ChristianaCare is receiving a financial boost to continue its research into a therapy for sickle cell disease.
Researchers at ChristianaCare’s Gene Editing Institute are getting $1 million from the Wilmington-based Lisa Dean Moseley Foundation to try to create a new treatment for the inherited blood disorder sickle cell disease by using CRISPR technology to edit a patient’s genetic makeup.
Institute Director Dr. Eric Kmiec likens the therapy to spell check.
“If you think about a gene as being a word, in the sickle cell disease state that word is misspelled," said Kmeic. "So it’s now believed that the gene can be repaired or edited so the base is put in the gene of the correct letter is put into the word.”
Sickle cell disease is an inherited blood disorder affecting a patient pool of 100,000 Americans that is disproportionately African-American.
Kmiec says the institute has been conducting this research for years using donated patient blood samples.
He notes patients with different genetic makeup often have a different response to the CRISPR therapy and the institute is studying how that patient diversity factors into finding a cure for sickle cell disease.
“The ultimate goal of the grant is to create kind of an algorithm or paradigm--molecular algorithm, not a software program--where we can begin to predict how patients will respond to the CRISPR therapy and that, of course, is the goal of all genetic medicine,” said Kmiec.
He says the grant will likely take the institute's research in this area to its final conclusion.