How Much Money Does A Speech-Language Pathologist Make In Canada History of Gene Therapy

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History of Gene Therapy

Gene therapy is an experimental medical procedure that attempts to correct a genetic mutation (missing or changed genes) so that properly functioning genes are returned to the cells. When gene therapy works, the correct instructions for building proteins (chemicals that direct and control chemical reactions in the body) are again available to the cells, and the body returns to normal or healthy function.

Scientists began to discuss the possibility of this method to cure diseases in the 1960s. In 1970, the American doctor Stanfield Rogers at the Oak Ridge National Laboratory in Tennessee tried to use gene therapy to treat two sisters who they had a genetic disorder called argininemia. With this genetic disorder, the body lacks an enzyme (a type of protein) called arginase. People with this disorder can have seizures and mental disorders. Rogers tried to treat the sisters by using a virus to bring the whole gene into their cells. In this case, gene therapy is not successful.

In 1977, scientists were able to use gene therapy techniques to deliver a gene into mammalian cells. American doctor W. French Anderson conducted one of the first studies of gene therapy in humans in 1990 on a four-year-old girl who had a rare genetic immune system disorder called severe combined immunodeficiency (SCID). The immune system fights infections from bacteria and viruses, and the disorder has made it difficult for his body to stay healthy. Anderson and his team genetically altered his white blood cells and then returned them to his body. The new white blood cells strengthened the girl’s immune system and allowed her to survive.

Another encounter with gene therapy occurred in 1999. An eighteen-year-old patient named Jesse Gelsinger was involved in a gene therapy trial for a genetic disease called ornithine transcarboxylase deficiency (OTCD). This rare disease prevents the liver from breaking down ammonia, which can accumulate in the body and become toxic. Gelsinger died of organ failure four days after starting treatment. Researchers believe that his immune system reacted to the virus that carried the new gene into his cells.

In 2000, the French researcher Alain Fischer was able to cure children of a similar type of disorder of the immune system. Fischer used retroviruses as gene carriers. Retroviruses are a type of virus that uses ribonucleic acid (RNA) as its genetic material, instead of DNA. Retroviruses produce an enzyme (a protein that controls a biochemical reaction) that builds DNA onto a strand of RNA. The best known of these retroviruses is the human immunodeficiency virus (HIV), the virus responsible for acquired immunodeficiency syndrome (AIDS). Fischer inserted a retrovirus that carries the normal gene into the children’s blood stem cells. Several months later, two of the children in the trial developed a disease similar to leukemia (a type of cancer that begins in the cells that make blood cells). As a result, the US Food and Drug Administration (FDA) stopped the use of retroviruses in the US.

Although research in this field has progressed slowly, it is still advancing. In 2003, the first officially licensed gene therapy was available in China. Several types of gene therapy are awaiting approval from the US Food and Drug Administration.

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