Gene Editing
The advent of genome editing has revolutionized the study of the human genome over the past few years, enabling investigators to learn more about how single gene products contribute to disease in organism. In discovering the secret of human genome, our understanding leads us to conclude that genetics plays a significant role in nearly all
diseases. Like sickle cell disease, some diseases are caused by mutations that are inherited from parents and have been present since birth. Other diseases are caused by the
acquisition of mutation in a gene or group of genes that occur during an individual's lifetime.
Mutations such as these do not occur as a result of inheritance, but occur either randomly or as a result of exposure to environmental factors such as cigarette smoke.This includes many types of cancer and some types of neurofibromatosis.
Genomes and their constituent genes are made up of double-stranded DNA; this DNA can be broken intentionally or accidentally by radiation. through enzymes, particularly nucleases that have been engineered to target a specific DNA sequence, with this proteins double- strand breaks (DSBs) in DNA can be generated.
In the 1950s and following the discovery of DNA’s double-helix structure, gene editing was proposed as a way to treat diseases or alter traits.In the age of gene discovery in the mid- 20th century,scientists led to the conclusion that small variations in the DNA's base sequence can make the difference between health and disease.
Over the past 5 years, a completely novel system has been developed based on
fundamental research on bacterial systems of immunity to viral infections. The first such system, called CRISPR/Cas9, was created for use in editing the human cell's genome. It is based on RNA-guided targeting and is considerably simpler, faster, and less expensive than earlier methods.
CRISPR-Cas9 has been used in a variety of ways. For instance, it has been used to modify early embryos to created genetically altered species, and it has been injected into lab animals' bloodstreams to significantly edit the genes in certain tissue subsets. This new technology held the key to alleviating much human suffering. About the same time, reports from China indicated that human embryos had been used in gene-editing tests. The world's first gene-edited human babies were announced to be twin girls who were born in late 2018;
the kids were said to carry an edited gene that decreased the chance of contracting HIV.
