Media Coverage
They didn't understand why their kids kept getting sick. After a rare diagnosis, a new gene therapy offered hope.
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CBS News
Gene therapy trial helps children with LAD-I
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The Medicine Maker
Inhalable gene therapies? Here’s how they could work
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The Medicine Maker
Good news: genical therapy has canceled the symptoms of a rare genetic disease in 9 children
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Evidence Network
UCLA team developing one-time, inhalable CF gene-editing treatment
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Cystic Fibrosis News Today
Going public: Doudna’s dream team launches groundbreaking sickle cell trial
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Genetic Engineering & Biotechnology News
Need for better, more specific delivery methods for gene therapies in muscular dystrophy: Melissa Spencer, Ph.D.
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Neurology Live
AAV vectors in gene therapy: Overcoming barriers for muscular dystrophy treatment
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Neurology Live
Health Matters: Bubble boy given new lease on life
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Global News