Behind gene editing breakthroughs are families who say yes

Recent headlines told a story that once was science fiction: A child received a groundbreaking in vivo gene editing treatment, a one-time infusion designed to rewrite the very DNA in their cells, potentially curing a devastating genetic condition. The science is breathtaking. But behind every clinical milestone lies a human story, one that deserves equal recognition: the extraordinary courage of the families who make these moments possible.

It’s easy to be captivated by the science. CRISPR-based gene editing has revolutionized medicine, allowing doctors to alter DNA inside a living body. Researchers are no longer just managing symptoms — they are rewriting the code of life, aiming for cures rather than treatments. But none of this would be possible without the families who bravely choose to enter the unknown.

When a parent gives consent for their child to receive an experimental gene therapy, they are stepping into uncharted territory. The data is limited. The risks are real. Often, the disease their child faces is relentless, with few or no treatment options. In these moments, families are not making decisions in the comfort of certainty, but in the fog of hope — hope that this new treatment might be the miracle their child needs, hope that their participation will help others down the road, even if the outcome is uncertain for their own child.

These decisions are not made lightly. They come at the end of sleepless nights, hospital stays, failed therapies, and devastating prognoses. They are made after hours of consultations with doctors and researchers, and often after conversations with other families in similar positions. They are acts of love — and of immense courage.

The families who say yes are often pioneers themselves. They raise funds for research when there is none. They create communities to connect with others around the globe. They build bridges between scientists, regulators, donors, and investors, trying to move the science forward not just for their own children, but for every child facing the same fate. Their bravery helps expand the boundaries of medicine. Their children become, in the most tragic but powerful sense, the torchbearers of progress.

We often hear the phrase "desperate parents" in the context of experimental medicine. But let’s reframe that. There is desperation, to be sure. But these are informed and fiercely determined parents, facing impossible odds with clarity and resolve. They are not reckless. They are rational actors choosing to participate in medical advancement when the alternative is slow or even rapid decline and suffering. They do not have the luxury of waiting decades for perfect data or long-term follow-up. They choose hope with eyes wide open.

It is critical that we honor their role — not just in emotional terms, but in practical and ethical ones. Regulators must build frameworks that recognize the urgency without compromising safety. Policymakers must ensure that compassionate access and ethical oversight go hand in hand. Researchers must be transparent and inclusive, treating families as partners, not just subjects. And the public must move past simplistic narratives of miracles or tragedies to understand the complexity of what these families live every day.

There is a long road ahead for in vivo gene editing. We do not yet know the full implications of editing human DNA within the body. There will be setbacks, and there must be safeguards. But as we rightly marvel at the scientific breakthroughs, let us also hold space for something just as remarkable: the bravery of the families who make such progress possible. They are advocates, visionaries, and heroes in a quiet, relentless fight to give their child — and countless others — a future.

In an age of precision medicine and astonishing biological tools, we remind ourselves that it is courage, more than any technology, that turns the impossible into reality.

This guest essay reflects the views of Port Washington resident Scott D. Reich, founder and chief executive of Believe in a Cure, a nonprofit seeking to develop a treatment for FOXG1 syndrome, the rare brain disease that afflicts his son, Eli.