Our world is now facing the challenges of rare diseases that are caused by faulty genes. Allen J Frances, MD, wrote, “Different genotypes (patterns of genes) produce different phenotypes. A few medical illnesses are phenotypes that are the downstream result of an aberrant allele of a single gene that produces a malfunctioning protein. For example, Huntington’s disease is caused by an abnormal protein called huntingtin produced by a single defective gene. If you have the gene, you will get the illness.”
Even major psychiatric disorders have a genetic component. “Anxiety disorders, PTSD, OCD, and major depressive disorder are about 20-45% inherited, alcohol dependence and anorexia nervosa are 50-60% inherited, whereas bipolar disorder, autism spectrum disorders, schizophrenia, and ADHD are upwards of 75% inherited,” wrote Emily Deans, MD.
Studying genetics is not just for physical diseases and disabilities. Rebecca A. Housel, PhD, said that “genetics are increasingly important in the study of mental health.”
Revolutionary technology has been very busy in finding the cure to these genetic disorders and scientists are leaving no stone unturned.
The genetic disorder occurs at a ratio of 1:10 of every American and approximately 350 million people worldwide. Studies have shown that 30% of deaths in children are caused by genetic disorders. Our doctors lack prior knowledge and cure to treat ailments and find sources of these diseases. It is statistically proven that there are about 7.9 million infants or 6% of world births where children are born with severe genetic conditions and 50% of the disease is yet to remain unknown.
Understanding Genetic Conditions
Today, a new study has come to light. In recent years, biologists have conducted thorough and dedicated studies on how to engineer sequences of the genome to understand genetic conditions better. Since the first link of how a disease makes a defect in a specific gene, the diagnosis of the gene becomes an aim for researchers and biologists. It allows them to understand more, and to identify genetic conditions and improve their treatment rapidly.
Innovations in the molecular model with advanced mathematical methods have permitted a better solution for new analytical techniques. It has become useful in explaining further the most complicated genetic conditions. Identifying the genetic defects requires next-generation sequencing to sequence genomes of the affected patient, producing a considerable amount of data.
CRISPR (Clustered Regularly Interspaced Short Palindromic Reports)Was Born
Here comes the light of this era. It is what biologists called the Genome editing and CRISPR “crisper.” CRISPR has become the most popular way of genome engineering. 2017 was the start of a new era in Molecular Biology. Researchers have successfully operated a molecular tool to infuse blood in a patient with a severe inherited disease.
The University of Alicante in Spain conducted the discovery of CRISPR. In the year 1993, Franco Mojica was the first researcher to develop what is now called CRISPR locus. He coined the term CRISPR utterly throughout the year 1990 and 2000 onwards.
The CRISPR system is the adaptive immune system that can be used by microbes to defend themselves against bad viruses invading and destroying their DNA sequences. CRISPR then could be a reliable way of editing genomes in living things, genomes of plants, mice, even humans. The ability of the CRISPR to remove unneeded traits and potentially add beneficial characteristics with more precision is what makes CRISPR the most popular way of genome engineering.
Researchers in MIT and Harvard also have made significant advance enhancement to CRISPR gene editing. This time, the refining focused on individual DNA “base pairs.” David Liu, a chemist and a chemical professor at Harvard, says that in about 50,000 certified genetic conditions known to humans, there are about 32,000 that are directly correlated with cystic fibrosis and sickle cell anemia. These are caused by a mutation in a single base pair. Refining CRISPR to solely focus on twisting DNA base pairs was their hope to lead scientists in a more precise and accurate form of treatment for genetic diseases.
CRISPR is said to have the capacity to change DNA sequences in the most effective means. It is programmable. It alters and removes DNA strands and can even tell what kind of change it needs to be done.
Innovations on CRISPR also brings molecular biology to its next level. CRISPR can now record every activity inside the reparation of DNA cells. It comes with an analog memory device responsible for its longitudinal recording.
The genetic field today is now changed because of revolutionary new technology entering the stage. CRISPR (Clustered Regularly Interspaced Short Palindromic Reports) changes everything we know about gene editing. It indisputably has a great potential to change humanity forever.
The innovation made its shift when scientists figured out that the CRISPR system is programmable. CRISPR can copy the DNA and modify and put the system in a living cell. Apart from being accurate, cheap, and easy, CRISPR renders the capacity to edit live cells, switch genes into an on and off mode, and target and study particular DNA sequences. It works for every type of cell: microorganisms, plants, animals, and humans. Scientists and researchers believed that in a few decades, CRISPR therapy might be able to cure HIV and other retroviruses that can be located in human DNA, even cancer.
The first clinical trial of CRISPR to cure cancer dates back 2016. Today, US companies are still in the process of conducting gene editing trials on patients who are eligible to participate in the first human-phase 1 CRISPR.
CRISPR is now taking us to a new level of molecular biology. Can it be the answer to our questions on evolution? Or can it give us a cure to the dreadful ailments caused by genetic defects?
Many are hoping that CRISPR can lead us to our fountain of youth that is said to be embedded in our DNA.
Cohen, J. (n.d.) CRISPR. Retrieved from http://www.sciencemag.org/topic/crispr.
Belluz, J., & Irfan U. (2017, October 25). 2 new CRISPR tools overcome the scariest parts of gene editing. Retrieved from https://www.vox.com/2017/10/25/16527370/crispr-gene-editing-harvard-mit-broad.