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Gene Therapy Revitalizes Lives of 9 LAD-I Children

5/2/2025

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Summary

Story summary

In a groundbreaking development, nine children suffering from severe leukocyte adhesion deficiency-I (LAD-I) are thriving two years post-investigational gene therapy led by UCLA. This innovative treatment revitalizes blood stem cells, removing the need for risky bone marrow transplants. With all patients experiencing enhanced immune function, the therapy is now under FDA review for wider application.

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Story summary

In a groundbreaking development, nine children suffering from severe leukocyte adhesion deficiency-I (LAD-I) are thriving two years post-investigational gene therapy led by UCLA. This innovative treatment revitalizes blood stem cells, removing the need for risky bone marrow transplants. With all patients experiencing enhanced immune function, the therapy is now under FDA review for wider application.