Cure Mito Foundation

A baby boy with a devastating genetic disease is thriving after becoming the first known person to receive a bespoke, CRISPR therapy-for-one, designed to correct his specific disease-causing mutation. https://lnkd.in/dP3gxpAR

Your choices today can bring hope and change for families. Do things that matter. #CureMito #Leighsyndrome #mitochondrialdisease

🎉 We’ve reached 3,000 followers on LinkedIn - thank you for being part of our community! Help us keep growing - follow us to stay connected and support progress for Leigh syndrome and mitochondrial diseases. 💚 #CureMito #LeighSyndrome #MitochondrialDisease

At the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), Sarah Holbrook, PhD will present a poster titled “Investigating allotropic expression and mitochondria-targeted base editors as therapeutic strategies to alleviate MT-ATP6-associated Leigh syndrome.” #LeighSyndrome #ASGCT #RareDiseaseResearch #GeneTherapy

Happy Mother’s Day to the moms who are grieving, the moms doing their best even when it’s hard, the moms who have lost their precious children but continue to fight for others, and the moms holding hope through the hardest days. To the moms navigating life with rare disease, and to those carrying the memories of the babies they held or only dreamed of holding — we see you, we honor you, and we walk beside you. Today, and every day, your love and strength inspire us. 💚 #MothersDay #CureMito #LeighSyndrome #RareDiseaseMoms #StrengthAndLove #YouAreNotAlone

The American Society of Gene & Cell Therapy (ASGCT) will host its 28th Annual Meeting in New Orleans. Dr. Qinglan Ling, who has been dedicated to advancing SURF1 gene therapy, will present a talk titled “Achieving ‘Just Right’: Preclinical Development of a Gene Therapy for SURF1 Leigh Syndrome Using Two Disease Models” on Tuesday, May 13th. We are proud to recognize and celebrate this important work. #leighsyndrome #mitochondrialdisease

📢 New study by Nijim et al. (JAMA Netw Open, 2025) highlights key factors behind successful rare disease drug repurposing efforts, offering a roadmap for nonprofits, collaborators, and policymakers to drive impact through systematic, data-driven approaches. https://lnkd.in/e_9sYvWs #RareDisease #DrugRepurposing #PatientDrivenResearch #CureMito

🚨 We’re proud to announce a $202,000 grant to Dr. Steven Gray's lab at UT Southwestern Medical Center to support the continued development of SURF1 gene replacement therapy. Since 2018, we’ve partnered closely with the Gray Lab, contributing over $1 million to this promising research. While more funding is still needed to move this therapy toward clinical trials, we remain deeply committed to advancing treatment options for the Leigh syndrome community. #CureMito #LeighSyndrome #GeneTherapy #MitochondrialDisease #SURF1 #RareDiseaseResearch

On May 22, we will participate in a virtual public workshop on “Primary Mitochondrial Diseases” hosted by the Reagan-Udall Foundation for the FDA in collaboration with the FDA. We’re honored to share our community’s perspective in this important conversation. Register here: https://lnkd.in/gji-GVhG #MitochondrialDisease #LeighSyndrome

We are proud to award a $34,000 grant to Dr. Ibrahim Elsharkawi at the Icahn School of Medicine at Mount Sinai for his study, “Investigating the Role of the Gut Microbiome in Primary Mitochondrial Disease.” More details to come soon! #mitochondrialdisease #leighsyndrome