[HTML][HTML] Gene therapy for β-hemoglobinopathies
M Cavazzana, C Antoniani, A Miccio - Molecular therapy, 2017 - cell.com
β-Thalassemia and sickle cell disease (SCD) are the world's two most widely disseminated
hereditary hemoglobinopathies. β-Thalassemia originated in the Mediterranean, Middle …
hereditary hemoglobinopathies. β-Thalassemia originated in the Mediterranean, Middle …
Thalassemias: from gene to therapy
G De Simone, A Quattrocchi, B Mancini… - Molecular Aspects of …, 2022 - Elsevier
Abstract Thalassemias (α, β, γ, δ, δβ, and εγδβ) are the most common genetic disorders
worldwide and constitute a heterogeneous group of hereditary diseases characterized by …
worldwide and constitute a heterogeneous group of hereditary diseases characterized by …
A molecular glue degrader of the WIZ transcription factor for fetal hemoglobin induction
PY Ting, S Borikar, JR Kerrigan, NM Thomsen… - Science, 2024 - science.org
Sickle cell disease (SCD) is a prevalent, life-threatening condition attributable to a heritable
mutation in β-hemoglobin. Therapeutic induction of fetal hemoglobin (HbF) can ameliorate …
mutation in β-hemoglobin. Therapeutic induction of fetal hemoglobin (HbF) can ameliorate …
Dual inhibition of DNA and histone methyltransferases increases viral mimicry in ovarian cancer cells
Ovarian cancer ranks as the most deadly gynecologic cancer, and there is an urgent need to
develop more effective therapies. Previous studies have shown that G9A, a histone …
develop more effective therapies. Previous studies have shown that G9A, a histone …
Recent progress in understanding and manipulating haemoglobin switching for the haemoglobinopathies
DS Vinjamur, DE Bauer… - British journal of …, 2018 - Wiley Online Library
The major β‐haemoglobinopathies, sickle cell disease and β‐thalassaemia, represent the
most common monogenic disorders worldwide and a steadily increasing global disease …
most common monogenic disorders worldwide and a steadily increasing global disease …
Pharmacological induction of fetal hemoglobin in β-thalassemia and sickle cell disease: An updated perspective
A significant amount of attention has recently been devoted to the mechanisms involved in
hemoglobin (Hb) switching, as it has previously been established that the induction of fetal …
hemoglobin (Hb) switching, as it has previously been established that the induction of fetal …
A specific G9a inhibitor unveils BGLT3 lncRNA as a universal mediator of chemically induced fetal globin gene expression
S Takase, T Hiroyama, F Shirai, Y Maemoto… - Nature …, 2023 - nature.com
Sickle cell disease (SCD) is a heritable disorder caused by β-globin gene mutations.
Induction of fetal γ-globin is an established therapeutic strategy. Recently, epigenetic …
Induction of fetal γ-globin is an established therapeutic strategy. Recently, epigenetic …
Fetal haemoglobin in sickle-cell disease: from genetic epidemiology to new therapeutic strategies
G Lettre, DE Bauer - The Lancet, 2016 - thelancet.com
Sickle-cell disease affects millions of individuals worldwide, but the global incidence is
concentrated in Africa. The burden of sickle-cell disease is expected to continue to rise over …
concentrated in Africa. The burden of sickle-cell disease is expected to continue to rise over …
Discovery of ligands for TRIM58, a novel tissue-selective E3 ligase
K Hoegenauer, S An, J Axford… - ACS Medicinal …, 2023 - ACS Publications
Redirecting E3 ligases to neo-substrates, leading to their proteasomal disassembly, known
as targeted protein degradation (TPD), has emerged as a promising alternative to traditional …
as targeted protein degradation (TPD), has emerged as a promising alternative to traditional …
β-Thalassemia intermedia: a comprehensive overview and novel approaches
C Asadov, Z Alimirzoeva, T Mammadova… - International journal of …, 2018 - Springer
Abstract β-Thalassemia intermedia is a clinical condition of intermediate gravity between β-
thalassemia minor, the asymptomatic carrier, and β-thalassemia major, the transfusion …
thalassemia minor, the asymptomatic carrier, and β-thalassemia major, the transfusion …