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Standing infant with dark skin tone and dark hair wearing onesie with colorful dinosaur print smiles as they stand up supporting themselves on a crib railing

Gene Therapy Saves Greigh From a Paralyzing Muscle Disease

September 22, 2023

Newborn screening found that Greigh had spinal muscular atrophy. He was sent to Children’s Hospital Los Angeles, where gene therapy saved him from paralysis.

Scientist in white coat holds a small tube of blood with a blue-gloved hand. A colorful DNA strand model is in the background.

Care Innovation

Improving Access to Cell and Gene Therapy in Pediatrics

October 2, 2024

Gene therapy holds tremendous potential for children, but many promising therapies are shelved. Here’s what a new model could look like.

A young boy with a medium skin tone sitting in a wheelchair and smiling while an adult woman with a medium skin tone steps out of a car and smiles at him, holding his hand.

Research and Breakthroughs

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

July 1, 2024

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.

Paul S. Viviano, President and Chief Executive Officer of Children’s Hospital Los Angeles; Alan S. Wayne, MD, Director of the Cancer and Blood Disease Institute at CHLA; and Lara Khouri, MBA, MPH, Executive Vice President and Chief Operating Officer of CHLA, at the grand opening of the cGMP Laboratory

Research and Breakthroughs

New cGMP Facility Aims to Accelerate Cell Therapy for Pediatric Cancer

May 12, 2023

Four investigators share their research—and how the USC/CHLA facility will help bring novel cell therapies to clinical trials faster.

Press Release

Children’s Hospital Los Angeles Treats First Patient on the West Coast Using Gene Therapy

March 21, 2018

Surgeons of The Vision Center at Children’s Hospital Los Angeles completed a revolutionary gene replacement procedure on Tuesday, March 20 at 9:20 AM, to restore vision in a patient with retinal

Computer illustration showing abundant lymphoblast cells in human bone marrow

Research and Breakthroughs

Can a Novel Approach Lead to Less-Toxic Treatments for Leukemia?

October 27, 2023

A new study finds that blocking Artemis significantly reduces the proliferation of leukemia cells, without affecting normal cells.

Computer illustration of CAR (chimeric antigen receptor) T cell immunotherapy, a process that is being developed to treat cancer

Research and Breakthroughs

5 Things to Know About Advances in Cancer Research

July 3, 2023

Dramatic advances in cancer research and treatment over the past two decades have made it possible for more children to survive a cancer diagnosis.

Nano, wearing mouse ears, poses with Buzz Lightyear at Disney World.

Our Patients

Nano's Next Chapter

July 29, 2024

Four years after bone marrow transplant, multidisciplinary care helps a Fanconi anemia patient focus on ‘kid stuff.’

A child wearing a white headscarf with yellow and blue circles on it sits in a hospital bed, looking at the camera.

Research and Breakthroughs

Study Targets Cisplatin Resistance in Pediatric Germ Cell Tumors

August 13, 2024

A CHLA oncologist is leading new research to improve outcomes for children, adolescents and young adults with relapsed or treatment-resistant germ cell tumors.

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