MDF Magazine Issue 72 December 2023

Testing Cure DMD
The team will also test how well the formulation works in
cells derived from human DMD patients.
“The gene editing efficiency will be a key performance endpoint
that will determine the efficacy of Cure DMD,” said
Niren Murthy, professor of bioengineering at the University of
California at Berkeley and co-investigator on this project. The
team anticipates achieving editing efficiency that will result in
more than 5% of dystrophin expression.
make a long-lasting impact,” Wang said.
Professors Aijun Wang and Diana Farmer with members of
their labs at UC Davis
A dream team for Cure DMD
How is Cure DMD different from other current therapies?
According to Wang, in-utero therapy holds unique advantages
for accessing tissues that are hard to edit after birth. It would
also allow the genetic disease to be corrected at an early developmental
stage.
“A single injection would be sufficient for this therapy given
that gene editing is permanent. The edited cells will proliferate
and populate the organs naturally as fetal development
continues in the womb,” Wang said.
The team also estimates that an injection in utero will be significantly
more affordable than the current treatments for
adult patients. Because of that, Cure DMD has the potential to
be accessible to low-income patients and those in underdeveloped
parts of the world.
“We can potentially treat many patients before it's too late,
just like in spina bifida.” —Aijun Wang, UC Davis professor of
surgery and biomedical engineering
Currently, there is no non-viral gene editing treatment that
can fully correct the mutation of the DMD gene or restore fulllength
dystrophin before birth. Cure DMD uses a non-viral
gene editing mechanism. This gives it significant advantages
over therapies now available and those currently in clinical
trials.
Wang expects that the success of this study will open the door
to treatments for many other diseases. Many patients with
genetic disorders are not diagnosed or treated prenatally due
to lack of treatment options. With the new gene editing and
delivery technologies and more prenatal diagnoses, Wang
thinks the field of treating genetic diseases will be very much
changed.
“We can potentially treat many patients before it's too late,
just like in spina bifida. If you can correct the spinal cord injury
and prevent the neurons from dying, that's a big win and will
McDonald is a professor and chair of the Department of Physical
Medicine and Rehabilitation. He is an internationally recognized
expert in the clinical management and rehabilitation
of neuromuscular diseases, including muscular dystrophies,
and the development of novel outcome measures for clinical
trials.
Smith, an assistant professor in the Departments of Physical
Medicine and Rehabilitation and Neurobiology, Physiology
and Behavior, is a collagen and muscle characterization expert.
Other UC Davis collaborators include:
Diana Farmer, distinguished professor and chair of the Department
of Surgery and a world-renowned fetal surgeon
Jan Nolta, director of the Stem Cell Program and the UC Davis
Gene Therapy Center in the Institute for Regenerative Cures
Nipavan Chiamvimonvat, professor and associate chief for
research in the Division of Cardiovascular Medicine and a
leading expert in cardiac physiology
Herman Locsin Hedriana, medical director of prenatal diagnosis
of Northern California and chief of the UC Davis Division of
Maternal-Fetal Medicine. Hedriana is a leading expert in prenatal
diagnosis and maternal-fetal medicine.
Article available at: https://health.ucdavis.edu/news/
headlines/uc-davis-health-to-develop-in-utero-therapy-forduchenne-muscular-dystrophy-/2023/02
“Know me for my abilities, not my disability.” – Robert M. Hensel