Eighteen children born with
a tragically rare disease can look forward to healthy lives.
The children were born with
the so-called Bubble Baby disease, scientifically known as severe combined
immunodeficiency (SCID). Those with it have a malfunctioning immune system and
must be isolated so that germs don't penetrate them.
But Dr. Donald Kohn, a stem
cell researcher at UCLA's Eli and Edythe Broad Center of Regenerative Medicine
and Stem Cell Research in Los Angeles, has developed a gene therapy that cured
a group of children with adenosine deaminase-deficient SCID, a subtype of the
illness that affects about 15% of SCID patients — but just 1 in every 200,000
to 1 million births around the world.
"All of the children
with SCID that I have treated in these stem cell clinical trials would have
died in a year or less without this gene therapy, instead they are all thriving
with fully functioning immune systems," Kohn, who spent 30 years on his
cure, said in a statement.
His experiments involved
removing children's blood stem cells then genetically altering them to correct
their errors. The treatment was shown to have no side effects, and the next
step is FDA-approval. There's hope that the approach may work in treating
sickle cell disease, too.
Now, kids like Evangelina
Padilla-Vaccaro, of Corona, Calif., can go to the store and get kisses from
their moms.
"To finally kiss your
child on the lips, to hold her, it's impossible to describe what a gift that
is," her mother, Alysia Padilla-Vacarro, told ABC News. "I gave birth to my daughter, but Dr.
Kohn gave my baby life."
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