Understanding Gene Therapy: Proteins And Their Roles
Proteins are responsible for a multitude of cellular functions that are crucial to the survival of cells and the organs they populate. They are produced by a process called translation. It is built based on the blueprint, or code, that outlines its structure. This code is found in the genes, also known as DNA. Therefore, defective genes cause malfunctions in metabolic pathways, hence diseases. When either too much or too little of a certain protein is produced or even when a defect in the production process occurs, it leads to a protein being formed incorrectly. And hence, these malfunctions lead to various genetic diseases.
If the precise code for making the protein is known, doctors can synthesize it in a test tube and then deliver it into the patient’s cells. Various advancements in the fields of Gene Therapy have led to successful treatment of many genetic disorders.
What Is Gene Therapy?
Merriam-Webster’s Collegiate Dictionary defines gene therapy as the insertion of normal or genetically altered genes into cells usually to replace defective genes especially in the treatment of genetic disorders. By using gene therapy, we can go to the base of the disorder instead of use conventional medicines only to alleviate the symptoms.
There are three methods used to deliver the genetically altered material.
- Retroviruses or Retrotransposons: Retroviruses are viruses that can transfer their own genetic information and also genetically alter the patient’s genome. These viruses are unable to copy themselves but still pose a problem in altering protein synthesis when these retroviruses splice a patient’s cells. This is when retrotransposons come into play. There are parts of DNA from a cell that can copy themselves onto other sites in cell’s genome. The only type of such a transposon is a yeast transposon, called Ty3. This yeast transposon is still under research.
- Helium gun: This technique involves bombarding target cells with gold molecules coated with genetically altered genes. This is done with the help of a pressurized gun that is filled with helium.
- Liposomes: Liposomes are hollow, fat molecules present in the form of a solution. This method is being researched by the Royal Brompton Hospital in London headed by Natasha Caplen (Glauisisus.1996). She is using liposomes in experimentation with cystic fibrosis. Cystic fibrosis is a disease that is caused by a chloride ion build up in the respiratory tract which causes difficulty in breathing. By inhaling liposomes coated with genetically altered genes prevented the buildup of chloride ion and they recorded a significant decrease in chloride ion levels. Unlike retroviruses, this method does not pose any harmful side effects.
Gene Therapy As Future Of Personalised Healthcare
The first disease that was approved for gene therapy was adenosine deaminase deficiency or ADA. Children who have this deadly disease are seriously prone to most of the minor illnesses. If untreated, it becomes the reason for death too.
The same procedure is under development and under research for AIDS.
Another major issue that attracts gene therapy’s attention is cancer. An experiment was done at the University of California in LA led by Habib Fakhrai, which involved studies on rats with tumors. They had sixteen rats of which eleven of them were treated with 9L gliosarcoma, the genetically altered gene. This treatment blocked the synthesis of a protein called TGF – beta. A molecule responsible for decreasing the immune action. After all was said and done the eleven rats that received the treatment were alive while the others died of cancer.
There are many other diseases that may be cured by gene therapy some of which include Rubinstein-taybi syndrome, partial epilepsy, cataract, prostate cancer , male infertility, Alzheimer’s, schizophrenia, usher syndrome, and maternal acute fatty liver of pregnancy. Genes of these diseases have been identified since 1995.
- Untapped Potential
One notable factor that gives gene therapy the edge is the remarkable therapeutic potential it has.
- Replacement of Defective Cells
We are constantly attacked by newer, more dangerous and vituperative types of germs and pathogens. Although many of such diseases can be treated or cured medically, there is no cure for genetic disorders unless defective cells are replaced by proper ones which is what gene therapy does.
- End of hereditary diseases
With gene therapy, the cells carrying the genetic disorders are altered. So fixing them before they could be passed on could end the line of family members getting the same illness that debilitated their relatives.
- Little Risk of Mutation
Unlike many other types of cellular treatments, gene therapy has a very low risk of the genes that are used mutating. This is because they are not necessarily “new” genes, but simply duplicate of genes that we already have within our body.
- Imagination comes true:
Gene therapy makes it possible for one to produce a cell, tissue, an organ, of even an organism of their choice.
- A Potential threat due to weaponization:
Genes are the mastermind of our functioning, if altered with bad intentions and modifying organisms to build a greater power, they can pose a serious threat to the society.
- Damage In The Gene Pool:
If gene therapy was performed to a certain degree, it possibly could permanently change the human gene pool.
This treatment therapy may possibly be for the rich only, and without the further advancement in technology, could make the rich richer and poor poorer.
- Rise in Disorders:
There’s an exact point in the host genome where the right genes should be brought in and there are no assurance that the viral enzyme responsible for this step will be able to bring in the right genes at the exact point in the host genome. If there’s an error in the process, the results could bring about severe disorders. In addition, the body may destroy the vector perceiving that it is a foreign body.