What is a stem cell transplant?
Hematopoietic stem cells are the precursors of all blood cells. They develop into red blood cells, which carry oxygen, white blood cells, which fight disease and infection, and platelets, which aid in blood clotting. Hematopoietic stem cell transplantation is the treatment of choice for NEMO.
Stem cells can be collected from peripheral blood, bone marrow or umbilical cord blood.
Transplantation of healthy stem cells from another individual can be used to help restore normal blood production in patients whose own ability to make any or all of these blood cells has been compromised by the NEMO mutation.

What is NEMO?
NEMO is the name of a gene that regulates functions of the immune system. NEMO stands for Nuclear Factor Kappa B Essential Modulator. It's like a "master switch" that is found on the cell of the human body and regulates signals between the cell membrane and the cell nucleus. It's expressed in almost all tissue cells. If NEMO is not present life is not possible.
Nuclear Factor Kappa B or NFKB is the name of the protein that is used for communication between the membrane and the nucleus. NEMO is known to be the pathway of skin, skeleton, blood vessel and other vital signals. For example, the EDA gene is known to dictate properties of the "ectoderm" or last layer of the skin. The EDA gene uses the NEMO pathway to the nucleus to make the "ectoderm" properties present.

What is ECTODERMAL DYSPLASIA?
The ectoderm is the outer layer of the skin, sweat glands, hair, teeth and nails. Ectodermal dysplasia (ED) is a heritable disorder that affects the ectoderm, the outer layer of tissue in a developing baby. ED syndromes affect both males and females of all races and ethnic groups. The ectoderm contributes to the formation of many parts of the body, including the skin, sweat glands, hair, teeth, and nails. During embryonic development, these and/or other parts of the baby’s body, including the lens of the eye, parts of the inner ear, the fingers and toes, or nerves, among others, may fail to develop normally.