Gene Therapy: Bursting the Bubble on SCIDs

Glossary

Antibody
A soluble protein molecule produced and secreted by B cells in response to an antigen, which is capable of binding to that specific antigen.

Antigen
Any substance that, when introduced into that body, is recognized by the immune system.

B cells
Small white blood cells crucial to the immune defenses. Also known as B lymphocytes, they are derived from bone marrow and develop into plasma cells that are the source of anitobodies.

Bone marrow
Soft tissue located in the cavities of the bones. The bone marrow is the source of all blood cells.

Cellular response
The immune response of the body represented by the T cells of the immune system.

Cytokines
Powerful chemical substances secreted by cells. Cytokines include lymphokines produced by lymphocytes and monokines produced by monocytes and macrophages.

Cytotoxic T cells
A subset of T lymphocytes that can kill body cells infected by viruses or transformed by cancer.

Ex vivo
A gene therapy technique in which the DNA of a person is removed, altered in a laboratory test-tube, and then returned to an individual.

Granulocytes
White blood cells filled with granules containing potent chemicals that allow the cells to digest microorganisms, or to produce inflammatory reactions. Neutrophils, eosinophils, and basophils are examples of granulocytes.

Helper T cells
A subset of T cells that typically carry the T4 marker and are essential for activating antibody production, cytotoxic T cells, and initiating many other immune responses.

Humoral immunity
Immune protection provided by soluble factors wuch as antibodies, which circulate in the body's fluids or "humors," primarily serum and lymph.

Immune response
The reactions of the immune system to foreign substances.

Immunoglobulins
A family of large protein molecules, also known as antibodies.

Immunosuppression
Reduction of the immune responses, for instance by giving drugs to prevent transplant rejection.

Interleukins
A major group of lymphokines and monokines.

Lymphocytes
Small white blood cells produced in the lymphoid organs and paramount in the immune defenses.

Monocyte
A large phagocytic white blood cell which, when it enters tissue, develops into a macrophage.

Natural killer (NK) cells
Large granule-filled lymphocytes that take on tumor cells and infected body cells. They known as "natural" killers because they attack without first having to recognize specific antigens.

PCR
Polymerase Chain Reaction. A technique for the rapid production of millions of copies of a particular stretch of DNA.

Severe combined immunodeficiency disease (SCID)
A life-threatening condition in which infants are born lacking all major immune defenses.

T cells
Small white blood cells that orchestrate and/or directly participate in the immune defenses. Also known as T lyphocytes, they are processed in the thymus and secrete lymphokines.

Bursting the Bubble on Severe Combined Immunodeficiency Syndrome (SCID)

Please click here to read the actual research paper by Drs. Cavazzana-Calvo, et. al.

Imagine living your entire life contained within a plastic bubble. Contact from the outside world, including your parents, is lethal. Rather than feeling the warm touch of a human hand, the clammy cold of laboratory gloves comforts you to sleep. Is this living or this surviving? You make the call.

SCIDs is an acronym for Severe Combined Immunodeficiency Syndrome. Persons born with SCIDs lack the ability to fight off infections. SCIDs creates a situation in which the common cold is just as deadly as pneumonia.

This family of diseases is obviously debilitating and life-threatening. That is why finding a cure is imperative. This disease is not contagious, it is genetic and is thus acquired through the simple role of the genetic dice.

There is absolutely nothing we can do about this sad syndrome, or is there...

We can look to gene therapy: an exciting and revolutionary new field of research and medicine which may reveal the key to unlocking a myriad of genetic diseases.

This web site will explore the problems posed by SCIDs and the answer offered by Marina Cavazzana-Calvo and Salima Hacein-Bey. Their work in gene therapy has great potential towards bursting the bubble on SCIDs.

Our thanks to the wonderful people of Corbis.com for this picture!

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