Biotechnology has made breakthroughs that laid groundwork for the modern biotechnology that came when the structure of DNA was discovered in the early 1950s. To understand how this insight eventually led to biotech therapy, it’s helpful to understand the role of DNA in health and disease.



What does DNA do?
DNA is a long molecule found in the nucleus of a cell. It provides the blueprint for the construction and operation of a life-form. The information in DNA is stored as a code made up of building blocks, called nucleotides. The order in which the nucleotides appear is akin to the order of the letters that spell words and form sentences and stories. In the case of DNA, the order of nucleotides forms different genes. Each gene contains the instructions for a specific protein.

With a few exceptions, every cell holds a complete copy of that organism’s DNA. The genes in the DNA of a particular cell can be turned on or turned off depending function and needs. Once a gene is activated, the information it holds is used for making, or expressing, the protein for which it codes. Many diseases result from genes that are improperly turned on or off.

How Does the Body Make a Protein?
Protein production is a process that includes transcription and translation. During transcription, the original DNA code for a specific protein is rewritten onto a molecule called messenger RNA (mRNA); mRNA has nucleotides similar to those of DNA. Each successive grouping of three nucleotides forms a codon, or code, of different amino acids, which are the building blocks of proteins.

During translation, a cell structure called a ribosome binds to a ribbon of mRNA. Other molecules, called transfer RNAs, assemble a chain of amino acids that matches the sequence of codons in the mRNA. Short chains of amino acids are called peptides. Long chains, called polypeptides, form proteins.

What functions do proteins control?
The amino acids that form a protein interact with each other, and those complex interactions give each protein specific 3-D structure. That structure determines how a protein functions and what other molecules are impacted. Common types of proteins are]Enzymes, which put molecules together or break them apart.
Signaling proteins, which relay messages between cells, and receptors, which receive signals sent via proteins from other cells.
Immune system proteins, such as antibodies, which defend against disease and external threats.
Structural proteins, which give shape to cells and organs.
Given the tremendous variety of functions that proteins perform, they are sometimes referred to as the workhorse molecules of life. However, when key proteins are malfunctioning or missing, the result is often disease....

How does genetic engineering work?
Genetic engineering is the cornerstone of modern biotechnology. It is based on scientific tools, developed in recent decades, that enable researchers to]Identify the gene that produces the protein of interest.
Cut the DNA sequence that contains the gene from a sample of DNA.
Place the gene into a vector, such as a plasmid or bacteriophage.
Use the vector to carry the gene into DNA of the host cells, such as Escherichia coli (E coli) or mammalian cells grown in culture.
Induce the cells to activate the gene and produce the desired protein.
Extract and purify the protein for therapeutic use.

When segments of DNA are cut and pasted together to form new sequences, the result is known as recombinant DNA. When recombinant DNA is inserted into cells, the cells use this modified blueprint and their own cellular machinery to make the protein encoded by the recombinant DNA. Cells that have recombinant DNA are known as genetically modified or transgenic cells.

Genetic engineering allows scientists to manufacture molecules that are too complex to make with chemistry. This has resulted in important new types of therapies, such as therapeutic proteins. Therapeutic proteins include those described below as well as ones that are used to replace or augment a patient’s naturally occurring proteins (such as melanocyte stimulating hormone or potent analog Melanotan 2), especially when levels of the natural protein are low or absent due to disease. They can be used for treating such diseases as cancer, blood disorders, rheumatoid arthritis, metabolic diseases, and diseases of the immune system.

Monoclonal antibodies are a specific class of therapeutic proteins designed to target foreign invaders—or cancer cells—by the immune system. Therapeutic antibodies can target and inhibit proteins and other molecules in the body that contribute to disease.

Peptibodies are engineered proteins that have attributes of both peptides and antibodies but that are distinct from each.
Vaccines stimulate the immune system to provide protection, mainly against viruses. Traditional vaccines use weakened or killed viruses to prime the body to attack the real virus. Biotechnology can create recombinant vaccines based on viral genes.
These new modes of treatment give drug developers more options in determining the best way to counteract a disease. But biotechnology research and development (R&D), like pharmaceutical R&D, is a long and demanding process with many hurdles that must be cleared to achieve success.