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11 Cards in this Set
- Front
- Back
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What does "Combined" mean as it relates to SCID
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Both humoral and cell-mediated immunities are involved
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A patient with SCID will present in what way
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Increased susceptibility to infections
Onset usually by 2 months old Especially seen in skin, respiratory, and GI systems Untreated = Death |
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Describe X-linked SCID
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No T-cells or NK cells
B cells present, but immature |
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Describe adenosine deaminase SCID and most autsomal recessive SCIDs
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B and T cells both absent
NK cells present |
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Why does a deficiency in adenosine deaminase result in SCID
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ADA removes/recycles adenosine released during cell death/recycling
ADA deficiency = increased dATP = inhibition of ribonucleotide reductase = lack of other dNTPs = arrest of DNA synthesis = messed up/decreased lymphoid cells |
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What are some treatment options for ADA deficiency SCIDs
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Hematopoietic stem cell replacement - HST/BMT
Enzyme replacement - PEG/ADA Gene therapy |
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Why is naked DNA not terribly useful in gene therapy
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Is only taken up properly into very few cells, not enough to be effective therapeutically
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What ?vector? appears to have the greatest efficacy in gene therapy
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Retroviruses
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Describe T-cell directed gene therapy
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T-cells isolated from patient, grown in culture with IL-2
ADA gene introduced by retroviral vector T-cells expressing ADA selected, grown, and reintroduced into patient |
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What cell surface protein is used to identify hematopoetic stem cells
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CD34
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Describe HST gene therapy
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Bone marrow sample taken, CD34+ cells (hematopoetic stem cells) isolated
CD34+ cells given gene therap, patient undergoes cytoreduction Reinfuse cells |
