China
Africa
Explore chapters and articles related to this topic
Gene Therapy for Cancer Treatment
Published in Yashwant V. Pathak, Gene Delivery Systems, 2022
Manish P. Patel, Mansi S. Shah, Mansi N. Athalye, Jayvadan K. Patel
The new human genome era is creating an increase in possibilites for genomics-based medicine, which is known as “personalized medicine.” In medicine, gene therapy is defined as a therapeutic strategy that transfers DNA to a patient’s cells to correct a defective/imperfect gene or a gene product in order to treat diseases which are not curable with conventional drugs [Shahryari et al. 2019]. Gene therapies can work by several mechanisms, like replacing a disease-causing gene with a healthy copy of the gene, inactivating a disease-causing gene that is not functioning properly or introducing a new or modified gene into the body to help in treatment of a disease. Studies have shown that gene therapy techniques have broad potential applications in cancer, and almost over 65% of all ongoing clinical gene trials are related to cancerous diseases [DAS et al. 2015].
Immune Reconstitution after Hematopoietic Stem Cell Transplantation
Published in Richard K. Burt, Alberto M. Marmont, Stem Cell Therapy for Autoimmune Disease, 2019
Andreas Thiel, Tobias Alexander, Christian A. Schmidt, Falk Hiepe, Renate Arnold, Andreas Radbruch, Larissa Verda, Richard K. Burt
Two types of immune system, innate and adaptive, co-exist in humans. The evolutionarily more primitive innate immune system consists of cells using germ line genes to express receptors that recognize specific bacterial, viral, or otherwise foreign antigens. These cells include granulocytes, macrophages, and natural killer (NK) cells, as well as proteins such as C3-like complement. There are approximately 33,000 genes in the human genome.1 Therefore, while specific for nonself determinants, the innate immune system has limited diversity. Receptors for cells of adaptive immunity (T and B lymphocytes) arises by somatic recombination of germ line variable (V), joining (J) and diversity (D) genes leading to a highly diverse number (1014 to 1019) of possible T and B cell receptors from a limited number ofV(D)J genes. This provides adaptive immunity with diversity but also allows for generation of self-reactive repertoires.
Ethics in the Era of Precision Medicine
Published in Lawrence S. Chan, William C. Tang, Engineering-Medicine, 2019
From the early days of the Human Genome Project (HGP) in the 1990s, the ethical, legal, and social implications (ELSI) of genetics have been a significant area of consideration accompanying the technical scientific research and its translational application in medicine. Alongside the establishment of the National Center for Human Genome Research, the U.S. Congress mandated through the National Institutes of Health Revitalization Act of 1993 that “‘not less than’ 5% of the NIH Human Genome Project budget be set aside for research on the ethical, legal, and social implications of genomic science” (McEwen et al. 2014). Such ELSI considerations were raised not only from the momentous nature of what was being undertaken in the HGP, but also from residual concerns related to the eugenics sentiments and practices of the first half of the 20th Century. Eugenic beliefs are often most noted for motivating practices of the Nazi Holocaust (including not only the mass extermination of specific people groups, but also euthanizing the elderly and the disabled who were deemed ‘unfit for life’). Eugenic beliefs and practices, however, extended to a variety of other countries including the U.S. and Britain resulting in forced sterilizations and “Fit Family” contests. Such concerns regarding eugenic beliefs and practices were compounded with developments in genetics and reproductive technologies of the 1950s–1970s that led to the possibility of genetic engineering. The mapping of the human genome led to legal questions regarding genetic property rights and gene patents and the use of genetic information by law enforcement (e.g., forensics), employers, insurers, and beyond (Andrews et al. 2015).
Epigenotoxicity: a danger to the future life
Published in Journal of Environmental Science and Health, Part A, 2023
Farzaneh Kefayati, Atoosa Karimi Babaahmadi, Taraneh Mousavi, Mahshid Hodjat, Mohammad Abdollahi
Since completing the Human Genome Project about twenty years ago, there has been a great deal of help from geneticists and physicians regarding having access to the human genome map and better diagnosing diseases. Today, epigenetics has enabled us to understand the complexities of the human biological system and the body’s processes of growth and regulation. Epigenetics indicates that the sequence of nucleotides and their bonds with different functional groups is crucial in DNA function [88] Mistakes in epigenetic mechanisms alter the expression of genes causing a range of disorders. Amongst, developmental and mental disorders, immunodeficiency, Chronic obstructive pulmonary disease (COPD), asthma, and organ defects, are the most common; each will be discussed separately below as well as in Table 2.[88,89]
A pilot study of exome sequencing in a diverse New Zealand cohort with undiagnosed disorders and cancer
Published in Journal of the Royal Society of New Zealand, 2018
Colina McKeown, Samantha Connors, Rachel Stapleton, Tim Morgan, Ian Hayes, Katherine Neas, Joanne Dixon, Kate Gibson, David M. Markie, Peter Tsai, Cherie Blenkiron, Sandra Fitzgerald, Paula Shields, Patrick Yap, Ben Lawrence, Cristin Print, Stephen P. Robertson
The search space within which to find a specific genetic explanation for monogenic disorders is becoming well-refined. There are approximately 19,000 protein-coding genes in the human genome, covering approximately 50 Mb of DNA sequence, and it is in this portion of the genome—the exome—that an estimated 85% of Mendelian molecular diagnoses will be found (Blackburn et al. 2015; Chong et al. 2015; Valencia et al. 2015). Whole exome sequencing (WES) is increasingly being used worldwide with studies of its utility demonstrating diagnostic rates of 16%–57% (de Ligt et al. 2012; Soden et al. 2014; Yang et al. 2014; Chong et al. 2015; Valencia et al. 2015; Wright et al. 2015; Monroe et al. 2016; Stark et al. 2016). Currently, mutations in 3849 genes have been proven to be responsible for 6121 Mendelian phenotypes (OMIM Gene Map Statistics 2017).