An exon-skipping technique using dual single-guide RNA/Cas9 ribonucleoproteins targeted at 3 novel COL7A1 exons with pathogenic heterozygous mutations achieved exon deletion rates of up to 95%.

The Johnson Haynes Jr. M.D. Comprehensive Sickle Cell Center is part of the journey of Addilee Mason, a 13-year-old girl who received gene therapy in 2024 after exhausting other treatment options ...

When a person inherits two copies of the HBB gene with the mutation that causes sickle cell disease, their body produces unusual hemoglobin, which doctors call hemoglobin S (HbS).