Gene Therapy

By Austin Champion

What is Gene Therapy?

It is when you transplant normal genes into cells in place of defective or missing genes in order to correct the disorders within the cells to cure whatever is happening.
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The History of Gene Therapy

The first attempt to alter human genes was performed by Martin Cline in 1980. However, the first successful and approved nuclear gene transfer in humans was performed in May of 1989. The first ever therapeutic use of transferring genes as well as the first human dissection of human DNA into a nuclear genome was did by French Anderson in a trial in September of 1990. Between September of 1990 and January 2014, 2,000 clinical trials have been conducted and also approved. Noted, not all medical procedures that introduce some alterations to the patient's genetic makeup are considered gene therapy. Bone marrow transplant, and organ transplants have been found to introduce foreign DNA to the patients.

The Techniques and Development of Gene Therapy

In 1983, scientists at Baylor College of Medicine, Houston, Texas, proposed that one day gene therapy would be a viable approach for treating diseases like Lesch-Nyhan disease, which is a rare type of neurological disorder. W. French Anderson and colleagues first performed gene therapy, at National Institutes of Health (NIH) Clinical Center in Bethesda, Maryland, took the white blood cells from a 4-year old girl, Ashanti DeSilva, hoping to experiment and be able to cure SCID (Severe Combined Immune Deficiency), a rare genetic disease when the body cannot produce enough ADA (adenosine deaminase). They inserted ADA producing genes into the cells, and then put them back in the patient on September 14, 1990. The girl showed a continuous increased ability to produce ADA. Before this scientists across the United States performed these actions on groups of defective cells, however, this trial was the first to succeed.

Fun fact:

Gene therapy besides curing numerous diseases can sometimes also be used to cure cancer!

Part 2-Biography

Summary of William French Anderson

He was born December 31, 1936 and was an American physician, geneticist, and molecular biologist. Graduate of Harvard College, and was the first to succeed in gene therapy of a 4-year old girl, suffering from SCID.

Biography of William French Anderson

Anderson was born on December 31,1936, in Tulsa, Oklahoma. His father was a civil engineer, and his mother a journalist, writer, and an university professor. As a child, he stuttered. Eventually he overcame his impediment and has been recognized for his great performance in track, debate, and theater. He graduated in 1954 of Tulsa Central High School. Afterwards, he entered Harvard University. There he became a track star and published several papers, with one outlining a method of mathematical operations using Maya numerals. His school teacher had written a letter to the Harvard classics department where Professor Sterling Dow made arrangements that Will would be admitted to Harvard and a freshman's paper published in Classical Philology. Following a year away in Cambridge, where he met Kathy Duncan, his future wife, did he return to Harvard, to the Medical School, attended by Kathy. They later married in 1961 and both graduated a few years later. Employed at National Institutes of Health, he began his search to find ways to fix defective genes. Using different microinjection methods, his approach was slow and inefficient. Finally abandoning his approach, his work to the field was non-existent until 1984 when Richard C. Mulligan of MIT published a method of inserting genes by using a retrovirus. Dr. Anderson wanted to test his theory with a disease. In 1988 his proposal on this idea to the Human Gene Therapy Subcommittee was denied. However, his request of a hearing before the entire committee proceeded, and his trial was approved. May of 1989, Anderson conducted the first human safety test for gene therapy, where they injected a harmless marker into a 53-year-old man. A year later, a therapeutic trial was begun when Anderson took the white blood cells of 4-year-old Ashanti DeSilva to fix a disease she suffered from called SCID cause her body couldn't produce enough ADA or adenosine deaminase. After time, they later put the new ADA-producing genes into Ashanti and by 2007 she was stable, at the age of 21, but still needed to take medication regularly. This scientific consensus on gene therapy trial was mixed, but no doubt did it have a great impact on the new emerging field. He joined the University of Southern California faculty and henceforth was the director of the Gene Therapy Laboratories at the Keck School of Medicine and also was a professor of biochemistry and pediatrics. Later, Anderson was arrested on July 30, 2004 for being suspected of sexual abuse of a minor. He was convicted and jailed on 3 accounts of lewd acts upon a minor and 1 being a continuous abuse. On Febuary 2, 2007 he was sentenced to 14 years in prison and was ordered to pay $68,000 in fines, fees, and restitution; he faced a maximum of 18 years for molesting a now 19-year-old girl. Following the conviction, Anderson was stripped of tenure, fired, and barred from the campus.

Fun fact:

William French Anderson was in his 50's when he figured out gene therapy!