Health
Researchers develop new gene therapy vector for blood disorders – The Science Board
Researchers at Children’s Hospital of Philadelphia have developed a new gene therapy vector for blood disorders that is safer and more effective than those currently…
The new vector is an engineered lentivirus that minimizes the risk of toxic side effects in disorders such as sickle cell disease and beta-thalassemia. Typically, lentiviral vectors include a truncated version of intron 2 that does not code for proteins. But the team hypothesized that inclusion of a full-length intron, in addition to other genomic elements, could promote a uniform expression of the beta-globin gene and enhance functional hemoglobin. Based on this, they generated five vectors by…
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