Health
Researchers are developing proof-of-concept therapies that raise adult and fetal hemoglobin
Dr. Stefano Rivera, Chairman Kwame Ohene Frenpon on Sickle Cell Anemia, Professor of Pediatrics at CHOP. Credit: CHOP
Researchers at the Children’s Hospital of Philadelphia (CHOP) have developed a proof-of-concept treatment for blood disorders such as sickle cell disease and beta-thalassemia. This treatment may increase hemoglobin levels by activating hemoglobin production in both the foetation and adults. Using viral vectors designed to reactivate fetal hemoglobin production, suppress mutant hemoglobin, and supply functional adult hemoglobin, researchers have found that a single vector produces more hemoglobin. I found that. We have developed a generative approach. The results were announced at. Hematologyka..
“So far, researchers have Cachexia of blood favorite Chewing erythrocytosis Or Beta Thalassemia: Adult hemoglobin Increased gene or fetal production hemoglobin“Dr. Stefano Rivera, Chairman of Sickle Cell Anemia and Senior Author of Pediatrics at CHOP, said: It produces more hemoglobin per vector. It provides an opportunity. In these patients, “”
Sickle cell disease and beta-thalassemia are hereditary blood disorders caused by abnormalities. gene Hemoglobin is a protein found in red blood cells that carries oxygen from the lungs to tissues throughout the body. In the womb, the gamma globin gene produces fetal hemoglobin, but after birth, this gene is turned off, the beta globin gene is turned on, and adult hemoglobin is produced. Patients with sickle cell disease and beta-thalassemia have a mutation in the betaglobin gene that causes the production of mutant hemoglobin, leading to severe growth retardation and yellow spots, pain attacks, pulmonary hypertension, and stroke. Causes health complications.
Current research focuses on treating these blood disorders, increasing fetal hemoglobin that does not mutate in these conditions, or adding a functional copy of adult hemoglobin by gene therapy. Viral vector Supply new genetic material. However, both of these approaches have limitations and neither has been established as a complete curative approach.
Increase Hemoglobin value One treatment is a researcher led by co-author Dr. Danuta Jarocha. Sylvia Lorenco-Combine two tactics into one Gene therapy vector. To that end, they focused on a transcription factor called BCL11A. BCL11A effectively operates a switch that turns off fetal hemoglobin production and turns on adult hemoglobin production. Researchers have found that the ability to suppress BCL11A using artificial vectors maintains fetal hemoglobin production and turns off mutant adult hemoglobin production, while the beta-globin gene is functioning. did. I assumed I could add a nice copy again. More hemoglobin production.
In cell lines of patients with sickle cell disease and beta-thalassemia, researchers tested a vector containing a gene encoding adult hemoglobin and a microRNA sequence targeting BCL11A found in the fetus. It was discovered that adults can be raised. Hemoglobin in vitro at the same time. BCL11A was not completely knocked down, but inhibition was sufficient to reduce the production of adult mutant hemoglobin. By increasing both fetal hemoglobin and functional adult hemoglobin, this vector was able to induce more functional hemoglobin production than a vector that expresses beta-globin alone.
“Future research will evaluate this approach using stronger vectors developed in the lab and recently published,” says Rivera. “We want to combine these two technologies to create a stronger vector that can provide therapeutic levels of hemoglobin to these patients.”
This study was supported by CHOP’s CuRED Frontier Program, which is dedicated to discovering new and improved curative therapies for blood disorders such as sickle cell disease and beta-thalassemia.
Silvia Pires Lourenco et al. Include shRNAs that target. The β-globin expression vector BCL11A enables the co-synthesis of curative adult and fetal hemoglobin. Hematologyka (2021). DOI: 10.3324 / haematol.2020.276634
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Researchers are developing proof-of-concept therapies that raise adult and fetal hemoglobin
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