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🥳Unprecedented Breakthrough in Diabetes Treatment
For the first time in medical history, pancreatic cells from a deceased donor have been genetically edited using CRISPR technology and transplanted into a man with type 1 diabetes. These cells have successfully produced insulin for months and responded to blood sugar changes without triggering the immune system or requiring immunosuppressive drugs.
Currently, the only way for individuals with type 1 diabetes to eliminate their dependence on insulin injections is through a transplant of pancreatic islet cells from a deceased donor. This method can restore insulin production for years but is rarely performed due to the limited availability of donor pancreases and the need for lifelong immunosuppressive drugs, which can lead to infections, cancer, and other serious side effects.
To address the shortage of donor pancreases, some companies have turned to stem cell technology. Vertex, a leader in this field, has transplanted pancreatic islet cells derived from stem cells into 12 individuals with type 1 diabetes. After one year, ten of these individuals no longer required insulin injections. The company plans to seek regulatory approval for this cell therapy next year.
In China, researchers are also developing pancreatic islet cells from reprogrammed stem cells derived from the patient’s own fat tissue, with early reports indicating success.
However, both approaches still require immunosuppressive drugs. The new CRISPR-based strategy offers a potential solution by eliminating this need.
In the new study, CRISPR gene editing was used to disable two genes that typically identify cells to T cells. Additionally, a virus was used to introduce genetic instructions for the CD47 protein into these cells. This protein acts as a protective “do not eat me” signal, preventing attacks from NK cells.
The patient received no immunosuppressive drugs and showed no immune response against the edited cells after 12 weeks. Measurements of C-peptide levels confirmed that the cells produced insulin consistently and in response to blood sugar levels.
Preliminary data from the study are highly promising for a complete cure for this autoimmune disease. However, the reproducibility of this method and its ability to fully shield edited cells from the immune system remain uncertain. Additionally, the study involved only one patient who received a low dose of cells for a short period, which was insufficient to eliminate the need for insulin injections. Thus, the clinical effectiveness of this approach has yet to be proven.
The ultimate goal is to develop stem cells into insulin-secreting pancreatic islet cells that, through gene editing, remain invisible to the immune system, eliminating the need for immunosuppressive drugs.
Original Article in NEJM:
https://www.nejm.org/doi/10.1056/NEJMoa2503822 0 reply
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