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Stem Cell Project

The Promise for
Severe Combined Immunodeficiency

Video length: 16:34

•  Importance of the Project
•  Principal Investigators
•  Project Components
•  Biology & Gene Therapy
•  Facilities & Technologies
•  Glossary
•  Additional Resources

The Childrens Hospital Los Angeles Stem Cell Project represents the combined work of some of the finest physician-scientists and researchers in the world. Here our researchers investigate stem cells that they have manipulated in order to direct the cells' development. By understanding the mechanisms that cause stem cells to act in predictable ways, we hope to harness the potential of these cells to treat human diseases. 

There are more than thirty research groups within The Saban Research Institute of Childrens Hospital Los Angeles.  Each group contributes a wide range of expertise in human embryonic and adult stem cells, as well as gene therapy, developmental biology, tissue engineering and clinical transplantation. 

By increasing our understanding of stem cell biology we move closer to our goal: the creation of effective therapies for life-threatening diseases.  Our research in this field has already yielded results.  A young patient born with a severe combined immune deficiency known as "Bubble Baby Disease" was cured after Childrens Hospital Los Angeles was able to effectively treat her by transplanting stem cells collected from blood in the umbilical cord. This is the beginnings of what our research could achieve.

The Childrens Hospital Los Angeles Stem Cell Project is uniquely positioned in California to apply embryonic and adult stem cell technologies as therapies .  The project goals align with the purpose of Proposition 71, the California Stem Cell Research and Cures Initiative, approved by California voters on November 2, 2004.

For the past three years, scientists at The Saban Research Institute have worked with human embryonic stem cells (hESC) approved by the National Institutes of Health (NIH).  These studies have focused on developing methods to culture and genetically manipulate hESC and to direct their differentiation to blood and pancreas cells.

New initiatives to generate liver and lung cells from hESC are underway. Investigators at The Saban Research Institute also are studying adult stem cells from bone marrow, cord blood, lung, liver and mesenchymal tissue (cells that develop into connective tissue, blood vessels and lymphatic tissue). Other research is exploring amniotic fluid stem cells, which may have the potential to form many tissues of the body.

The Childrens Hospital Los Angeles Stem Cell Project
Childrens Hospital Los Angeles
4650 Sunset Blvd., MS #62
Los Angeles, CA  90027
(323) 361-5690