Wan Hee Yoon

Proud to share our new publication in The American Journal of Human Genetics, establishing NRDC as an essential gene for early human brain development and providing long-awaited molecular diagnoses for affected families! Thank you for all contributors!
www.cell.com/ajhg/fulltex...

Rare genetic disorder treated in womb for the first time The child, who is now almost three years old, shows no signs of the often fatal motor neuron disease.

Rare genetic disorder treated in womb for the first time

www.nature.com/articles/d41...

Outstanding discovery! Unveiling a novel role of the mitochondrial solute carrier SLC25A38 in mitochondrial pyridoxal 5'-phosphate (Vitamin B6) accumulation, identified through a CRISPR/Cas9-mediated screen. Great work!

CRISPR genome-editing grows up: advanced therapies head for the clinic Gene-editing technologies for cancer and blood disorders are maturing a little more than a year after the first CRISPR drug was approved.

CRISPR treatments helped people with either of two heritable blood disorders, sickle-cell disease and β-thalassaemia!
www.nature.com/articles/d41...

Accelerating Research and Driving Rare Disease Breakthroughs Citizen Health and CZI partner to empower rare disease patients, accelerate clinical trial launches, and advance drug development.

Rare diseases are not rare; There is the urgent need for innovative solutions!
chanzuckerberg.com/newsroom/cit...

I am honored to receive this prestigious award in honor of Dr. Neufeld, with Dr. Tamar Harel. A heartfelt thank you to
US-Israel Binational Science Foundation.

Dr. Hugo Bellen is coming to OMRF and will present his exciting research on Nov. 14!

Congratulations to Jim on his Lifetime Achievement Award! He is an great scientist and mentor, someone I truly aspire to follow in the footsteps of.