First CRISPR Trials on Humans Are Now Being Implemented in the US

Table of Contents

1 Case study
2 The therapy might not work
3 CRISPR could edit the wrong genes
4 Conclusion
- - 4.0.1 References

NPR reported that the first trials of CRISPR in human beings have begun in the U.S. There are two patients who are currently getting treatment as part of a study by the University of Pennsylvania. According to NPR, the patients have a form of cancer that is difficult to treat and they have both relapsed even after regular treatments. The trial involves researchers taking immune cells from the bodies of patients. They then edit them using CRISPR and later put them back in. DNA Editing

This is in hope that the edited cells will be better when it comes to identifying the cancer cells and attacking them not the unaltered cells. The researchers hope to enroll 18 more people in their study. However, according to Jon Fingas, it is not assured that they will get approval for that many subjects.

Case study

It will not be possible to get the results of the study or the progress of the enrolled patients until an uncertain time in the future. Other trials have been planned in the U.S. and Canada. The World Health Organisation is dealing with ethics about human gene editing of any kind.

Altering specific kinds of cells using CRISPR in humans is not like editing germline DNA using CRISPR. This is what was done by scientists in China to twins Lulu and Nana last year. It is clear that we are not ready to carry out the editing of germline DNA normally passed down to future generations. However, a therapeutic application that is happening in this new trial is a huge deal.

Some of the things that could go wrong are:

The therapy might not work

Technically, this is the first time that CRISPR is being used in human patients. This is according to John Doench who is a Broad Institute geneticist and an expert in gene editing. He said that CAR-T cells are engineered immune cells made using CRISPR. They are considered better when it comes to attacking tumors than the regular immune cells.

Doech said that it was important to note that CAR-T cells are also a technology by themselves. It means that if the therapy does not work for the patients, it may not be related to CRISPR. CAR-T is very effective in some patients but not effective in others. They both have a long way to go and it is a possibility that they will not work.

CRISPR could edit the wrong genes

Another worrying thing about the treatment is off-target edits. CRISPR guides enzymes to the spot on DNA strands scientists intend to alter. Other genes could end up getting edited if the CRISPR enzymes end up making changes in additional spots.

Conclusion

There are a lot of potentials to create better T-cell defenses in gene editing. If it ends up being safe, it could turn out to be a huge advancement for the treatment of cancer.