UCLA Doctors Hail Potential Cure For Bubble Baby Syndrome

Posted: November 19, 2014 at 6:44 pm

CBS Los Angeles (con't)

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WESTWOOD (CBSLA.com) Doctors say a groundbreaking stem cell therapy treatment out of UCLA may have cured Bubble Baby syndrome once and for all.

KNX 1070s Brian Ping reports Dr. Donald Kohn has perfected a gene therapy that has now cured 18 children born without an immune system, known as ADA-deficient severe combined immunodeficiency (SCID).

Only weeks after giving birth to fraternal twins in 2012, Alysia and Christian Padilla-Vaccaro found out their daughter Evangelinas immune system was so deficient that she could have no exposure to the outside world.

After enrolling their daughter in Dr. Donald Kohns revolutionary stem cell gene therapy treatment which was nearly three decades in the making doctors extracted stem cells from the bone marrow in Evangelinas hip, then used a modified mouse virus to correct her faulty gene before replacing the marrow.

You hear the words mouse virus and you want to run the other way, said mom Alysia. But they modify it so that its teaching it to do something that they want it to do, which is put something in there that was missing.

Evangelinas new immune system developed without side effects and she is now living a healthy normal life.

Her mother Alysia said while the process was difficult for any mom to go through, it was all worth it.

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UCLA Doctors Hail Potential Cure For Bubble Baby Syndrome

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