CRISPR/Cas9-based gene-editing technology for sickle cell disease

L Ma, S Yang, Q Peng, J Zhang, J Zhang - Gene, 2023 - Elsevier
Sickle cell disease (SCD) is the most common monogenic hematologic disorder and is
essentially congenital hemolytic anemia caused by an inherited point mutation in the β …

Hope on the Horizon: New and Future Therapies for sickle cell disease

FA Kuriri - Journal of Clinical Medicine, 2023 - mdpi.com
This article provides an overview of conventional, new, and future treatment options for
sickle cell disease (SCD), a genetic disorder affecting the production of hemoglobin. Current …

Voxelotor for the treatment of sickle cell disease in pediatric patients

C Brown, M Tonda, MR Abboud - Expert Review of Hematology, 2022 - Taylor & Francis
Introduction Sickle cell disease (SCD) describes a group of heritable blood disorders
caused by the polymerization of sickle hemoglobin (HbS). HbS polymerization leads to …

Design, Synthesis, and Evaluation of Allosteric Effectors for Hemoglobin

NA Enakaya, A Jefferson… - Accounts of Chemical …, 2023 - ACS Publications
Conspectus Sickle cell disease (SCD) is an inherited blood disorder caused by a point
mutation in hemoglobin (Hb), the protein in the red blood cell (RBC) responsible for the …

Recent progress in the treatment of sickle cell disease: an up-to-date review

B Acharya, DP Mishra, B Barik, RK Mohapatra… - Beni-Suef University …, 2023 - Springer
Background Sickle cell disease is a fatal systemic condition characterized by acute painful
episodes, persistent anemia, ongoing organ damage, organ infarction, and a markedly …

Controversies in the pathophysiology of leg ulcers in sickle cell disease

J Catella, N Guillot, E Nader, S Skinner… - British Journal of …, 2024 - Wiley Online Library
Patients with sickle cell disease (SCD) often experience painful vaso‐occlusive crises and
chronic haemolytic anaemia, as well as various acute and chronic complications, such as …

Concise approach for the synthesis of Voxelotor for sickle cell disease

J Manda, BVS Reddy - Tetrahedron, 2024 - Elsevier
A novel approach has been developed for the synthesis of Voxelotor, which is used for the
treatment of Sickle Cell Disease (SCD). The key steps involved in this approach are the ring …

Increased oxygen affinity: to have and to hold

CT Quinn, RE Ware - Blood, The Journal of the American …, 2021 - ashpublications.org
Two generations ago, SCA was recognized as the first “molecular disease.” 2 This launched
decades of research into normal and sickle hemoglobin (HbA and HbS, respectively). These …

Emerging drugs for the treatment of sickle cell disease: a review of phase II/III trials

JM Ross, S Forté, D Soulières - Expert Opinion on Emerging Drugs, 2022 - Taylor & Francis
Introduction The substitution of glutamic acid by valine on the ß-globin chain produces the
hemoglobin S variant responsible for sickle cell disease (SCD), a disorder that affects …

Doping control approach: Identification of equine in vitro metabolites of voxelotor (GBT440), a hemoglobin S polymerization inhibitor

MB Subhahar, AK Karakka Kal, M Philip… - Rapid …, 2024 - Wiley Online Library
Rationale Sickle cell disease, a debilitating genetic disorder affecting numerous newborns
globally, has historically received limited attention in pharmaceutical research. However …