Artemis is a chemical in all cells in the body that is essential for the normal development of the immune system and repairing damaged DNA. Artemis deficiency (AD) causes Severe…
Duchenne muscular dystrophy (DMD) is the most common and serious form of muscular dystrophy. One out of every 3500 boys is born with the disorder, and it is invariably fatal.…
Despite significant advances in treatment and prevention programs, HIV infection with progression to Acquired Immunodeficiency Syndrome (AIDS) is still prevalent in California. The CDC Estimates >56,000 new cases of HIV…
The primary aim of this project is to develop treatments for incurable diseases of the blood and immune system. X-linked Severe Combined Immunodeficiency (X-SCID) and Fanconi anemia (FA) are two…
This proposal will define the biology of stem cell engineering to produce a cancer-fighting immune system. The immune system protects our body against most outside threats. However, it frequently fails…
Sickle cell disease (SCD), which results from an inherited mutation in the hemoglobin gene that causes red blood cells to "sickle" under conditions of low oxygen, occurs with a frequency…
RNA interference is a naturally occurring means to block the function of genes in our body. We propose that RNA interference can be used to block HIV-1 infection and its…
Genetic skin diseases constitute a diverse group of several hundred diseases that affect up to 2% of the population and include common disease such as psoriasis, atopic dermatitis, and wound…
Some years ago it was discovered that patients homozygous for a natural mutation (the Δ32 mutation) in the CCR5 gene are generally resistant to HIV infection by blocking virus entry…
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