How to Detect and Treat Genetic Diseases

There are many genetic diseases which may become dangerous if we do not know how to prevent early. Gene therapy is one of the prominent ways of treating genetic diseases. According to gene therapy, the genes are inserted into individual cells and tissues. Depending on the disease, the specific genes are corrected and inserted into the cells. However, there are still numerous researches based on gene therapy for various kinds of genetic disorders.

For people affected by some genetic diseases, antibiotics and a potent immune system hormones are used during the treatment. However, it actually does not cure the disease. It only reduces the infection, and consequently the vigor of the disease.

Some studies have proved that genetic diseases can be treated by altering the method of conversion of DNA into proteins. This method of treatment worked well for some genetic diseases such as Spinal muscular atrophy. This method is being tested for treating few other genetic diseases too. Gene transfer methods, which were initially used in animals like dogs are now being used to treat human eye diseases.

Gene identification is done as a part of treatment for genetic eye diseases. This will help in identifying the appropriate treatments for some genetic eye diseases and hence getting treated on a timely basis like wearing appropriate glasses becomes the absolute requirement of the patient. Moreover, gene identification also helps in preventing few genetic eye diseases.

However, obesity, which is one of the most common genetic diseases or disorders, as the case may be, is treated with a healthy diet program or weight loss program. Regular exercises will help in losing some weight. Yet, it is essential to consult a physician or geneticist to confirm that your obesity is due to genetic disorder only. The weight of your family members has to be taken into account for confirming that it is a genetic disorder.

People having diabetes (type I) must get their children regularly checked. They must prevent their family members including children from taking excess sugar or sweets. Diabetes can be controlled by regular medication. Cardio exercises can keep the insulin level in control. It is one of the most common genetic diseases, which actually has no permanent cure.

Not all genetic disorders can be cured. There are however drugs to control most of these genetic diseases or disorders. These drugs can only control or reduce the vigor of the disease. Treatments for many genetic diseases are still under study. Unless proper treatments are being introduced, it is essential to pay more attention to prevention of these type of diseases.

All You Need To Know About Gene Therapy

The human body is comprised of various organs that each have a specialized role in maintaining the proper health of a person. The brain is involved in thought, reasoning and, in general, controlling our actions; the heart sends blood around our body supplying all the organs with oxygen; the lungs oxygenate our blood thus providing the energy we need to function; the stomach, kidneys, liver, intestine and bladder extract nutrients from our diet and remove toxins. Each organ plays an essential and unique part keeping us alive.

To function correctly, an organ comprises billions of cells of different types, each arranged in tightly controlled structures that form the overall architecture of the body. It is the cells that are in point of fact responsible for the proper functioning of the organ. If an organ is malfunctioning, then to treat it, we must restore the smooth operation of these cells.

Basic Cell Biology

Most cells are made up of similar components: a nucleus, the part of the cell containing genetic information; a variety of organelles, small elements that carry out processes such as energy production, much like the way that different organs perform specific functions of the body (e.g. lysosome, mitochondrion, Golgi etc); the cytoplasm, the liquid medium that comprises the cell, and the plasma membrane, the element that gives the cell its shape.

The nucleus codes for all the information required to produce the cell. Each organelle and cellular makeup are made up of protein, sugars, and lipids (fatty compounds), and the nucleus not only encodes for the production of each of these components but also the information necessary for their correct assemblage and final position. This information is enclosed within the cell's DNA, which is the principal constituent of the nucleus and is tightly condensed in a highly organized manner in the nuclear membrane.

Our Genes

Within the nucleus, our DNA is arranged into 23 sets of chromosomes (or 22 pairs, and one X chromosome and Y chromosome if you are male). These 46 chromosomes are communally known as the human genome, as they contain all of the genes that act as the blueprint for the human body. We can imagine of our DNA as a long linear molecule that is split into 46 separate parts (i.e. the chromosomes). Within each chromosome, there are thousands of genes lined up one after the other one after another and split by intergenic regions. Each gene is a unit of DNA that encodes for a certain protein, with a unique function. It is the combination of many discrete proteins, and their actions on other molecules like sugars and lipids, that comprises the basis of the organelle, and by consequence, of the cell itself.

So one can imagine that in a pathological situation, where an organ is malfunctioning, we can time and again trace the dysfunction to a particular protein that is not working correctly. These protein malfunctions can either be genetic, or acquired in the course of

• An infection,
• An autoimmune reaction,
• Untimely tissue degeneration
• The formation of cancer.

So, in any condition where a disease can be traced to a malfunction of protein, or where a protein of known activity can restore the proper functioning of a cell, gene therapy can be used. This is just because we can now use the correct gene to deliver the exact type of the protein to the cell we want to fix. It is significant to note that by delivering genes exclusively into diseased cells, there is no opportunity of conveying this new genetic information in the future to our children. To do so, our germ cells would have to be the target for gene transfer, a process that is illegal, and extremely technically demanding.

Application of Genetic Therapy

Effectual gene transfer into human cells is known to be the major challenge the Gene Therapy. Gene transfer vector have to be safe, introduce its DNA cargo into a satisfactorily large amount of cells to create a biological response and mediate transcription of the desired gene for a long duration. Identifying a vector that meets these criteria has proven to be a testing task.

Gene Therapy is more often than not used in pathologic conditions where A cells of a distinct organ or system do not function correctly because they do not have the right protein WHICH is required to perform the desired task.