How CRISPR saved a baby’s life: a story of how science can move at unprecedented speed when lives are on the line. https://lnkd.in/gBjcvGwx In August 2024, a baby was born with a genetic mutation known to cause fatal liver disease within just a few months. But thanks to decades of foundational research, rapid collaboration across scientific disciplines, and cutting-edge gene editing technology, this child’s life may have been saved—perhaps even cured. Here's is the timeline from Diagnosis to Therapy: ~1 week old: Doctors diagnosed the baby with a lethal liver condition caused by a rare genetic mutation. ~1 month old: Scientists engineered a cell line carrying the same mutation to test whether CRISPR could correct it. Scientists screened guide RNAs and base editors to find the most effective combinations. ~2 months old: The team conducted toxicology studies in monkeys to ensure the safety of the CRISPR tools—specifically the mRNA-based base editor, guide RNAs, and lipid nanoparticle delivery systems. ~5 months old: Scientists created Transgenic mice carrying the same genetic mutation to test the CRISPR therapy in living organism. The results were promising as they showed the mutation was corrected in ~40% of liver cells. ~6 months old: Additional safety studies in mice showed no major side effects, giving confidence to move forward. ~6.5 months old: The research team submitted a request for regulatory approval. Remarkably, just one week later, the FDA granted approval. ~7 months old: The baby received the first dose of the CRISPR-based therapy. Despite simultaneous viral infections, there were early signs of improvement. ~8 months old: A second dose was administered. The baby showed further improvement, though longer-term follow-up will be essential to assess safety and effectiveness. This work was published yesterday in NEJM: https://lnkd.in/gg93Xczm The work was funded by the National Institutes of Health and other organizations. Two key lessons from this story: 1. Science saves lives—but it builds on decades of discovery. CRISPR gene editing has its roots in basic research conducted over 30 years ago. What began as curiosity-driven science has evolved—thanks to the work of hundreds of researchers—into a powerful therapeutic tool. Breakthroughs like these are only possible because of sustained investment in basic and translational research. 2. Animal models are absolutely essential in developing and testing therapies. Transgenic mice and studies in higher mammals were crucial in assessing whether the therapy could work—and whether it was safe to try in a human. Transgenic mice and animal studies remain indispensable for ensuring new treatments are both effective and safe before being used in patients.

Many of us, especially those who left our home countries to pursue bigger dreams, face many struggles and roadblocks in our careers. We often hide these…

We discuss the potential consequences and ethical concerns of polygenic genome editing of human embryos to alter specific variants associated with polygenic diseases, highlighting the possibility of r...

CRISPR genome engineering and prokaryotic defense systems.

Meet the Legend Lunch     Sign Up The 1st International SynBYSS Conference will unite in-person a previously online-only...

Is there a formula for a competitive NIH grant application? The Serenity Prayer may provide one: "Grant me the serenity to accept the things I cann...

Now, scientists and researchers can more easily track conversation around their research.

Labs around the world are trying to turn cells into autobiographers, tracking their own development from embryos to adults.

Recent advances in genome engineering are enabling the recording of cellular histories into genomes, with single-cell and spatial omics technologies enabling their reconstruction into cellular lineage...

A compound that enhances homology-directed repair in CRISPR editing leads to genome instability.

By developing a long-read sequencing method to simultaneously map replication status and protein-DNA contacts in cells, Ostrowski, Yang, et al. show that newly replicated chromatin is enriched for unw...