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Lecture 4

BIMS 201 Lecture Notes - Lecture 4: Sickle-Cell Disease, Hydroxycarbamide, Fetal Hemoglobin


Department
Biomedical Science
Course Code
BIMS 201
Professor
Elizabeth A.Crouch
Lecture
4

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4/19/17
Sickle Cell Discussion
General Questions
Lentivirus=retrovirus known to cause disease
o Long incubation period (ex: HIV, FIV)
Hydroxyurea: oral chemotherapy given to patients for sickle-cell anemia
o Adverse effects: vomiting, hair loss, mouth lesions
Sickle cell incidence in African American populations: 1/230 live births
o Sickle cell disease: 1/1146
Gene therapy (lentivirus)
Use as vector to carry gene and insert it (delivery method)
Considerations:
o must infect correct cells (NOT RBCs bc no DNA/nucleus)
o target bone marrow (precursors to RBC)
o making sure to not infect w/retrovirus
o oitor that etor auses reoal of ad gee that’s affeted NOT faily
member gene
treatment is working: timing=critical (7-year success)
o how long b/w treatments?
Prefer to hydroxyurea treatment bc:
o Side effects and daily pill that can be absorbed thru skin
Not long-lasting
o gene therapy=long-lastig ad < dotor’s isits
a lot > hospitalization @ the beginning
Hydroxyurea as treatment
stimulates production of fetal heogloi: INC gaa (does’t ause siklig i lo O2
conditions) = prevent symptoms of disease
acts in pathway in production in change of cytosine to deoxycytosine
acts in pathway and prevents incorporation of thymine in DNA strand during DNA
replication
want to prevent production of beta hemoglobin (feedback mechanism)
cost:
o hospital per year $1.8M
o treated patient: $11,072 vs. non-treated patient: $13,962
o patient saves $2,000/yr. and hospital saves $700,000/yr.
o treating disease and using preventative measures is always cheaper than having
a sick person in a hospital waiting room
HU over therapy:
o significant DEC in hospital admission
o incidence of chest syndrome and liver problems
o red. Blood transfusion requirements by as much as 50%
daily therapy is > accessible to > ppl than gene therapy
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