Baby KJ One Year Later: Gene-Editing Breakthrough Update
Автор: The Children's Hospital of Philadelphia
Загружено: 2026-02-24
Просмотров: 9857
Описание:
In the one year since a historic medical first, when he became the first in the world to receive a personalized gene-editing therapy, KJ has thrived. http://www.chop.edu/kjs-breakthrough
In a medical first, on Feb. 25, 2025, a child at Children’s Hospital of Philadelphia (CHOP) was successfully treated with a personalized gene editing therapy. The baby, KJ, had been diagnosed with a rare and often fatal urea cycle disorder called CPS1 deficiency. In just six months, a CHOP and Penn Medicine team led by Rebecca Ahrens-Nicklas, MD, PhD, and Kiran Musunuru, MD, PhD, created a CRISPR-based gene editing therapy customized to fix KJ’s defective gene. Today, that same team is working with the U.S. Food and Drug Administration to create a clinical trial platform to expedite and expand this research. They recently visited Washington, DC, with KJ and his family to advocate with policymakers with the goal of helping more children.
Gene editing therapy
Personalized gene editing
Urea cycle disorder
CPS1 deficiency
CRISPR
Clinical trial
KJ
KJ’s breakthrough
Rebecca Ahrens-Nicklas
Kiran Musunuru
MB01I1YMSTVM1CB
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