Somatic Genome Manipulation: Advances, Methods, and Applications

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On the other hand, American research groups remained conservative, reiterating their position of not supporting this type of experiment and declaring that they await improvement in the techniques and of the definitions of ethical issues. Since the declaration of James Watson in , in reference to the likely optimization of human genetics, gene therapy has advanced throughout the decades, whether by optimization of the types of vectors, by the introduction of new techniques, such as induced pluripotent stem cells in combination with current models of genetic editing CRISPR-Cas9 , and even by trials in germ cells, bringing with it the contradictory ethical and moral aspects that accompany the technique.

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Local successes have already solidified the viability of treatments using gene therapy in clinical practice, as an alternative form for patients with congenital diseases or monogenic disorders and cancer, especially when the pharmacological or surgical interventions do not show good results. The design of new experimental vectors, the increase in efficiency, the specificity of the delivery systems, and the greater understanding of the inflammatory response induction may balance the improvement of safety with the expansion of techniques in clinical applications.

Yet the knowledge and experience acquired with the careful assessment of toxicity of these technologies also allow significant advances in the application of these methods. Therefore, historically, gene therapy and the discovery of antibiotics and chemotherapy agents, or any new technology, need more clarifying preclinical studies.

In the future, there is the promise of applying these techniques in several fields of Medicine and a greater percentage of clinical trials. National Center for Biotechnology Information , U. Journal List Einstein Sao Paulo v. Einstein Sao Paulo.

Find articles by Raquel de Melo Alves Paiva. Author information Article notes Copyright and License information Disclaimer. E-mail: rb.


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  2. The Mahler Family Letters;
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  4. Gene therapy: advances, challenges and perspectives.

Received Feb 14; Accepted Jun Copyright notice. This is an Open Access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

This article has been cited by other articles in PMC. ABSTRACT The ability to make site-specific modifications to the human genome has been an objective in medicine since the recognition of the gene as the basic unit of heredity. Gene therapy The ability to make local modificiations in the human genome has been the objective of Medicine since the knowledge of DNA as the basic unit of heredity. Open in a separate window. Gene therapy process: release of the gene In gene therapy, a normal gene is inserted into the genome to replace an abnormal gene responsible for causing a certain disease.

Gene therapy and hematopoietic stem cells Hematopoietic stem cells have become ideal targets for gene transfer due to the high potential for longevity and the capacity for self-renovation. Figure 1. Source: Adapted from Kay MA. State-of-the-art gene-based therapies: the road ahead.

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Figure 2. In: Pereira TC, organizador. Ethical issues The possibility of genetically modifying germlines has been the object of heated discussion in the field of science for a long time. COMMENT Since the declaration of James Watson in , in reference to the likely optimization of human genetics, gene therapy has advanced throughout the decades, whether by optimization of the types of vectors, by the introduction of new techniques, such as induced pluripotent stem cells in combination with current models of genetic editing CRISPR-Cas9 , and even by trials in germ cells, bringing with it the contradictory ethical and moral aspects that accompany the technique.

Mukherjee S. The gene: an intimate history.


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    Somatic Genome Manipulation

    The advantage of somatic genome manipulation is maintenance of the general genotype while correcting one or more traits. Somatic genome manipulation is also an option for genetic improvement of sexually propagated plants in polyploidy breeding or in overcoming issues of sexual incompatibility. Recent novel technologies in somatic genome manipulation are developing quickly but much of this literature is fragmented and difficult or inconvenient to access.

    This book represents the first attempt to assemble updated reviews, detailed protocols, and their applications in all fields in which somatic genome manipulation has thrived.