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Nanomedicinal Genetic Manipulation: Promising Strategy to Treat Some Genetic Diseases
Published in Sarwar Beg, Mahfoozur Rahman, Md. Abul Barkat, Farhan J. Ahmad, Nanomedicine for the Treatment of Disease, 2019
Biswajit Mukherjee, Iman Ehsan, Debasmita Dutta, Moumita Dhara, Lopamudra Dutta, Soma Sengupta
Genetic diseases may be categorized as single gene Mendelian inheritance, single gene non-Mendelian inheritance, chromosomal abnormalities and multifactorial inheritance (Stoppler, 2017; Robinson et al., 2017; National Human Genome Research Institute, 2017) Single gene Mendelian inheritance: It is also known as monogenetic inheritance. This is due to alterations and mutations in DNA sequence of a single gene only. This inheritance is again divided into three groups such as autosomal dominant, autosomal recessive and sex-linked. In every 200 births, one birth affects by single gene Mendelian inheritance causing genetic diseases. Some examples of such diseases are sickle cell anemia, cystic fibrosis, Marfan syndrome, hemochromatosis, Huntington’s disease, etc.Single gene non-Mendelian inheritance: It occurs because of triple recurrence expansions within or near specific genes, mutations within imprinted genes or mitochondrial DNA, etc. Fragile-x syndrome, Prader-Willi syndrome, Angelman syndrome, etc. belong to this category.Chromosomal abnormalities: Chromosomes, which are made by DNA and protein, are the transporters of genetic substances. Numerical and structural abnormalities in autosome or abnormalities in sex chromosomes are the probable reason for this disorder. Examples of such genetic diseases or disorders are Down syndrome (due to copies of chromosome 21 thrice), Klinefelter syndrome (47, XXY), Turner syndrome (45, X0), etc.Multifactorial inheritance: It is related to the mutations in multiple genes as well as blend of environmental influences. This is also acknowledged as polygenic inheritance. Diabetes, cancer, obesity, high blood pressure, arthritis, Alzheimer’s disease, etc. are the examples of this category. As mentioned earlier, in this chapter, we have selected and discussed on genetic manipulation of three diseases by nanomaterials of this category (Table 5.1).
The potential for the use of gene drives for pest control in New Zealand: a perspective
Published in Journal of the Royal Society of New Zealand, 2018
Peter K. Dearden, Neil J. Gemmell, Ocean R. Mercier, Philip J. Lester, Maxwell J. Scott, Richard D. Newcomb, Thomas R. Buckley, Jeanne M. E. Jacobs, Stephen G. Goldson, David R. Penman
Building on previous ideas (Craig et al. 1960), Burt (2003) described how gene drive systems based on a homing endonuclease gene (HEG) could be a means for population suppression of pests (Burt 2003; Gould 2008). HEGs are enzymes that cut both strands of a DNA helix at a specific sequence (Belfort & Bonocora 2014). Organisms try to repair such cuts by homology-directed DNA repair (HDR), a process whereby an organism with a double-stranded break in its DNA will try and repair that break by copying any similar sequence it can find in the cell (Jasin & Rothstein 2013). To cause a homing event in a heterozygote, the HEG is inserted into one allele of the site that the HEG actually cuts. When the HEG cuts an empty site, HDR leads to the allele containing the HEG being copied into the cut site. Thus, a heterozygote for the HEG is converted to a homozygote (Figure 1). It is this ‘homing’ property that leads to non-Mendelian inheritance of the HEG and is the basis for the gene drive mechanism (Burt 2003). Modelling has shown that population suppression is particularly efficient if the HEG is targeted to a gene essential for females but not males, or a gene required for reproduction in one sex (Burt 2003; Deredec et al. 2008).