FRAXA Research Foundation and snapshot of recent research, with Katie Clapp, 13 July 2023 скачать в хорошем качестве

FRAXA Research Foundation and snapshot of recent research, with Katie Clapp, 13 July 2023

Presentation from “Fragile X Syndrome Research Roundup” webinar 1 of 2, 13 July 2023, by Katie Clapp, President and Co-Founder of FRAXA Research Foundation. Katie outlined the mission of FRAXA as being to find effective treatments and ultimately a cure for Fragile X syndrome, through directly funding research, tackling bottlenecks and co-funding/collaborating with partners. Katie provided a snapshot of some recent discovery work that has the potential to lead to effective treatments: (1) mRNA therapy for Fragile X syndrome – Dr Katherine Whitehead and team from Carnegie Mellon University (2) New insights into the FMR1 gene reactivation (CRISPR Cas9) – Dr Jeannie Lee and Dr Hungoo Lee from Harvard Medical School (3) The promise of Antisense Oligonucleotide (ASO) Therapy - Dr Karl Richter, University of Massachusetts Worcester and Dr Elizabeth Berry-Kravis, Rush University Medical School. Their work has resulted in the discovery that the Fragile X gene is not “shut down” in the way previously thought. Webinar AV hosting and video production by AlbaPrime. https://www.albaprime.com/