C9orf72 repeat expansions are difficult to measure. This paper describes a protocol utilizing the CRISPR/Cas9 system and PacBio single-molecule sequencing to accurately sequence repeat expansions without amplification, overcoming the limitations of traditional methods.
It takes a village of patients, families, scientists and clinicians to get new and groundbreaking therapies to clinical trial. Thank you for creating this village with us!
Big thanks to @hoperhenderson for the fantastic write-up! 🙌🏼 Congrats to the Clelland Lab for the groundbreaking work! 🎉 #C9orf72#CRISPR
https://t.co/Cyca2mPrES
CRISPR gene therapy for the brain and spinal cord: What is it, why isn't it a reality yet and what needs to be done to bring it to the clinic. Check out our latest @JAMANeuro review article:https://t.co/z9icOuzy93
Our perspective on CRISPR for the CNS was just published @JAMANeuro . Find it here:https://t.co/z9icOuzy93. Congrats to Dr. Salomonsson on such a well-written and easy to read piece!
Our latest research on creating validated methods for neurodegenerative research, #C9orf72, CRISPR outcomes is now available @SciReports . Read here: https://t.co/4ZBtM2BjFC
Our latest research on #C9orf72 human DNA, RNA, protein quantification methods is published: https://t.co/4ZBtM2BjFC. Well done to the team of talents post-docs and post-baccs who made this work possible and special thanks to @PacBio for their collaboration.
Thank you @Jeanc9orf72 for sharing your experience as a C9 carrier, family member, and activist. Hearing patient stories is so important for scientists, and we really appreciate the work you are doing!