Gene/Protein Disease Symptom Drug Enzyme Compound
Pivot Concepts:   Target Concepts:
Query: UMLS:C0019045 (hemoglobinopathies)
 2,704 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

Patient-derived induced pluripotent stem cells as well as human embryonic stem cells are pluripotent and their derivation has been used for the understanding of numerous diseases. Currently they are also used for the treatment of neurologic disorders such as Parkinson disease or cardiac disorders. Gene therapy has been successful for the treatment of hemophilia A and B, hemoglobinopathies and immunodeficiencies. Hemopoietic stem cell transplantation is a well-accepted therapeutic strategy for Leukemias, whereas CAR-T cells is a new promising approach even for lymphomas and myeloma.
 ...
PMID:[Human cells for therapeutics purpose: State of the art]. 3283 90