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- NCIT_C113438 IAO_0000115 "A preparation of autologous, CD34-positive stem/progenitor cells transduced with a lentrivral vector encoding the human adenosine deaminase (ADA) gene under the control of the human elongation factor alpha short promoter (EFS), with potential to restore ADA expression and function. Autologous hematopoietic CD34+ cells are isolated from the patient's own bone marrow, peripheral blood or cord blood, and transduced with the EFS-ADA lentiviral vector ex vivo. Upon re-infusion of the EFS-ADA vector-transduced lymphocytes back into the patient, these cells may both restore ADA activity and prevent severe combined immunodeficiency (SCID) due to ADA deficiency. ADA, an enzyme that catalyzes the deamination of adenosine to inosine, plays a key role in the development and functioning of the immune system." @default.
- NCIT_C113438 NCIT_NHC0 "C113438" @default.
- NCIT_C113438 NCIT_P106 "Cell" @default.
- NCIT_C113438 NCIT_P106 "Pharmacologic Substance" @default.
- NCIT_C113438 NCIT_P108 "EFS-ADA Lentiviral Vector-transduced CD34-positive Autologous Lymphocytes" @default.
- NCIT_C113438 NCIT_P208 "CL458232" @default.
- NCIT_C113438 NCIT_P329 "756296" @default.
- NCIT_C113438 NCIT_P330 "756296" @default.
- NCIT_C113438 NCIT_P399 "756296" @default.
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- NCIT_C113438 normalizedInformationContent "100" @default.
- NCIT_C113438 referenceCount "1" @default.
- NCIT_C113438 hasExactSynonym "EFS-ADA Lentiviral Vector-transduced CD34-positive Autologous Lymphocytes" @default.
- NCIT_C113438 inSubset NCIT_C176424 @default.
- NCIT_C113438 type Class @default.
- NCIT_C113438 isDefinedBy ncit.owl @default.
- NCIT_C113438 label "EFS-ADA Lentiviral Vector-transduced CD34-positive Autologous Lymphocytes" @default.
- NCIT_C113438 subClassOf NCIT_C113438 @default.
- NCIT_C113438 subClassOf NCIT_C142078 @default.
- NCIT_C113438 subClassOf NCIT_C154231 @default.
- NCIT_C113438 subClassOf NCIT_C1908 @default.
- NCIT_C113438 subClassOf NCIT_C1909 @default.
- NCIT_C113438 subClassOf NCIT_C1962 @default.
- NCIT_C113438 subClassOf NCIT_C28681 @default.
- NCIT_C113438 subClassOf NCIT_C307 @default.
- NCIT_C113438 subClassOf NCIT_C308 @default.