CRISPR Genes – A way to eradicate Autism to Cancer to Zellweger syndrome and everything in-between
It was September 20th 2001. It was the day my mother would be screened if the fetus (i.e. me in my glorious self) showed an elevated risk for Downs Syndrome. A triple screen blood was performed which measured the quantities of three substances: Alpha-fetoprotein (AFP) which is produced by the fetus, human chorionic gonadotropin (hCG), and unconjugated estriol which is produced by the placenta. The next 24 hours were one of the most stressful, my dad, tells me until the nurse called with the good news that all the bio markers were normal. Four weeks later they were not so lucky when my mother was diagnosed with Gestational Diabetes. So for the next 6 months my mother was on a diet so strict she needed to wake up at 2AM in the morning to eat just enough so I would develop normally. I think everything turned out OK for me but such is not the case for many who are affected by a variety of diseases from Autism and Alzheimer’s to Zellweger syndrome and the emperor or all maladies, Cancer that trace their way back to faulty genes. THE genome is written in an alphabet of just four letters. Four letters that result in a dizzying number of combinations. In almost every cell in every human body there are two copies of the human genome, one from the mother, one from the father. In each of those genomes there are about 20,000 genes, each of which contains the recipe for a specific protein in the form of a sequence of chemical “letters”. To date, medicine has recognized about 6,000 diseases that can be traced to a problem with one or another of those genes—a disorder in which a missing or garbled sequence of DNA leaves the body unable to make a particular protein, or causes it to be made in an abnormal form. Some of these single-gene disorders are well known: Tay Sachs; sickle-cell anaemia; haemophilia. Others, such as Sanfilippo syndrome, are the sort of thing you learn of only when a child you care about turns out to be the one in 70,000 that it afflicts. Since genetic engineers assembled their first tool kits in the 1970s heart-broken parents and medical researchers have longed to use such technologies to fix these faulty genes. The first clinical attempts at such “gene therapy” began in the 1990s, with viruses used to add needed genes to cells that lacked them. The new genes could not be guaranteed to slot into the right place in the genome; this often meant they did not in practice produce much protein; it also meant there was a risk that, by disrupting other genes, they could cause cancer. There were indeed cancers in some early trials; there was also a case in which a patient died of a lethal immune reaction to the virus used to carry the gene. Some years ago, biologists discovered an odd feature in the genomes of some bacteria that they described as “Clustered, Regularly Interspaced Short Palindromic Repeats”—CRISPR for short. Bacteria use them …show more content…
Although these entire products have the prospective for incredible patient benefit, yet these products still have the unknown risks. So to ensure the patient get the safest treatment, the scientist must accept all the responsibility of the procedure so that irrational risks can be prevented. In a nutshell, gene therapy is a very vital method for treating patient with disease especially genetic disorder. From this research, gene therapy can be seen as the new revolutionary technique advancing in the genetic engineering to treat the diseases. Current treatment so far has given excellent results from all standpoints. Upcoming endeavors should be focusing on how to improve the efficiencies of gene therapy so that the society will not argue the importance of this method in treating the patient. Even though human gene therapy treatment raises many controversial, the outcome of this technique is symbolized as a very high level of expertise and should be accepted. Viewing from the positive angle, the advantages of gene therapy overshadow the disadvantages. Gene therapy should be legalized because it is body-friendly, cost effective and improve the quality of
It was September 20th 2001. It was the day my mother would be screened if the fetus (i.e. me in my glorious self) showed an elevated risk for Downs Syndrome. A triple screen blood was performed which measured the quantities of three substances: Alpha-fetoprotein (AFP) which is produced by the fetus, human chorionic gonadotropin (hCG), and unconjugated estriol which is produced by the placenta. The next 24 hours were one of the most stressful, my dad, tells me until the nurse called with the good news that all the bio markers were normal. Four weeks later they were not so lucky when my mother was diagnosed with Gestational Diabetes. So for the next 6 months my mother was on a diet so strict she needed to wake up at 2AM in the morning to eat just enough so I would develop normally. I think everything turned out OK for me but such is not the case for many who are affected by a variety of diseases from Autism and Alzheimer’s to Zellweger syndrome and the emperor or all maladies, Cancer that trace their way back to faulty genes. THE genome is written in an alphabet of just four letters. Four letters that result in a dizzying number of combinations. In almost every cell in every human body there are two copies of the human genome, one from the mother, one from the father. In each of those genomes there are about 20,000 genes, each of which contains the recipe for a specific protein in the form of a sequence of chemical “letters”. To date, medicine has recognized about 6,000 diseases that can be traced to a problem with one or another of those genes—a disorder in which a missing or garbled sequence of DNA leaves the body unable to make a particular protein, or causes it to be made in an abnormal form. Some of these single-gene disorders are well known: Tay Sachs; sickle-cell anaemia; haemophilia. Others, such as Sanfilippo syndrome, are the sort of thing you learn of only when a child you care about turns out to be the one in 70,000 that it afflicts. Since genetic engineers assembled their first tool kits in the 1970s heart-broken parents and medical researchers have longed to use such technologies to fix these faulty genes. The first clinical attempts at such “gene therapy” began in the 1990s, with viruses used to add needed genes to cells that lacked them. The new genes could not be guaranteed to slot into the right place in the genome; this often meant they did not in practice produce much protein; it also meant there was a risk that, by disrupting other genes, they could cause cancer. There were indeed cancers in some early trials; there was also a case in which a patient died of a lethal immune reaction to the virus used to carry the gene. Some years ago, biologists discovered an odd feature in the genomes of some bacteria that they described as “Clustered, Regularly Interspaced Short Palindromic Repeats”—CRISPR for short. Bacteria use them …show more content…
Although these entire products have the prospective for incredible patient benefit, yet these products still have the unknown risks. So to ensure the patient get the safest treatment, the scientist must accept all the responsibility of the procedure so that irrational risks can be prevented. In a nutshell, gene therapy is a very vital method for treating patient with disease especially genetic disorder. From this research, gene therapy can be seen as the new revolutionary technique advancing in the genetic engineering to treat the diseases. Current treatment so far has given excellent results from all standpoints. Upcoming endeavors should be focusing on how to improve the efficiencies of gene therapy so that the society will not argue the importance of this method in treating the patient. Even though human gene therapy treatment raises many controversial, the outcome of this technique is symbolized as a very high level of expertise and should be accepted. Viewing from the positive angle, the advantages of gene therapy overshadow the disadvantages. Gene therapy should be legalized because it is body-friendly, cost effective and improve the quality of