Therapeutic/Technology: Gene-modified, personalized cell therapy
Gene Correction of Autologous Hematopoietic Stem Cells in Artemis Deficient SCID
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 Combined Immunodeficiency (SCID-A), a “bubble baby” syndrome associated with increased sensitivity to radiation and chemotherapy. SCID-A is hard to treat with a bone marrow stem […]
Stem Cell Therapy for Duchenne Muscular Dystrophy
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. Until recently, there was little hope that the widespread muscle degeneration that accompanies this disease could be combated. However, stem cell therapy now offers that […]
Development of RNA-based approaches to stem cell gene therapy for HIV
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 infection each year in the US with over 148,000 cumulative cases reported in California alone (as of 2009). Multi-drug therapy has been helpful in reducing […]
Curing Hematological Diseases
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 blood diseases where mutations in a single gene results in the disease. XSCID, more commonly known as the “bubble boy” disease, is characterized by a […]
Stem Cells for Immune System Regeneration to Fight Cancer
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 to protect us from cancer. The T cell receptor (or TCR), a complex protein on the surface of an immune cell (or lymphocyte), allows to […]
GENE-MODIFIED HEMATOPOIETIC STEM/PROGENITOR CELL BASED THERAPY FOR HIV DISEASE
Stem Cell Gene Therapy for Sickle Cell Disease
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 of 1/500 African-Americans, and is also common in Hispanic-Americans, who comprise up to 5% of SCD patients in California. The median survival based on 1991 […]
HPSC based therapy for HIV disease using RNAi to CCR5.
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 reproduction within the body. When RNA interference is introduced into a stem cell, its blocking activity will be present throughout the lifetime of the stem […]
iPS Cell-Based Treatment of Dominant Dystrophic Epidermolysis Bullosa
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 healing. Patients with one genetic disease, dystrophic Epidermolysis bullosa (EB), lack a normal collagen VII (COL7A1) gene and suffer from debilitating blistering and scarring that […]
ZINC FINGER NUCLEASE-BASED STEM CELL THERAPY FOR AIDS
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 to a cell. Building on this observation, a study published in 2009 reported a potential “cure” in an AIDS patient with leukemia after receiving a […]