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A Genetic Hail Mary That Ended in Loss

When the first patient is also the last, what do we learn?

GENEBRIEF

Welcome to GeneBrief — your fast, clear guide to gene editing and biotech.

Here’s what we’re decoding in this issue:

🧬The man who risked everything on a one-of-one CRISPR therapy

😡Hamsters turned into rage-mutants by accident

💰The $330,000 burger that sparked a food revolution

🔍DEEP DIVE

A Cure Meant for One

If a gene edit could save your life… but had never been tested on anyone else… would you still say yes?

In 2022, 27-year-old Terry Horgan became the first person to receive a personalized CRISPR therapy built just for him. He had Duchenne muscular dystrophy, a brutal, incurable disease that wastes muscle away. His brother, Richard, wasn’t ready to lose him.

Richard founded Cure Rare Disease, partnered with Yale researchers, and helped develop an experimental therapy using CRISPR to activate backup copies of dystrophin, the gene Terry's body desperately needed.

It was approved by the FDA after 3 years of development as an n-of-1 trial, the first of its kind. But just days after receiving it, Terry’s condition worsened. He went into rapid respiratory failure and passed away.

He never had a chance to show whether the CRISPR worked.

Later reports suggested the likely cause wasn’t the gene-editing tech, but the vector. The high dose of AAV (adeno-associated virus) used to deliver the therapy may have triggered a lethal immune response. Terry’s muscles were already fragile. His body couldn’t recover.

Terry tried. The therapy failed him, but his courage didn't.

QUICK CUTS

Scientists Made Rage-Mutant Hamsters

In 2022, researchers knocked out Avpr1a, a vasopressin receptor tied to social behavior. The hamsters became hyper-aggressive: more fighting, more biting, more dominance displays. Genes don't act alone. One change can ripple across the whole system.

The $330,000 Burger

In 2013, Dutch scientists grew beef from cow stem cells and cooked it live on stage. It tasted like meat, because it was meat, just without the animal. That stunt launched an industry. Lab-grown chicken is now on some menus in the U.S. and Singapore.

🧠 BRIEF BYTE

Your gut is home to over 100 trillion microbes, vastly outnumbering your human cells.

Nature, 2022

YOUR TURN


If a therapy had never been tested in humans, but it was your only shot — would you still take it?

Featured Take

Is there any future where germline editing is justified, or is consent the line we can never cross?

“No one consented to being born with a genetic disease either. If we can prevent suffering before it starts, isn't that a form of care, not control?”

— Lauren F

Hit reply. Sharpest take gets featured next week.
Mario