Gene Therapy In Children

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Since its inception, gene therapy has been considered to be the future's ultimate

cure for terminal diseases that have afflicted many people, and whose treatment still remains to be discovered. The concept behind such therapy is to replace a defective gene with a good gene and thus function normally.

A few of such diseases that can be treated with gene therapy are the ones which have genetic component including cystic fibrosis, hemophilia, AIDS, cancer and heart diseases. Admittedly, the application of stem cell involve many risks, hence, only children afflicted with incurable diseases are placed under the clinical trials of gene therapy.

The genes, which are inherited from parents, are significant in determining the physical traits and characteristics of individuals, the sex of individuals, as well as the IQ and the amount of oxygen the individual's blood carries.

Found on the genes in a single file within the chromosomes are strands of a molecule known as the DNA. The genetic information is encoded by the nucleotides, DNA's building blocks. In a human cell are 3 billion pairs of nucleotides which are a unique sequence that composes the person's genetic makeup. The person's genetic makeup is what makes him apart from the rest.
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Each human has 25,000 genes for every cell. A disease or physical disability or curtailed life span is a result of a mutation or alteration in any of the genes in the human body. The mutations can be inherited from parents to their offspring or can happen spontaneously outside heredity. Gene therapy seeks to treat or eliminate the genetic diseases or physical conditions because of the said mutations. To accomplish such objective, genes are manipulated by introducing a 'good' gene into a patient who suffers from an illness triggered by a 'bad' gene.

Gene therapy comes in two types, the somatic and the germline. In somatic, the 'good' gene that is introduced into the patient's body cannot affect the genes of the future offspring. Which means that the disease is unlikely to be passed along onto the patient's children because the genes are not inherited by them.

In germline approach, however, the modification of the faulty genes is done in egg or sperm cell. Which means that any modification in the gene can be passed on to the offspring. Although experiments done on mice prove to be promising, very little attention is given to germline gene therapy because of the controversy arising from the entirely new genes for the future children.

The most active research being initiated in gene therapy for children is for genetic disorders such as cystic fibrosis. Severe immunodeficiency diseases like the adenosine deaminase deficiency and ones with familial hypercholesterolemia are being put in gene therapy trials.

The trials in children pose some ethical challenges as presented by the experts in the field. Children who have modified gene may have mild to severe reaction and the severity cannot be determined in infants. Children could be tested for illnesses to check that they are not carrier of defective gene, and thus, do not have to undergo annual screenings.

by: Charles Godbout

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