Haemonetics V50 Pheresis System

Description (Brief):

A blood cell separator, or apheresis machine, is a piece of medical equipment used to separate out one component of the blood while returning the rest to the patient’s body. Doctors used this particular machine to remove white blood cells from patient Ashanti DeSilva for the first human gene therapy treatment approved by the National Institutes of Health (NIH). Gene therapy refers to a kind of genetic engineering whereby sections of DNA (genes) are introduced into cells in order to treat disease.

Description (Brief)

Ashanti, four years old at the time of her treatment in September 1990, suffered from a genetic disorder known as ADA deficiency (also known as ADA-SCID or “bubble boy” syndrome), which led to her having a compromised immune system which left her extremely vulnerable to infection. Though she was receiving medicine to treat her disease, a team of doctors at NIH (Dr. W. French Anderson, Dr. Michael Blaese, and Dr. Kenneth Culver) thought they might be able to alleviate her condition by “fixing” some of her cells with new genes.

Description (Brief)

To do this, they used the apheresis machine to remove some of her white blood cells. Using a modified mouse virus, the doctors then introduced working copies of the ADA gene into the removed white blood cells. The modified cells were grown for a time in the lab and were then injected back into Ashanti’s blood. Due to ethical concerns, however, doctors continued to treat Ashanti with her previous medication, meaning it was not possible to tell if the gene therapy worked or if she was simply staying healthy due to the medicine. While the treatment did not “cure” her, doctors were able to reduce the amounts of medication they gave her with no resulting decline in her health, suggesting that the new genes were indeed functioning in her body.

Description (Brief)

Sources:

Description (Brief)

Accession File

Description (Brief)

“Human Gene Therapy Debuts at NIH.” Thompson, Larry. Washington Post. 15 September 1990. p.A1.

Description (Brief)

“Gene Implant Therapy Is Backed For Children with Rare Disease.” Angier, Natalie. New York Times. 8 March 1990. p. A1.

Description (Brief)

“Success Stories.” Jaroff, Leon. Time. 11 January 1999.

Description (Brief)

“Scientists Report First Success of ‘Gene Therapy.’” Weiss, Rick. Washington Post. 20 October 1995. p. A1.

Description (Brief)

“Hope for Gene Therapy.” PBS. http://www.pbs.org/saf/1202/features/genetherapy.htm

Description (Brief)

“T Lymphocyte-Directed Gene Therapy for ADA SCID: Intitial trial Results After 4 Years.” R. Michael Blaese et al. Science. Vol. 270, No. 5235. 20 October 1995. p.475.

Date Made: ca 1980

Maker: Haemonetics Corporation

Location: Currently not on view

See more items in: Medicine and Science: Biological Sciences, Health & Medicine, Biotechnology and Genetics, Science & Mathematics

Exhibition:

Exhibition Location:

Credit Line: Gift of Haemonetics Corporation through Bill Cook

Data Source: National Museum of American History

Id Number: 1999.0008.01Accession Number: 1999.0008Catalog Number: 1999.0008.01

Object Name: blood cell separator

Physical Description: metal (overall material)plastic (overall material)Measurements: overall: 50 in x 16 1/4 in x 30 in; 127 cm x 41.275 cm x 76.2 cm

Guid: http://n2t.net/ark:/65665/ng49ca746a1-afa3-704b-e053-15f76fa0b4fa

Record Id: nmah_553290

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