CLINICAL NEWS
On Location
American Society of Hematology’s
WORKSHOP ON GENOME EDITING
An attendee taking notes at the ASH Workshop on Genome Editing
Harnessing the Power of Gene Editing
or years, scientists have been manipulating genes in animal and human cells to gain a better understanding of how genomes work, with an eye toward developing improved medical
treatments. Gene editing, in particular, involves using nucleases – enzymes that essentially
act as “molecular scissors” – to cut DNA strands and insert, delete, or replace a specific
piece or sequence of DNA.
Until recently, the complexity of gene-editing techniques has limited scientists’ ability to fully
leverage this tool in basic and clinical research. Now, evolving and easier-to-use genome-editing platforms are accelerating the pace of scientific research and may help generate promising new therapies
for a range of diseases, including genetic disorders, cancer, and blood disorders such as hemophilia,
sickle cell anemia, and immunodeficiencies.
Modifying DNA within hematopoietic stems cells (HSCs) has the potential
to alter blood cells or the immune system. What’s more, it is conceivable
that improved outcomes in patients with certain genetic disorders could be
achieved through a single, or shortened, course of treatment.
Despite the progress that is being made and the enthusiasm about potential new therapies, gene editing is not always efficient or reproducible on
a large scale, and can cause unintended genetic changes, or “off-target effects.” This problem raises important questions about the long-term safety
of gene editing, an issue that needs to be resolved before the approach is
routinely used to treat patients.
Recognizing the potential of genome editing to correct genetic flaws
that cause many inherited blood disorders and other diseases, the American Society for Hematology (ASH) is helping to advance this emerging
technology. On July 14 and 15, 2016, ASH hosted a Workshop on Genome
Editing in Washington, DC, that brought together more than 150 leading
clinical and laboratory-based scientists, funders, and regulators to discuss
this cutting-edge technology. (Notably, this workshop focused on somatic
ASHClinicalNews.org
cell editing, not germline editing.)
ASH Clinical News spoke with program co-chairs Mitchell Weiss,
MD, PhD, the Arthur Nienhuis Endowed Chair in Hematology at St. Jude
Children’s Research Hospital, in Memphis, Tennessee, J. Keith Joung, MD,
PhD, professor of pathology at Harvard Medical School and pathologist
at Massachusetts General Hospital in Boston, Massachusetts, and keynote
speaker Stuart Orkin, MD, the David G. Nathan Professor of Pediatrics at
Harvard Medical School, to learn more about the state of genome editing
and highlights from the meeting.
Gene editing has become a powerful laboratory research tool for modeling diseases. By manipulating the genome of various organisms, scientists
are now able to develop new cell lines and animal models, which will
greatly enhance the quality and pace of basic and translational research.
According to Dr. Weiss, “the major discoveries in this area came from
basic research studies of protein structure, plants, and bacteria. Without
basic research, there would be no translational research and, for this reason, we must pursue, support, and fund basic research with the same vigor
and enthusiasm [or more] as translational research.”
Technological Advances Driving Rapid Progress
A recurring theme at the meeting was the rapid pace with which these
technologies are being developed, refined, and put into practice. “Our
ability to manipulate the genome precisely and easily is accelerating at a
remarkable pace,” said Dr. Weiss.
Dr. Joung agreed, commenting “It is very exciting that there is such
rapid progress and the number of diseases being studied.”
Several gene-editing platforms are being used in basic and cl