This study is looking at a new way to help people with a serious blood problem called Sickle Cell Disease (SCD). SCD can cause pain and other health issues because red blood cells, which carry oxygen, become sickle-shaped and can't move easily through blood vessels. Scientists are using a special technique called CRISPR/Cas9 to change the genetics of blood cells and help them work better. This research is for people aged 12 to 35 with severe SCD.
The trial is being conducted in two stages to check if the treatment is safe. Initially, it will involve 6 adult participants, and if it's safe, 3 younger participants aged 12-18 will be included. The study involves just one infusion (injection) of the modified cells.
- **Duration**: Single infusion with follow-up visits.
- **Eligibility**: Age 12-35 with severe SCD symptoms and specific health criteria.
- **Risks**: Must consider potential side effects and eligibility requirements.
If you are eligible and decide to join, you will need to give informed consent. The treatment aims to help manage SCD symptoms, but participation is voluntary and should be carefully considered.