Bone marrow transplant strategies for the correction of sickle cell disease Flashcards
Immunosuppresion is not necessary in which type of transplantation?
Autologous gene therapy
Which current therapies are used to treat sickle cell anemia?
L-glutamine; hydroxyurea
Donor red cells persist and donor chimerism stable after withdrawal of immunosuppression with ___________
sirolimus
Single substitution at position ________ of _________ chain causes sickle cell anemia.
6; Beta-globin
_______________launched in 2018 to accelerate progress.
NHLBI Cure Sickle Cell Initiative
What has been used to treat transfusion-dependent Beta thalassemia?
Lentiglobin
First disease for which molecular defect identified
Sickle Cell Disease
Abnormal Hb ________ upon ___________ results in severe anemia, frequent pain, and organ damage.
polymerization; deoxygenation
•Allogeneic transplantation can cure ___________ of children and adults with SCD who have a matching sibling donor
>90%
What are the differences between allogeneic transplantation and autologous gene therapy?
In allogeneic transplantation, bone marrow is transplanted from someone who does not have SCD. The donor is usually a HLA-matched sibling but can include cord blood or half-matched family member.
In autologous gene therapy, bone marrow transplant is from patients own bone marrow. Beta-globin gene is transferred via an engineered virus or gene is edited with an engineered endonuclease
Mean age of death for sickle cell patients, in 2006, was_______
36
Is Myeloablation necessary?
No. Stable mixture of bone marrow is sufficient
Bone marrow transplant replaces the defective ________
RBCs
What is currently the only curative option to sickle cell anemia?
Allogenic bone marrow stem cell transplantation
