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Lecture 16

MCD BIO 90 Lecture 16: MCDB90-8-2


Department
Molecular, Cell, and Developmental Biology
Course Code
MCD BIO 90
Professor
Mitra Hooshmand
Lecture
16

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Wednesday, November 20, 2019
MCDB 90
Lecture 8-2!
-ADA-SCID with Donald Kohn!
Viral vectors!
ADA is a specific gene that is missing in the SCID kids!
-Which causes the abnormality in T cells!
Process!
-Extract the HSCs!
-Genetically insert the ADA gene!
-Inject the cell back to the kids!
-Repopulate the bone marrow!
Sickle cell is also a single locus mutation of the gene!
Beta/Alpha thalassemia!
-Alpha is fetal while in uterus, babies cannot be born!
-Orphan diseases!
-Treat the baby in uterus with stem cell!
-First baby born !
-Treatment!
Mother’s cell!
High dose (get rid of the sick cells)!
Transplant to fetus’s bloodstream (umbilical vein)!
Take on mother’s healthy HSCs!
Spinal Cord Injuries!
-Human ESC-derived oligo progenitors!
-Transplant within a period of 20-40 days post injury!
-Thoracic injury with 7-10 days post injury!
-Going into the phase III!
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