Recent advances in gene editing are turning what once seemed like science fiction into real treatments. One striking example is a personalized therapy developed for a baby born with a rare metabolic disorder. Researchers at Children's Hospital of Philadelphia and Penn Medicine used a base-editing version of the CRISPR-Cas9 system to correct a mutation in the CPS1 gene of a child who suffered from carbamoyl phosphate synthetase 1 deficiency. Within months of receiving the therapy, the child was able to tolerate increased protein in his diet and required fewer nitrogen-scavenging medications.
This breakthrough shows the feasibility of tailored gene-editing for ultra rare diseases, where traditional drug development isn't practical. However, as discussed in Innovative Genomics Institute's review, the case raises ethical questions about cost, accessibility, and equity in access to gene therapy.
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