and editing an affected embryo so that it does not have the disorder? Rehmann-Sutter argues that human germline gene editing does raise more ethical issues, because the manipulation of the human germline will introduce a permanent change that has effects on intergenerational relationships. He presents an analysis of the human germline, defining it as ‘a relationship that links the generations and has its lived reality in families’. He argues that germline gene editing is contentious primarily because the changes it instils are inherited by the next generation. These concerns encompass both issues of safety – the changes we make now may have unforeseen consequences that future generations have not consented to – and questions on the impact on relationships between generations (such as the perceived moral responsibility to ‘produce children with optimal genetic starting conditions’).
Transhumanist Genetic Enhancement: Creation of a ‘New Man’ Through Technological Innovation
Published in The New Bioethics, 2021
Genetic modification of the germline occurs when for example, foreign DNA is introduced into parent gametes or an early embryo; these modifications will be passed to the offspring and consequently to future generations. Interest in editing the genome of germline cells stems from the number of inherited pathological conditions caused by mutations in single genes.