Gene Transfer. To understand how genetic therapy works, you need to have a fundamental knowledge of the anatomy and exactly how a cell functions. In this section, we give you a very brief intro to human cell biology, providing enough background so that the you can now understand how it works. It’s our make an effort to try and dispel any possible misconceptions that interested parties could have about genetic therapy, and to introduce the subject to those thinking about pursuing further education in this area.

The body. Our body is composed of multiple different organs that each have a given role to maintain the good health of an individual. The mind controls our thought and reasoning; the center pumps blood around our own bodies supplying all the organs with essential nourishment; the lungs oxygenate our blood thus offering the energy we need to function; the stomach, kidneys, liver, intestine and bladder all function in unison to extract nutrients from your food and get rid of unwanted toxins. Each organ plays an vital as well as part keeping us alive.



To be able to accomplish its appointed role, a body organ contains huge amounts of cells of discrete types, each arranged in tightly controlled structures that from the overall architecture in the organ. It is the cells which are in reality to blame for the appropriate functioning of the organ. If an organ is misfunctioningn, then in order to treat it, we must fix cellular structure.

Basic Cell Biology. Most cells include similar components: a nucleus, has the genetic blueprint; a number of organelles, small elements that perform processes like wind turbine, similar to the manner in which different organs carry out specific functions in the body (e.g. lysosome, mitochondrion, golgi etc); the cytoplasm, the liquid medium that comprises the cell, and the plasma membrane, the structure that surrounds the cell and maintains its shape.

In several ways, it’s the nucleus that is the most important organelle of an cell, because its content has every piece of information required to produce each constituent with the cell. Each organelle and cellular makeup comprises of protein, sugars and lipids (fatty compounds), and the nucleus not merely encodes to the synthesis of every of the components, but the contains the instructions for their correct assemblage and final location. This information is contained inside cell’s DNA, which is major consituent in the nucleus which is tightly condensed in a highly organised manner inside the nuclear membrane.

THe Nucleus. Within the interior the nucleus our DNA is arranged into 23 multiple chromosomes (or 22 pairs, and something X chromosome and Y chromosome a high level man). These 46 chromosomes are together called the human genome, as they contain each gene that represents the blueprint of the body system. We are able to imagine in our DNA being a long straight molecule that’s split into 46 separate units (i.e. the chromosomes). Inside each chromosome you will find hundreds of thousands of genes arranged consecutively one after another, and separated by intergenic regions. Each gene is often a unit of DNA that encodes for a specific protein, with a exclusive function. It is the mixture of numerous proteins, as well as their actions on different molecules like sugars and lipids, that define the cornerstone of the organelle, and thus, with the cell itself.

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