In past years, there has been a spike in interest in gene therapies as certain technologies move towards being commercially viable. In some cases, therapies are being developed to help individuals with genetic or acquired disabilities, but, to date, no gene-editing technique has attracted as much attention in scientific circles as CRISPR.
This gene editing technique has been touted as revolutionary because of how easily it allows researchers to change the code of cells. Functionally, this means that scientists can develop certain treatments and therapies much more quickly, because the process of actually editing genes can go much more quickly.
What is CRISPR?
CRISPR-Cas9 (known colloquially as just CRISPR) is a gene editing technique. This technique stems from the discovery of CRISPRs (Clustered Regularly Interspaced Short Palindromic Repeats), which can be found in bacteria. The system was discovered by a team of researchers headed by Jennifer Doudna. It allows researchers to edit DNA in very precise locations. The end result of this technology is that researchers can permanently modify genes in living cells and organisms. There is also a simpler technique, developed in 2015 by Feng Zhang that uses a slightly different system, CRISPR-Cpf1.
Why is CRISPR Important?
There are several reasons why CRISPR has attracted a great deal of attention. First, it allows researchers to precisely identify gene function. This allows them to more quickly and easily develop models of disease and create therapies for them. While such therapies are years, or even decades, away, this development still greatly accelerates the rate at which gene research can progress. This system is cheap, quick and easy to use.
What Makes CRISPR Important in the Context of Birth Injury Research?
Much research into the mechanisms of birth injury has gone to the study of how to help children with birth injuries using stem cell therapies – a field in medicine that is still in its infancy but has been garnering a great deal of attention in the media recently, especially with the rising trend of cord blood banking. Stem cell therapy research has had mixed results in clinical trials, but such therapies offer hope to families with children who have disabilities, who believe that the procedure can help their children’s symptoms.
In some cases, stem cell therapy has helped adults with traumatic brain injuries regain motor and/or cognitive function, and clinical trials are currently underway. However, the brain structure and function of adults and newborns can differ significantly, so this is a development that must be approached with cautious optimism.
Much of the media attention surrounding this gene editing technique focuses on the speed and low cost of research. The technique is, in the end, a tool – it increases the speed of research and allows researchers to successfully conduct gene editing, an important part of stem cell therapies.
Disabilities such as hypoxic ischemic encephalopathy (HIE) and cerebral palsy are not generally genetic. Gene therapies, however, have, in some cases, seemed to improve functional outcomes in cerebral palsy and HIE, though more rigorous testing is needed to fully understanding when, how, and to what extent gene therapies are effective.
Read more about CRISPR:
- CRISPR: How does it work?
- Why is CRISPR so important?
- CRISPR: A primer
- Advances in CRISPR research
- CRISPR helps improve Duchenne’s muscular dystrophy symptoms
- CRISPR: How it revolutionizes research
Read more about Birth Injury and Gene Therapy
- [Infographic]: CRISPR: Cost and Applications
- ABC Law Centers: HIE Treatment and Stem Cell Therapy
- ABC Law Centers: Local boy enrolled in experimental cerebral palsy treatment trial
- Stem Cell Therapy for Neonatal Diseases Associated with Preterm Birth
- Could Cord Blood Cell Therapy Reduce Preterm Brain Injury?
- Stem Cells and Cerebral Palsy