Crispr and autism. Could the former cure the latter?
Scientists at University of California, San Diego, have developed a way to use the gene editing tool CRISPR-Cas9 to track and alter RNA processes within living cells, potentially leading to treatment breakthroughs for certain diseases and disorders such as cancer and autism.
In a recent publication of the bimonthly science journal Cell the research team describe how they were able to reconfigure CRISPR-Cas9 to recognize RNA sequences in cells rather than DNA sequences, thereby opening up a new method for studying the movement and functioning of RNA molecules within cells without damaging the RNA strand.
“Our results establish RCas9 as a means to track RNA in living cells in a programmable manner without genetically encoded tag,” say the study’s authors, led by David Nelles, a doctoral student in Materials Science and Engineering at UC San Diego.
So far, CRISPR-Cas9 has proven to be a revolution in the field of genetics, essentially providing scientists with a handy, all-purpose tool for isolating gene sequences within DNA strands and either rendering them inoperative or replacing them with other DNA segments. The possibilities for altering the basic building blocks of life now seem endless, from fighting antibiotic-resistant microbes to eliminating different types of inherited disease to creating disease-resistant crops.
“We are just beginning to see the implications of genome engineering using the CRISPR technology, but many diseases, including cancer and autism, are linked to problems with another fundamental biological molecule: RNA,” says the study’s co-author Gene Yeo of UC San Diego.
“Future developments of this work could enable researchers to measure other features of RNA processing or support therapeutic approaches to correct disease-causing RNA behaviors.”
RNA molecules serve to regulate the expression of genes and to transfer information necessary for the synthesis of proteins within the cell.
Diseases like cancer and neurodegnerative disorders like amyotrophic lateral sclerosis (ALS) have been linked to problems in RNA functioning. For instance, in its most common form among children and young adults, leukemia has recently been found to be driven by an RNA binding protein normally active in fetal tissue but switched off in adults. Researchers at UC Santa Cruz have shown that this protein is involved in the proliferation of cancer cells.
“This protein, IFG2BP3, has been correlated with many types of malignancies and with the worst prognoses,” said co-author Jeremy Sanford, associate professor of molecular, cell, and developmental biology at UC Santa Cruz. “What is exciting about this study is that it goes beyond correlation and shows causation, because we demonstrated for the first time that aberrant expression of this protein is sufficient to induce pathology.”
Recently, the Canada Gairdner International Awards announced its 2016 winners, this year for only the second time in its history giving all five of its international awards to pioneers in CRISPR-Cas9 technologies. Every year the Canada Gairdner International Awards hand out five awards of $100,000 each to biomedical scientists “who have made original contributions to medicine resulting in increased understanding of human biology and disease.”
Below: Genome Editing with CRISPR-Cas9, McGovern Institute for Brain Research at MIT