What is CRISPR?

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Is This a Gene Therapy or Cell Therapy?

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A confusing topic for the layperson is what is CRISPR. CRISPR stands for clustered regularly interspaced short palindromic repeats. People have raised concerns about this technology, especially in light of the recent claim that a Chinese scientist used CRISPR to genetically re-engineer human embryos. To read the article of CRISPR from the Science website, click here.

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Two common questions from laypeople are: (1) What is CRISPR and why are people so excited and concerned about it?; (2) Is CRISPR a gene therapy or a cell therapy? The answer to the first question is that CRISPR is a genetic tool that allows the ability to make reasonably precise edits (changes) within human chromosomes to affect permanent genetic changes? The interest in CRISPR lies in the fact that gene mutations may be corrected from those that suffer from genetic diseases. The ethical controversy of CRISPR depends on what target cell it is applied to. If applied to a human embryo, it's ethically controversial. Under some conditions, if it's applied to a human cell outside the body, it's not ethically controversial and has scientific value. However, even under this condition, there are some moral and medical concerns (an explanation will be posted later).

 

The answer to the second question is that CRISPR can be used for either a gene therapy or a cell therapy. It can be used as a gene therapy in which it is administered directly into a patient with a genetic disease with the rationale to correct the genetic defect at the cellular level. CRISPR can also be used as a tool to modify a stem cell or other human cells artificially outside of the body to improve the safety and efficacy of a cell therapy. Once the genetic modification is complete, the cell can be administered to a patient as a cell therapy.