This week, a remarkable scientific breakthrough was achieved with the first-ever application of a personalized in vivo gene editing treatment in a baby. Researchers developed a therapy targeting a rare mutation in the *CPS1* gene, showing significant improvement after three doses and opening new possibilities in precision medicine.
Can vision loss be reversed?
🧬This Breakthrough Study Reveals New Hope👁️ A rare cause of congenital (present at birth) vision loss is severe retinal dystrophy associated with the gene AIPL1. Until recently, this condition, like many other rare diseases, had no available treatment. However, a recent study published in The Lancet has demonstrated the transformative potential of gene therapy to change this … Sigue leyendo Can vision loss be reversed?
CRISPR to the rescue of trisomy 21
Leer en Español Promising Advances in Down Syndrome Down syndrome is a genetic condition caused by the presence of an extra chromosome, chromosome 21. In February 2025, a study published in the journal PNAS by researcher R. Hashizume and his team opened a new door in Down syndrome research. The study explored the possibility of removing the … Sigue leyendo CRISPR to the rescue of trisomy 21
Dra Adriana Perez-Grovas Saltijeral 
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