Quiz 11: Medical Biotechnology


Molecular biology concerns with the study of living structures at the molecular level that includes proteins, deoxy ribonucleic acid (DNA) and ribonucleic acid (RNA). It forms the foundation for increasing growth areas such as cell biology, genomics, diagnostics, and therapeutics. Diagnostic techniques of molecular biology are largely used in several areas comprising immunology, pathology, haematology, and microbiology. Allele specific oligonucleotide (ASO) is one of the techniques of molecular biology. It helps in the recognition of single base pair mutation. Short tagged probes that are about 20-25 nucleotides long, are left to aligned with the stacked target DNA. Hybridization between complementary base pairs would occur with high precision. Alteration at a single base can hamper the process of hybridization. Then, the target DNA is washed and the tagged probes that were not able to hybridize were removed. Then, examination of target DNA is done to spot the hybridized probe through the process of radioactivity and fluorescence. In such an experiment, a control experiment must be used to illuminate the difference. This technique can be used for detection of genetic diseases such as sickle cell anemia. Western blotting is another technique of molecular biology. In this process, proteins are parted according to their size, on a thin gel through the process of sodium dodecyl sulfate polyacrylamide gel electrophoresis (SDS-PAGE). The proteins parted on the gel are then shifted to a nitrocellulose or nylon membrane. Then, the membrane can be explored with a solution of antibodies. Labeled antibodies explicitly bind with a specific protein. This can be visualized by number of methods such as chemiluminescence, or autoradiography. This technique can be used to diagnose cancer and infection of Hepatitis B.

Gene therapy includes the application of deoxy ribonucleic acid (DNA) as a therapeutic agent for the treatment of a disease. This term has been derived from the notion that DNA can be used for making alteration in the mutated genes in the defective cell of an individual to treat the ailment. Some of the techniques used for transporting therapeutic genes in the cell involve using viruses as vectors, by inserting naked DNA in the cell, or through liposomes. Differences between ex vivo and in vivo gene therapy are listed in the table below: img Some of the ongoing researches on gene therapy are described below: • A set of patients suffering from severe coronary artery infection are treated through angiogenic gene therapy. They were not responding to any other treatments during clinical trials. With the gene therapy, however, their injured blood vessels are rebuilt. • After years of studies, a novel gene has been found that strongly regulates the immune system of the body and increases the risk of Alzheimer's disease three-fold. It has been discovered by whole genome sequencing through deCODE genetics that a variant of gene TREM2 is common in patients with Alzheimer's in comparison with a control group. • The first gene therapy to get consent for use is glybera. It includes treatment for the patients with deficiency in the enzyme lipoprotein lipase, thus suffering from acute pancreatitis. Prophylactic gene therapy brings mutated gene that is associated with a disease back to its normal function. However, before using any gene therapy, there are a number of things that need to be considered: • It is very difficult to get an approval from the Food and Drug Administration of the United States for the treatment of a suggestive condition. It is important to look after the complications that may come up along with the therapy. • It is comparatively easy to get consent for gene therapies that are for short-term. • It cannot be said with confidence that the repair of the particular mutation will certainly treat the ailment completely.

Stem cells: These are a kind of mother cells that have the ability to become any kind of cell in the body. These cells have two special characters. They are self-renewal and differentiation. These cells can grow and multiply indefinitely through mitosis to produce populations of identical stem cells. They can differentiate into any kind of specialized cell in the body. Therefore, they are called with the name "pluripotent." There are different types of stem cells. They are embryonic stem cells, adult stem cells, amniotic stem cells, and induced pluripotent stem cells. • The stem cells that are derived from embryos are called "embryonic stem cells." They are derived from the embryos of humans and animals and grown in the laboratory using in vitro fertilization techniques. Those derived from human embryo are called "human embryonic stem cells (hESCs)." • The stem cells that are derived from the adults are called "adult stem cells (ASCs)." They are otherwise called somatic stem cells. These cells reside in the tissue of mature adults and can be cultured and differentiated to produce other kinds of cells. Usually these are in small numbers. They are isolated from different places such as brain, hair, skin, intestine, bone marrow, pancreas, fact, mammary glands, teeth, blood, and muscle. It has been found that these cells are also present in fat tissue and can be isolated using liposuction. • Some specialized adult cells are reprogrammed genetically to assume a stem-cell like state. Such new types of stem cells are called "induced pluripotent stem cells (iPSCs)." • Recently, scientists have isolated stem cells from human amniotic fluid in the lab. Those stem cells are called amniotic fluid derived stem cells (AFS). The uses of stem cells: • Stem cells can be used for treating different types of diseases. Research has been going on to treat Parkinson's disease, heart failure, diabetes, cerebral palsy etc. with the stem cells. The diseased tissue will be replaced with the new healthy tissue developed from the stem cells. • Stem cells can also be used to screen new drugs and toxins, without using them on humans and animals directly. • Stem cells can also be used for understanding various birth defects.

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