scholarly journals Curative options for sickle cell disease: haploidentical stem cell transplantation or gene therapy?

2020 ◽  
Vol 189 (3) ◽  
pp. 408-423 ◽  
Author(s):  
Alexis Leonard ◽  
John Tisdale ◽  
Allistair Abraham
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Sickle Cell Disease ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
Cell Disease ◽  
Haploidentical Stem Cell Transplantation ◽  
Or Gene

Hematopoietic stem cell transplantation and gene therapy are the sole treatments for sickle cell disease and other hemoglobinopathies

2021 ◽  
Author(s):  
Moataz Dowaidar
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Sickle Cell Disease ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
The United States ◽  
Cell Disease ◽  
Hematopoietic Stem ◽  
Hbf Induction

Linus Pauling and colleagues originally discovered sickle cell disease (SCD) as a molecular genetic abnormality in 1949, and after more than five decades of study, the FDA authorized hydroxyurea as the first HbF-inducing drug for the treatment of VOC in SCD patients in 1998. L-glutamine, crizanlizumab, and voxelotor were authorized by the FDA for the treatment of SCD in adults 20 years ago. There are several FDA-approved medications for treating SCDs in the United States, but the HU is the only EMA-approved medication in Europe.Hematopoietic stem cell transplantation (HSCT) and gene therapy are the sole treatments for SCD and other hemoglobinopathies. Furthermore, miRNAs contribute to the development of novel therapeutics by delineating the molecular mechanisms and signaling networks involved in HbF induction. The transcription factors BCL11A, MYB, KLF-3, and SP1 regulate miRNAs, which have a variety of consequences on target expression. HbF induction is now an important aspect of minimizing hospitalizations and improving survival. A comprehensive perspective on miRNA regulation mechanisms and comprehensive study on miRNAs as a possible SCD treatment are relevant, based on the rising number of detailed miRNA functional investigations. As a biomarker, it might be used to quantify VOCs and discriminate between acute and chronic pain. This is the first study we've seen that suggests miRNAs may be used to treat SCD.

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

scholarly journals How I treat sickle cell disease with hematopoietic cell transplantation

Blood ◽  
2019 ◽  
Vol 134 (25) ◽  
pp. 2249-2260 ◽  
Author(s):  
Elizabeth O. Stenger ◽  
Shalini Shenoy ◽  
Lakshmanan Krishnamurti
Keyword(s):  
Decision Making ◽  
Stem Cell ◽  
Sickle Cell Disease ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
Hematopoietic Cell Transplantation ◽  
Cell Disease ◽  
Hematopoietic Stem

Hematopoietic stem cell transplantation can be curative for sickle cell disease, but decision-making is often complex. This How I Treat provides a perspective on how to negotiate this process for an individual patient.

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