For his part, Collins, who has led NIH since 2009 and been kept on by the Trump administration,
pointed to an array of promising NIH activities, including the development of new technologies to provide insights into human brain circuitry and function through the Brain Research through Advancing Innovative Neuroethologies (BRAIN initiative) and the use of the gene - editing tool CRISPR - Cas9 to correct
mutations and clear the way to develop and test a «curative therapy» for the first molecular disease:
sickle cell disease.
Working in human cells, Liu and coworkers used adenine base editing to correct a
point mutation that causes the iron - storage disorder hemochromatosis and to install
mutations that protect against
sickle cell anemia.