Back to today’s briefing

Story perspectives

10-Month-Old Returns Home After Revolutionary Gene Therapy

6/6/2025

50 5

Summary

Story summary

After 307 long days at the Children's Hospital, 10-month-old KJ Muldoon from Philadelphia has finally gone home, thanks to groundbreaking CRISPR gene-editing therapy for a rare genetic disorder. This innovative treatment not only transformed KJ's life but also ignites hope for families facing similar challenges, marking a monumental leap in gene therapy for ultra-rare diseases.

1 of 1

Story summary

After 307 long days at the Children's Hospital, 10-month-old KJ Muldoon from Philadelphia has finally gone home, thanks to groundbreaking CRISPR gene-editing therapy for a rare genetic disorder. This innovative treatment not only transformed KJ's life but also ignites hope for families facing similar challenges, marking a monumental leap in gene therapy for ultra-rare diseases.