scholarly journals Allogeneic cellular and autologous stem cell therapy for sickle cell disease: ‘whom, when and how’

2011 ◽  
Vol 47 (12) ◽  
pp. 1489-1498 ◽  
Author(s):  
J Freed ◽  
J Talano ◽  
T Small ◽  
A Ricci ◽  
M S Cairo
Keyword(s):  
Stem Cell ◽  
Sickle Cell Disease ◽  
Cell Therapy ◽  
Sickle Cell ◽  
Stem Cell Therapy ◽  
Cell Disease

scholarly journals Inhibiting in function of Gene in sickle Cell Disease

2018 ◽  
Vol 1 (01) ◽  
Author(s):  
Ashay Harish Nandeshwar
Keyword(s):  
Stem Cell ◽  
Sickle Cell Disease ◽  
Cell Therapy ◽  
Sickle Cell ◽  
Stem Cell Therapy ◽  
Genetic Diseases ◽  
Marrow Transplant ◽  
Genome Project ◽  
Base Substitution ◽  
Cell Disease

I was much struck with the fact that Sickle-Cell Disease and most of the genetic diseases are incurable. The knowledge I gained about base substitution mutation in DNA and the Human Genome Project, showed me light to the possibility of a cure to the disease. Stem Cell Therapy was the first thing that came into my mind, while thinking of a cure to SCD. Eventually I found that researches about using stem cell therapy for curing SCD were already being carried in various academic and research institutions across the globe. As a result, bone marrow transplant became a ray of hope for the sufferers of the disorder; but still, the scientists and doctors would assert the non-existence of a definite cure to the disease

scholarly journals RH genotyping in a sickle cell disease patient contributing to hematopoietic stem cell transplantation donor selection and management

Blood ◽  
2010 ◽  
Vol 116 (15) ◽  
pp. 2836-2838 ◽  
Author(s):  
Ross M. Fasano ◽  
Alessandro Monaco ◽  
Emily Riehm Meier ◽  
Philippe Pary ◽  
A. Hallie Lee-Stroka ◽  
...  
Keyword(s):  
Stem Cell ◽  
Sickle Cell Disease ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Blood Group ◽  
Sickle Cell ◽  
Disease Patient ◽  
Nucleotide Sequencing ◽  
Cell Disease ◽  
Hematopoietic Stem

Abstract African individuals harbor molecular RH variants, which permit alloantibody formation to high-prevalence Rh antigens after transfusions. Genotyping identifies such RH variants, which are often missed by serologic blood group typing. Comprehensive molecular blood group analysis using 3 genotyping platforms, nucleotide sequencing, and serologic evaluation was performed on a 7-year-old African male with sickle cell disease who developed an “e-like” antibody shortly after initiating monthly red blood cell (RBC) transfusions for silent stroke. Genotyping of the RH variant predicted a severe shortage of compatible RBCs for long-term transfusion support, which contributed to the decision for hematopoetic stem cell transplantation. RH genotyping confirmed the RH variant in the human leukocyte antigen–matched sibling donor. The patient's (C)ces type 1 haplotype occurs in up to 11% of African American sickle cell disease patients; however, haplotype-matched RBCs were serologically incompatible. This case documents that blood unit selection should be based on genotype rather than one matching haplotype.

Demands and challenges for patients with sickle-cell disease requiring hematopoietic stem cell transplantation in Saudi Arabia

2016 ◽  
Vol 20 (6) ◽  
pp. 831-835 ◽  
Author(s):  
Abdulrahman Alsultan ◽  
Wasil Jastaniah ◽  
Sameera Al Afghani ◽  
Muneer H. Al Bagshi ◽  
Zaki Nasserullah ◽  
...  
Keyword(s):  
Saudi Arabia ◽  
Stem Cell ◽  
Sickle Cell Disease ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Sickle Cell ◽  
Cell Disease ◽  
Hematopoietic Stem

Sickle Cell Disease Hematopoietic Stem Cell gene therapy with globin gene addition is promising

2021 ◽  
Author(s):  
Moataz Dowaidar
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Sickle Cell Disease ◽  
Hematopoietic Stem Cell ◽  
Sickle Cell ◽  
Globin Gene ◽  
Autologous Transplantation ◽  
Gene Repair ◽  
Cell Disease ◽  
Hematopoietic Stem

Autologous transplantation of gene-modified HSCs might be used to treat Sickle Cell Disease (SCD) once and for all. Hematopoietic Stem Cell (HSC) gene therapy with lentiviral-globin gene addition was optimized by HSC collection, vector constructs, lentiviral transduction, and conditioning in the current gene therapy experiment for SCD, resulting in higher gene marking and phenotypic correction. Further advancements over the next decade should allow for a widely approved gene-addition therapy. Long-term engraftment is crucial for gene-corrected CD34+ HSCs, which might be addressed in the coming years, and gene repair of the SCD mutation in the-globin gene can be achieved in vitro using genome editing in CD34+ cells. Because of breakthroughs in efficacy, safety, and delivery strategies, in vivo gene addition and gene correction in BM HSCs is advancing. Overall, further research is needed, but HSC-targeted gene addition/gene editing therapy is a promising SCD therapy with curative potential that might be widely available soon.

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