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Could a pill replace gene therapy for sickle cell disease ... - MSNOrkin's team has now reported the crystal structure of the BCL11A tetramer and suggested several ways to attack it with small molecules.
The switch from fetal γ-globin to adult β-globin is controlled by BCL11A. Genetic manipulation of BCL11A improves disease outcomes by turning γ-globin expression back on to compensate for ...
They then labeled BCL11A-producing neurons in mice with fluorescent molecules, which then lit up under the microscope.
In a 2013 Science paper, we showed that common genetic variation in BCL11A that leads to small but measurable differences in fetal haemoglobin levels resides in the intron of the gene.
Molecular glue -based degraders, small molecules that shunt proteins of interest to the cell’s garbage disposal system, have been making waves in oncology.
New small molecule could treat sickle cell disease in adults that don't respond to hydroxyurea, alone Date: July 31, 2024 Source: Boston Medical Center Summary: Researchers found a small molecule ...
A self-inactivating lentivirus vector with a short hairpin RNA designed to interfere with BCL11A expression was inserted into autologous hematopoietic stem cells of patients with sickle cell ...
Researchers from the UK have found that overactivity in a gene called BCL11A may lead to the development and progression of triple-negative breast cancer.
To investigate other options, Daniel Bauer at Harvard Medical School in Boston and colleagues scanned the genomes of 1178 people with sickle cell anaemia to look for variants in the BCL11A gene ...
They then labeled BCL11A-producing neurons in mice with fluorescent molecules, which then lit up under the microscope.
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