[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 …

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 …

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 …

Dual inhibition of DNA and histone methyltransferases increases viral mimicry in ovarian cancer cells

M Liu, SL Thomas, AK DeWitt, W Zhou, ZB Madaj… - Cancer research, 2018 - AACR
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 …

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 …

Pharmacological induction of fetal hemoglobin in β-thalassemia and sickle cell disease: An updated perspective

R Bou-Fakhredin, L De Franceschi, I Motta… - Pharmaceuticals, 2022 - mdpi.com
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 …

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 …

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 …

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 …

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 …