The Biological Basis for Gene Therapy

Gene therapy is a burgeoning field of medicine in which genes are introduced into the body to treat disease. inheritance of gene control and provide the basic biological code for determining specific functions in the cell. Gene therapy aims to deliver genes to correct or replace functions to control the disease cells that are essential to do their job.

Gene Therapy
Gene therapy is a burgeoning field of medicine in which genes are introduced into the body to treat disease.  inheritance of gene control and provide the basic biological code for determining specific functions in the cell.  Gene therapy aims to deliver genes to correct or replace functions to control the disease cells that are essential to do their job.  Somatic gene therapy introduces therapeutic genes in tissues at the cellular level, or to treat an individual.  germline gene therapy inserted genes in reproductive cells, or possibly into embryos to correct genetic defects that can be transmitted to future generations.  Originally conceived as an approach to treat inherited diseases like cystic fibrosis and Huntington’s disease, the extent of gene therapy was expanded to include potential treatments for cancer, arthritis and infectious diseases.  Although gene therapy studies in humans have developed rapidly, many questions surrounding its use.  For example, some scientists are concerned that therapeutic genes can cause disease.  Others fear that the treatment of seeds can be used to control human development for other diseases, such as intelligence or appearance.

The biological basis for gene therapy
Gene therapy has emerged from the science of genetics and how inheritance works.  Scientists know that life begins in a cell, the basic component in all multicellular organisms.  Humans, for example, consists of billions of cells, each of which performs a specific function.  In the cell nucleus (the center of a cell to regulate the chemical functions) are pairs of chromosomes.  These filamentary structures consisting of a single molecule of DNA (deoxyribonucleic acid), which represents the blueprint for life in the form of codes, or genes that determine hereditary characteristics.

A DNA molecule looks like two ladders with a far-side at a time, then wound around each other.  Steps to respond (resulting in a spiral structure type) and are called base pairs.  base pairs of molecules consists of nitrogen and arranged in specific sequences.  one million base pairs, or sequences, you can have a single gene, specifically defined as a segment of chromosome and DNA that contains the hereditary information is determined.  The gene, or a combination of genes that are formed by these base pairs eventually guide the growth of an organism and properties through the production of certain chemicals, mainly proteins, which carry most of chemical and biological features body reactions.

Scientists have long known that changes in the genes found in the cells can cause hereditary diseases like cystic fibrosis, sickle cell anemia and hemophilia.  Similarly, errors in the total number of chromosomes can cause diseases such as Down syndrome or Turner’s syndrome.  As the study of genetics advanced, but scientists have discovered that modified gene sequence can also make people more vulnerable to diseases such as atherosclerosis, cancer and even schizophrenia.  These diseases have a genetic component, but is also influenced by environmental factors (such as diet and lifestyle).  The goal of gene therapy is the treatment of diseases by introducing functional genes into the body to change the cells involved in the disease process, either by replacing missing genes or a copy of functioning genes to replace others who do not work.  The inmates genes can be naturally occurring genes that produce the desired effect or may be genetically modified (or altered) genes.

Scientists have managed to manipulate the structure of a gene in the laboratory of the early 1970s through a process called gene splicing.  The process involves taking a DNA fragment containing the desired sequence-specific gene and inserted into the DNA of another gene.  The resulting product is called recombinant DNA and genetic engineering process.

There are basically two types of gene therapy.  germ of inmates genes in gene therapy of sex cells (sperm and eggs) or embryos may one day hope to correct the genetic defects that can be transmitted to future generations.  Most current work on the application of gene therapy, but has been in somatic gene therapy.  In this type of gene therapy, the therapeutic genes into tissues or cells produce a protein or a natural substance that is missing or not functioning in a patient.

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