The New York Times has just reported a historic milestone in medicine: A child has been cured using the first gene-editing therapy custom-built for an individual patient. The therapy corrected a single-letter mutation in the genome — the root cause of a deadly rare disease.
This breakthrough offers new hope for the more than 30 million Americans living with one of over 7,000 rare genetic diseases, the vast majority of which have no effective treatment. It also brings renewed attention to the importance of continued investment in fundamental research, which laid the groundwork for this moment.
Rice University is home to a growing concentration of researchers advancing the science and tools that make these therapies possible — from CRISPR-based gene correction to cell reprogramming and next-generation synthetic biology.
Gang Bao, a pioneer in nanomedicine, molecular imaging and genome editing with long-standing experience researching CRISPR-based gene correction techniques for treating sickle cell diseases and other disorders, is available for comment.
“It’s an extremely exciting moment for the field of gene editing,” said Bao, the A.J. Foyt Family Professor of Bioengineering at Rice. “While ex vivo gene editing therapy has received FDA approval for treating sickle cell disease and transfusion-dependent beta-thalassemia, this represents a historic milestone on in vivo gene editing for treating genetic diseases. There will be unprecedented opportunities in performing in vivo base editing for treating single-gene disorders with high efficiency and safety.”
Isaac Hilton develops genome editing technologies to precisely control human cell functions, aiming to better understand and treat diseases like cancer.
“This is a landmark moment for the field that highlights the therapeutic promise of genome editing,” said Hilton, associate professor of biosciences and bioengineering at Rice.
Jerzy Szablowski researches methodologies for controlling and monitoring cells in one of the most complex systems in nature — the mammalian brain. His lab focuses on developing noninvasive gene therapy and diagnostics for brain disorders, including Parkinson's disease.
"Gene therapy is one of the most exciting frontiers in medicine — including for treating diseases in the brain, where targeted, noninvasive approaches could make an enormous impact," Szablowski said.