A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders
Ex-vivo gene therapy (GT) with hematopoietic stem and progenitor cells (HSPCs)
engineered with integrating vectors is a promising treatment for monogenic diseases, but …
engineered with integrating vectors is a promising treatment for monogenic diseases, but …
Sickle cell disease
Sickle cell disease (SCD) is a group of inherited disorders caused by mutations in HBB,
which encodes haemoglobin subunit β. The incidence is estimated to be between 300,000 …
which encodes haemoglobin subunit β. The incidence is estimated to be between 300,000 …
[HTML][HTML] CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia
Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are severe
monogenic diseases with severe and potentially life-threatening manifestations. BCL11A is …
monogenic diseases with severe and potentially life-threatening manifestations. BCL11A is …
[HTML][HTML] Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease
EB Esrick, LE Lehmann, A Biffi, M Achebe… - … England Journal of …, 2021 - Mass Medical Soc
Background Sickle cell disease is characterized by hemolytic anemia, pain, and progressive
organ damage. A high level of erythrocyte fetal hemoglobin (HbF) comprising α-and γ …
organ damage. A high level of erythrocyte fetal hemoglobin (HbF) comprising α-and γ …
Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease
Sickle cell disease (SCD) is the most common serious monogenic disease with 300,000
births annually worldwide. SCD is an autosomal recessive disease resulting from a single …
births annually worldwide. SCD is an autosomal recessive disease resulting from a single …
Exagamglogene Autotemcel for Severe Sickle Cell Disease
Background Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to
reactivate fetal hemoglobin synthesis by means of ex vivo clustered regularly interspaced …
reactivate fetal hemoglobin synthesis by means of ex vivo clustered regularly interspaced …
Recent advances in the treatment of sickle cell disease
G Salinas Cisneros, SL Thein - Frontiers in physiology, 2020 - frontiersin.org
Sickle cell anemia (SCA) was first described in the Western literature more than 100 years
ago. Elucidation of its molecular basis prompted numerous biochemical and genetic studies …
ago. Elucidation of its molecular basis prompted numerous biochemical and genetic studies …
Defining global strategies to improve outcomes in sickle cell disease: a Lancet Haematology Commission
Executive summary All over the world, people with sickle cell disease (an inherited
condition) have premature deaths and preventable severe chronic complications, which …
condition) have premature deaths and preventable severe chronic complications, which …
Effect of donor type and conditioning regimen intensity on allogeneic transplantation outcomes in patients with sickle cell disease: a retrospective multicentre, cohort …
M Eapen, R Brazauskas, MC Walters… - The Lancet …, 2019 - thelancet.com
Background Donors other than matched siblings and low-intensity conditioning regimens
are increasingly used in haematopoietic stem cell transplantation. We aimed to compare the …
are increasingly used in haematopoietic stem cell transplantation. We aimed to compare the …
Sickle cell disease
RE Ware, M de Montalembert, L Tshilolo, MR Abboud - The Lancet, 2017 - thelancet.com
Sickle cell disease is a common and life-threatening haematological disorder that affects
millions of people worldwide. Abnormal sickle-shaped erythrocytes disrupt blood flow in …
millions of people worldwide. Abnormal sickle-shaped erythrocytes disrupt blood flow in …