WHAT ARE GENES? AND HOW TO IT APPLY TO GENE THERAPY?
Written by , Article reviewed and edited by Dr. Fine M.D.
Published on September 4th, 2019
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What is a Gene?
Genetics and Gene Therapy
What Are Genes?
Genes are the simplest functional components of life on earth. The entire collection of genes in an organism is known as its genetic code, also known as its DNA. The genetic code is a literal blueprint which has the capacity to create an organism.
A gene is a single data-point on a piece of DNA which controls some aspect of the organism. Genes control everything about us via the way that they allow for the creation of proteins and enzymes that are the building blocks of all life forms.
What Are Chromosomes?
Chromosomes make up the next level of genetic organization. All genes are particular aspects of a DNA molecule, and DNA is arranged into helix structures known as chromosomes. All organisms which have a nucleus have a specific number of chromosomes associated with their species which comprises a complete genetic code. There are rare instances where certain organisms have more or less chromosomes that normal, but this represents a mutation, and does not represent the species as a whole.
What is RNA?
RNA is derived from DNA and the genes, and is directly responsible for the production of proteins and enzymes which control the function of the body. The genetic code controls both the metabolic function as well as the structure of each individual cell within an organism.
Reproductive Chromosomes Unique
Sperm and eggs are unique cells within the body, because they only contain split chromosomes. When reproduction occurs, the two sets of half-chromosomes combine, creating an entirely new and randomized set of chromosomes, which has the capacity to create a brand new organism which shares the individual traits of both the mother and the father.
Molecularly, every gene is comprised of what are known as nucleotides. These nucleotides are the central building blocks of the genes, themselves. All nucleotides are designed in the same way. They contain three components: a phosphoric acid, a sugar, and a compound containing nitrogen. These nucleotides form the double-helix structures which are associated with the DNA molecule.
Importance of Junk DNA
Although a full set of chromosomes contain all of the data needed to completely design an organism, many of the genes within the DNA strands actually do not serve any direct purpose at all. Any DNA Segment that does not contribute to the production of proteins is known as Junk DNA. Junk DNA are scientifically referred to as Introns. All parts of the genetic structure that actually have the capability to create proteins are known as Exons.
Fascinatingly, the vast majority of DNA in the human genetic code is actually junk DNA. Only three percent of the DNA contained within your genetic code actually contribute to your development. Although it sounds like Junk DNA is absolutely worthless, it actually plays a real and significant role in the proper function of the chromosomes.
Every species has a specific number of chromosomes, and each of these chromosomes are organized in the same way. Changing the composition of the genes or the number of chromosomes, causes what is known as a mutation. When these mutations occur in normal cells, it can cause any number of problems. Junk DNA reduces the odds that these mutations occur, while also stabilizing DNA Structure.
What is the Science of Genetics?
Genetics is the scientific study of heredity and inheritance. The term Genotype refers to an organism's specific genetic makeup. Genotype refers to traits as they are expressed in the genetic code.
Phenotype, on the other hand, accounts for what we actually perceive. Phenotype refers to how the genes are ultimately expressed within the organism. The term Genome refers to the full blueprint of the genetic code of a specific species.
What Was the Human Genome Project
Our scientific knowledge of human genetics is incredibly new. The Human Genome was not fully uncovered until 2003. The Human Genome Project was responsible for the mapping of the entire human genetic code. The project was first propodddddsed in 1990 and took thirteen years and three billion dollars to complete. When we completed the blueprint in 2003, what we had was akin to a map without a legend.
Scientists have spent the last decade decoding the blueprint, attempting to discover the purpose of every gene in the genetic code. As researchers and scientists more fully understand the Human Genome, we will be able to take advantage of that knowledge in countless different ways.
What is Genetic Therapy?
Genetic Therapy is the use of Genetic Engineering and our knowledge of the Human Genome and those of other species in order to treat chronic diseases and disorders associated with various medical issues. Because the science of Human Genetics is so new and young, many of these techniques remain in the experimental phase.
At this point, the most complicated aspect of Genetic Therapy is how to appropriately administer the treatment to specific target cells. In order for these forms of treatment to have a powerful and long-lasting effect, they must be delivered to the necessary target so that they can reproduce and propagate among the surrounding tissues. There are two primary means by which Gene Therapy takes place:
Ex Vivo Therapy - In this form of treatment, tissue is extracted from the patient, which is then Genetically Altered and returned to the body. This form of therapy involves either treating human cells after they have been extracted, or altering the cells before returning them to the body.
The most promising research in regard to Ex Vivo Gene Therapy is in regard to neuro-degenerative diseases such as Parkinsons'. One way that scientists have learned to do this is through Ex Vivo Therapy. Defective cells are removed from the patient and fortified with copies which are genetically healthy. After the healthy cells have been given the chance to propagate, they are re-administered to the patient.
In Vivo Therapy - In this form of treatment, therapies which feature Genetically Engineered Medicines are administered directly to the patient. One way to do this in to inject a gene into a non-symptomatic or inactivated virus and let the virus spread the healthy genes to target cells.
Another In Vivo approach is to attach the corrective gene to a liposome so that it may enter the cell through its membrane. Liposomes are microscopic pouches surrounded in fat which have the ability to breach the cell membranes because of their lipid composition.
A third In Vivo approach is known as Chimeraplasty. In this form of Gene Therapy, Bio-Engineered Nucleic Acids are delivered via liposome in order to resolve pathogenic mutations. One way that this form of treatment works is by releasing a chemical that the human body is unable to produce naturally. Another way is by counteracting a pathogenic mutation, correcting it or rendering it inert. A third way that this form of treatment works is by marking cancer cells so that they can be more readily eradicated by other medical treatments.
First Use of Gene Therapy
Gene Therapy is one of the newest medical techniques available to mankind. In human patients, it was first used as a medical treatment in 1990, in order to treat a pediatric patient suffering from Adenosine Deaminase Deficiency.
ADA Deficiency is a significant medical disorder that severely restricts the immune system from functioning, and in some cases, it completely shuts down the Immune System, leaving the patient dangerously exposed to outside pathogens. The future potential for Gene Therapy is incredibly bright. Today, scientists around the world are coming up with new and innovative ways to treat disorders such as AIDS, various genetic disorders, as well as numerous cancers.
Gene Therapy Roadblocks
In spite of the obvious and exciting potential of Genetic Therapy, there are a number of roadblocks which slow down the development of new and useful treatments. Scientists have to be careful to create gene therapies which the human immune system does not perceive as a threat. In addition to this, researchers must be absolutely certain that the viruses that they use are unable to become virulent and dangerous over time. It is also important that Gene Therapies do not alter the genetic code of reproductive cells, in order to preserve the normal function of heredity.
State of Gene Therapy in the USA
In the United States, all Gene Therapies are considered medications and must be approved by the American Food and Drug Administration. The United States also has a panel which belongs to the National Institute of Health which is dedicated to the monitoring of Gene Therapies, known as the Recombinant DNA Advisory Committee.
Although Gene Therapy is a new field, or perhaps because of it, it is an incredibly lucrative and competitive field in both the public and the private sector. Many techniques have also been successfully patented.
Recombinant Hormone Replacement Therapy
Hormone Replacement Therapy is another common use of Recombinant DNA Technology. As opposed to En Vivo or Ex Vivo Therapy, Bio-Identical Hormone Replacement Therapy alters the genetic structure of microorganisms so that they produce a useful form of medication.
Bio-Identical Human Growth Hormone HRT is an example of this type of therapy. Although HGH can be derived directly from the pituitary gland of cadavers, the safest and most common method today is to re-engineer bacteria to secrete Human Growth Hormone which can be safely and sterilely extracted for medical use.
Specifically, E.Coli bacteria are redesigned to release Growth Hormone molecules that are completely identical to those created by the human body.
Bio-Identical Hormone Replacement Therapy is the future of safe and natural Hormone Restoration.
What Can Gene Therapy Treat?
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