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Gene therapy breakthrough allows boy with severe immune disease to lead a normal life.
A groundbreaking gene therapy has enabled Eisa Hussain, a boy with the severe immune disease leukocyte adhesion deficiency 1 (LAD-1), to lead a normal life.
Previously, patients with LAD-1 typically died before age two without a stem cell transplant, which required a close-match donor.
The new therapy modifies the patient's cells to produce a crucial protein for fighting infections.
After receiving the treatment at Great Ormond Street Hospital, Eisa can now walk, attend school, and enjoy activities like football.
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El avance de la terapia genética permite que un niño con una enfermedad inmunológica grave lleve una vida normal.