It leads to anemia, vaso-occlusive events and crises (painful episodes in which small blockages starve tissue of oxygen), strokes, progressive and irreversible organ damage, decreased quality of life, and early death.
“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field, especially for individuals whose lives have been severely disrupted by the disease, by approving two cell-based gene therapies today,” said Nicole Verdun, director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research, said in the FDA’s announcement.
The central problem is with adult hemoglobin, the iron-containing protein in red blood cells that transports oxygen from the lungs to the rest of the body.
HbF is optimized for pregnancy, transferring oxygen from maternal blood to fetal tissue, and the gene that encodes it is shut off shortly after birth as the body transitions to HbA.
Moreover, when it’s mixed with HbS, it gets in the way of the mutated protein polymerizing with itself, preventing it from forming structures that deform red blood cells.
The BCL11A transcription factor is the protein responsible for shutting off the gene for HbF shortly after birth as the body transitions to the adult version.
The original article contains 780 words, the summary contains 208 words. Saved 73%. I’m a bot and I’m open source!
This is the best summary I could come up with:
It leads to anemia, vaso-occlusive events and crises (painful episodes in which small blockages starve tissue of oxygen), strokes, progressive and irreversible organ damage, decreased quality of life, and early death.
“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field, especially for individuals whose lives have been severely disrupted by the disease, by approving two cell-based gene therapies today,” said Nicole Verdun, director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research, said in the FDA’s announcement.
The central problem is with adult hemoglobin, the iron-containing protein in red blood cells that transports oxygen from the lungs to the rest of the body.
HbF is optimized for pregnancy, transferring oxygen from maternal blood to fetal tissue, and the gene that encodes it is shut off shortly after birth as the body transitions to HbA.
Moreover, when it’s mixed with HbS, it gets in the way of the mutated protein polymerizing with itself, preventing it from forming structures that deform red blood cells.
The BCL11A transcription factor is the protein responsible for shutting off the gene for HbF shortly after birth as the body transitions to the adult version.
The original article contains 780 words, the summary contains 208 words. Saved 73%. I’m a bot and I’m open source!