A baby with a rare genetic disorder that’s often fatal has been cured, probably, by a newly developed targeted gene therapy.

Thanks to decades of research, gene sequencing rapidly identified KJ’s disorder.

An overnight success that was years in the making. Then there’s this:

Doctors at the Children’s Hospital of Philadelphia and University of Pennsylvania medical school then raced to design a gene-editing therapy using CRISPR technology to correct the defective gene. …
This process ordinarily take[s] years, though doctors in collaboration with several biotech firms were able to do it within weeks. The Food and Drug Administration also rushed to approve the treatment.

Then the overnight part was itself rushed. We may be seeing the effects of rushing genetic- or RNA-centered therapies with the conflicting reports of a number of the Wuhan Virus mRNA-based vaccines. We’ll learn more about the efficacy of KJ’s gene-editing treatment over the course of his lifetime and whether other genetic-related disorders develop as he ages, particularly as his body passes through the hefty disruption of its hormonal development as it grows into adulthood.

My larger concern, though, is this. This sort of gene-editing to cure genetic diseases also can be harnessed to cure “ordinary” and “normal” genetic complements in efforts to develop “better” babies—stronger, smarter, immune to this or that, or just attempts to “improve” humans.

The disease-curing/preventing advantages of gene treatment are huge and well worth pursuing. But in parallel with that, there need to be serious and draconian controls put on the techniques in order to control (that genie is out of the molecular bottle; it won’t ever be perfectly controlled) and mitigate misuses of the technique—beginning with clear definitions of “misuses” and “better.”