Focusing on analysis of the function of members of the Rho GTPase family
Stories and News from the Lab
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New York Times: Pioneering Gene Therapy Freed Her of Sickle Cell. Is a Cure at Hand?
Three gene therapy trials, including one at Boston Children's Hospital, have produced remarkable results. The question remains, are we closer to a cure that is accessible to everyone?
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![Matthew, who underwent gene therapy for ALD, playing soccer]()
Matthew, the ‘wee marvel’
Eleven-year-old Matthew from Enniskillen, UK, made history in 2013 when he became one of the first in the world to receive gene therapy for adrenoleukodystrophy (ALD).
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After decades of evolution, gene therapy arrives
From viral vectors to custom antisense oligonucleotides and mRNA: A brief history of gene therapy.
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![Agustín received gene therapy for SCID.]()
Gene therapy turns 10
10-year-old Agustín is an active fifth-grade soccer and tennis player — none of which would be possible without a genetic breakthrough by doctors at Boston Children’s 10 years ago.
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Gene therapy for CGD shows promising early results
Good early results with gene therapy for rare immune deficiency for CGD provides promising early results.
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New York Times: At 16, She’s a Pioneer in the Fight to Cure Sickle Cell Disease
Helen Obando is the youngest person ever to get a gene therapy that scientists hope will cure the disease, which afflicts 100,000 Americans.
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Gene therapy to boost fetal hemoglobin continues to do well in sickle cell trial
A pilot gene therapy treatment for sickle cell disease, restoring patients’ ability to make fetal hemoglobin, has produced good results.
