
Scientists Examine CRISPR as a Therapeutic Approach for Treating Blood Disorders
A new study reveals a potential new approach to treat sickle cell disease and beta thalassemia using CRISPR-Cas9 gene-editing technology.
A group of scientists have developed a potential new approach to treat common blood disorders by using CRISPR-Cas9 gene-editing technology. In a new
In the Nature
The use of CRISPR-Cas9 provides a potentially viable option for
“Several techniques have emerged in recent years that use gene editing to manipulate hematopoietic stem cells for the treatment of blood disorders. Some of them use artificial restriction enzymes like zinc finger nucleases, another method exploits structural changes that can be introduced into chromatin, and others, including our method, involve CRISPR gene editing,” study author Mitchell J. Weiss, MD, PhD, chair of the Hematology Department at St. Jude told BioPharm International. “Each of these approaches is different and none have been tested in clinical trials in humans. Our approach uses CRISPR-Cas9 to disrupt the mutated genes and restore fetal hemoglobin levels in hematopoietic stem cells. It remains to be established which is the best and safest approach to gene editing for patients with sickle cell disease.”
According to Weiss, some other methods that may prove effective for treating blood disorders include the correction of sickle cell-associated mutations by using
“Our study is a proof-of-principle that our gene-editing approach can be successful in isolated blood-forming stem cells. Further work is needed to improve the gene editing process and to ensure that no off-target mutations arise with this approach,” Weiss continued. “More research is definitely needed before moving this work towards the clinic. What would be beneficial is a head-to-head comparison of the merits of each of the gene editing processes that have been proposed to date. It is likely to be some time before any of these approaches are tested in a clinical trial but the potential is clearly there.”
The use of CRISPR-Cas9 technology has been a highly debated topic in the scientific community as
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