How Does Gene Therapy Work?

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Gene Therapy - Correct Faulty Gene With The Normal One

Gene therapy corrects defective genes that lead to diseases. Human gene therapy may include the insertion of normal genes, swapping abnormal genes for normal genes, repairing abnormal genes or regulating genes. The basics of gene therapy work on the premise that most diseases are caused from defective genes.

By altering or replacing genes, diseases can be treated or cured.

ABOUT GENES

Genes are carried on chromosomes and are the basic units of heredity. They encode instructions on how to make proteins. When genes are altered the encoded proteins are unable to carry out normal functions, resulting in genetic disorders. Hereditary diseases occur as a result of faults within the genes and chromosomes and gene therapy seeks to correct these faults.

HISTORY OF GENE THERAPY

The history of gene therapy first began in 1966, when it was first discussed. The first attempt to treat people using gene therapy was made in 1970, but it was unsuccessful. In 1990, the first approved gene therapy clinical trial was held. In the year 2000, Alain Fischer succeeded in correcting children with SCID-X1 - making it the first gene therapy cure.

How Does Gene Therapy Work?

So how does gene therapy work? In most types of gene therapy, a normal gene is inserted into the body to replace an abnormal gene. A vector is used to deliver the gene to the patient's cells. A vector may be a virus that has been altered to carry normal human DNA. Viruses encapsulate and deliver their genes to human cells. In gene therapy, the vector gives its genetic material into target cells, which helps restore the cell to a normal state.

Different Types of Viruses used as Gene Therapy Vectors

Some of the different types of viruses that are used as gene therapy vectors include:

Retroviruses. These are a class of viruses that can create double-stranded DNA copies of RNA genomes.
Adenoviruses. These viruses have double-stranded DNA genomes that cause respiratory, intestinal and eye infections.
Adeno-associated viruses. These are a class of single-stranded DNA viruses that can insert genetic material on chromosome 19.
Herpes simple viruses. These are a class of double-stranded DNA viruses that infect neurons.