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Prime Editing: A Revolutionary Technology for Precise Treatment of Genetic Disorders
This review outlines the latest research advancements in prime editing technology, delivery strategies, and the challenges that must be addressed to fully realize its therapeutic potential, emphasizing the high potential of prime editing in the remission or cure of genetic diseases. ABSTRACT Genetic diseases have long posed significant challenges, with
Mengyao Li, Yi Lin, Qiang Cheng, Tuo Wei
wiley +1 more source
Thalassemia and Hemoglobin E in Southern Thai Blood Donors
Thalassemia and hemoglobin E (Hb E) are common in Thailand. Individuals with thalassemia trait usually have a normal hemoglobin concentration or mild anemia. Therefore, thalassemic individuals who have minimum acceptable Hb level may be accepted as blood
Manit Nuinoon+4 more
doaj +1 more source
BRAF mutation in hairy cell leukemia
BRAF is a serine/threonine kinase with a regulatory role in the mitogen-activated protein kinase (MAPK) signaling pathway. A mutation in the RAF gene, especially in BRAF protein, leads to an increased stimulation of this cascade, causing uncontrolled ...
Ahmad Ahmadzadeh+7 more
doaj +1 more source
FURTHER STUDIES ON THE GENETICS OF THALASSEMIA [PDF]
James V. Neel, William N. Valentine
openalex +1 more source
Evaluation of mathematical indices as tools for distinguishing β-thalassemia trait from iron deficiency anemia in Portuguese females with microcytic anemia [PDF]
Microcytic anemia is a common condition frequently caused by iron deficiency anemia (IDA) or β-thalassemia trait (BTT). Some mathematical indices have been described as fast and inexpensive tools for distinguishing these two conditions.
Faleiro, Bárbara D.+2 more
core
Under normoxic conditions, the SUMOylated fraction of TFAP2A cannot interact with the Ku70/Ku80 complex, nor it can bind to chromatin. The unSUMOylated fraction pre‐occupies the chromatin of a subset of hypoxia‐induced genes together with the Ku70/Ku80 complex for basal euchromatin formation. Under hypoxic conditions, the SUMOylation equilibrium shifts
Amalia Kanoura+8 more
wiley +1 more source
Multiple Inherited Erythrocyte Abnormalities in an American Negro Family: Hereditary Spherocytosis, Sickling and Thalassemia [PDF]
Flossie Cohen+3 more
openalex +1 more source
Study of Pregnancy Outcome in E-Beta Thalassaemia Mothers [PDF]
Forty eight E-Beta thalassaemia patients were studied in NRS Medical College, Kolkata, West Bengal during the period from 2000-2006. In all patients Hb% ranged from 5.2g% - 9.6g%.
Bhattacharyaya, Maitryaee+1 more
core +1 more source
Classifying Copy Number Variations Using State Space Modeling of Targeted Sequencing Data: A Case Study in Thalassemia [PDF]
Thalassemia, a blood disorder and one of the most prevalent hereditary genetic disorders worldwide, is often caused by copy number variations (CNVs) in the hemoglobin genes. This disorder has incredible diversity, with a large number of distinct profiles corresponding to alterations of different regions in the genes. Correctly classifying an individual'
arxiv