WFAA Original: How two children with a rare genetic disease could lead to breakthrough treatments

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A UT Southwestern scientist hopes gene therapy can treat rare diseases. His work could lead to breakthrough treatments for Alzheimer’s, autism or schizophrenia.
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This is amazing, gene therapy will be the answer to many diseases. This gives me so much hope for the future.

jameswest
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Can we correct other diseases by taking out the old gene and putting in one that was made? This is very interesting.

Seacoastgal
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@1:35
What is the name of the diseases

alexisfuhr
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Why spend money to treat these rare diseases, people ask? They would not ask if their child or a child they know has that rare disease.

RobinPM
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Genetics labs are quite sensitive to contamination

The_Vanished