Children's Hospitals Today - Fall 2023 - 13

RESEARCH / FEATURE
LEADING THE WAY IN GENE THERAPY
Gene therapy corrects underlying genetic issues, curing conditions not
previously treatable and diseases that demand extraordinary efforts. Children's
hospitals have been at the forefront of much of the research, and they continue
tailoring pediatric-specific therapies for chromosomal disorders.
DUCHENNE MUSCULAR
DYSTROPHY
Research at Nationwide
Children's Hospital has led to
the development of Elevidys,
the first gene therapy to treat
Duchenne muscular dystrophy
in 4- and 5-year-olds. This is the
second gene therapy developed
at Nationwide's Abigail Wexner
Research Institute's Center
for Gene Therapy to receive
FDA approval.
ARTEMIS-SCID
In late 2022, 10 children born
with Artemis-SCID, a rare
genetic disorder leaving
newborns without functioning
immune systems, were treated
with a new gene therapy. This
treatment, pioneered by UC San
Francisco, home to UCSF Benioff
Children's Hospital, allows
researchers to treat patients
with their own stem cells by
adding a healthy copy of the
Artemis gene to their harvested
stem cells.
SPINAL MUSCULAR ATROPHY
SMA is the No. 1 genetic cause
of infant mortality. The only
FDA-approved gene therapy,
Zolgensma, was tested in clinical
trials led by Boston Children's
Hospital. At least 3,000 children
globally have been treated with
the therapy.
BLOOD DISORDERS
Researchers at Children's
Hospital of Philadelphia have
developed a proof-of-concept
model that delivers gene editing
tools capable of modifying
diseased blood cells within
the body. Standard treatment
requires children to have
chemotherapy and stem cell
transplants. The gene editing
tool would eliminate the need
for such treatments, and it
could expand access and reduce
the cost of gene therapies for
blood disorders.
CHILDHOOD CANCERS
The latest advancement in
treating childhood cancer is
chimeric antigen receptor
(CAR) T-cell therapy, which
genetically modifies a
patient's T-cells to enhance
their cancer-fighting abilities.
Many children's hospitals
have been at the forefront of
new CAR-T developments for
pediatric cancers, including
Johns Hopkins All Children's
Hospital's Center for Adoptive
Cellular Therapy.
CHILDREN'S HOSPITALS TODAY Fall 2023
13

Children's Hospitals Today - Fall 2023

Table of Contents for the Digital Edition of Children's Hospitals Today - Fall 2023

Children's Hospitals Today - Fall 2023 - Cover1
Children's Hospitals Today - Fall 2023 - Cover2
Children's Hospitals Today - Fall 2023 - 1
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Children's Hospitals Today - Fall 2023 - Cover3
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