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A national leader in pediatric gene therapy, CHLA has treated more patients with the sight-saving Luxturna than any other center in the country.

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No Longer Night-Blind, Natalia Isn’t Afraid of the Dark

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After being treated with a breakthrough gene therapy at Children’s Hospital Los Angeles for a genetic eye disorder, 5-year-old Natalia can now see clearly for the first time in her life.

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‘I Can See! I Can See!’

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Research and Breakthroughs

What Is Gene Therapy?

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How next-generation gene therapies target the underlying causes of genetic diseases.

Work That Matters

Trainee Spotlight: Leila Bahmani, PhD

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Since high school, Leila has been fascinated by how DNA molecules define our fundamental characteristics.

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Research and Breakthroughs

As FDA Expands Approval of a New Gene Therapy to Treat Duchenne Muscular Dystrophy, a CHLA Expert Weighs in

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The new approval opens the gene therapy to a wider range of children with Duchenne muscular dystrophy, a rare genetic disorder that worsens over time.

Laura Perin and Stefano Da Sacco with members of the GOFARR lab

Research and Breakthroughs

A Closer Look: Why Is Research Essential at a Children’s Hospital?

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The ambitions of pediatric researchers are global in scale: Beyond patients at their own hospital, they look to shape national and international standards for pediatric care.

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Research and Breakthroughs

Elevidys Gene Therapy: A Neurologist’s Perspective

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A CHLA expert shares her experience of administering this gene therapy for Duchenne—and why comprehensive care is critical for patients.

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Managing Congenital Aortic Stenosis in the Operating Room

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