CRISPR is setting the benchmark in scientific discovery and treatment for various incurable diseases. Recently the University of Pennsylvania has successfully conducted a trial on two patients suffering from multiple myeloma and sarcoma. The scientist used gene editing tool CRISPR to engineer the T cells isolated from blood with a cell surface receptor commonly found in cancer.
What is CRISPR?
CRISPR stands for “Clusters of Regularly Interspaced Short Palindromic Repeats”. These are the specific region on DNA containing repeating units of nucleotide sequence and spacers. The spacers are the region of DNA with small units of DNA interspersed between repeated nucleotide sequences. CRISPR has revolutionised the way genetic engineering can make scientist edit the DNA in the easiest way. CRISPR can be used for a wide variety of cure such as repairing any inborn genetic defect or genetically modifying human cells as immunotherapies.
CRISPR in Immunotherapy
Scientist – Edward Stadtmauer explained how CRISPR can be a strategic tool in immunotherapy. He explained the process of using CRISPR to transform T cells and treat multiple myeloma and sarcoma in patients. The method involves the following steps –
• T-cells from the blood were filtered properly to get pure T-cells.
• The filtered T-cells were then subjected for Gene Editing.
• The T-cell receptors such as TCRα, TCRβ, and PD-1 were edited.
• These receptors on T-cells were removed using Gene editing.
• Once T cell receptors were removed Lenti Viral Vector was used to insert NY-ESO-1.
• The NY-ESO-1 receptor is characteristic receptor present on cancer cells.
• The modified cells were then cultured for a few weeks under lab conditions.
• Once the patient receives the chemotherapy the cells are infused back
It was observed that there was a significant improvement in the condition of both the patients suffering from multiple myeloma and sarcoma.
The CRISPR based gene editing is transforming the way science can solve problems of diseases which were once incurable or hard to cure. The scientists are continuously exploring the applicability of the CRISPR technique wherein the cells can be altered under ex vivo conditions and then can be infused back into patients. This can help in treating deadly diseases such as thalassemia and sickle cell anaemia.
Reference:
https://www.the-scientist.com/news-opinion/two-patients-treated-with-crispred-cells-in-immunotherapy-trial-65744
