CRISPR is an enzyme developed by scientists in an effort to remove potentially harmful genetic traits and paste the remaining sequences together. During the 1980s, researchers discovered that bacteria protect themselves by cutting and pasting themselves. When bacteria identify viral RNA molecules within its DNA, the Cas enzyme grabs the anomaly and divides it in two to prevent reproduction. Scientists called the act clustered regularly interspaced short palindromic repeats or CRISPR for short. However, as researchers attempted to replicate the process in the laboratory, they uncovered problems.
The CRISPR discovery gave researchers hope that they had uncovered an innovative means of editing DNA. If scientists could use the technique, they dreamed that the abnormal DNA sequences that cause a number of disease processes might be effectively removed to prevent their development. However, as research and experimentation continued, CRISPR caused gaps in the cell’s genome. Scientists from the Wellcome Sanger Institute in Great Britain published the finding in an issue of “Nature Biotechnology” explaining that potential drawbacks of CRISPR have not been formally noted.
In a laboratory setting, researchers used the Cas-9 enzyme to remove a specific gene in the embryonic stem cells of a mouse. However, the process did not go as predicted. The group soon found that large sequences of DNA were missing. If the scientists were using traditional methods of assessing the DNA sequences, they may not have discovered the error. The billions of altered cells could have devastating effects that might include making them susceptible to cancer development.
The study showed that researchers need to assess larger strands of DNA to determine why CRISPR did not perform as hoped. Instead of cutting one strand of the gene, the team cut both strands of the double helix, which was one of the reasons for the error. They also need to find ways to prevent the cellular DNA from attempting to repair itself, which led to the problem.
Representatives of companies using CRISPR reported that their scientists had not encountered the problem with the techniques they used. As such, the study did not make an impact on their research. However, the published findings caused an immediate and substantial financial loss for several CRISPR companies.
The drawbacks did not dismay the Wellcome Sanger scientists. They explain that new discoveries are not without problems. The group plans to continue working on the project and making improvements so that one day the technology might be a successful medical tool.