It's official - final C9orf72 CRISPR screen now live! www.sciencedirect.com/science/arti...

Arrayed dual-gRNA CRISPR screening platform for C9orf72 repeat expansion excision in patient iPSCs

An intronic hexanucleotide repeat expansion in C9orf72 is the leading genetic cause of both frontotemporal dementia and amyotrophic lateral sclerosis …

sciencedirect.com

Now live:preprint for our gRNA screening paper for C9orf72. This was a multiyear effort by Bankole Arogundade and Katie Jing Kay Lam who screened over 120 pairs(!) of gRNAs to excise the C9 repeat expansion in patient iPSCs.

Dr. Clelland's presented at DOC on state of the science for preventing dementia and using genetics as a path to cure dementia. DOC is a leading conference on longevity and wellness. doc.health/dr-claire-cl...

Dr. Claire Clelland: From Genetic Mutations to Targeted Cures - DOC

Dr. Claire Clelland of UCSF is pioneering a safer path to accelerate biologic therapies into clinical trials using genetic barcodes to target FTD, ALS, and other neurodegenerative diseases.

doc.health

Honored that Claire won the 2025 Bold Pioneer Award from WashU for her work to transform translational research! neurology.wustl.edu/clelland-sel...

We weigh risks vs. benefits in fatal diseases—waiting for perfection shouldn’t delay life-saving therapies. Our framework guides research on CRISPR off-targets and clinical decisions. Congrats to Ariella Angelini Stewart on her first, first-author publication! www.nature.com/articles/s41...

www.nature.com/articles/s42...

Opposing role of phagocytic receptors MERTK and AXL in Progranulin deficient FTD

Communications Biology - Human, mouse and iPSC-derived microglia demonstrate that interaction between GRN and phagocytic receptors can rescue (MERTK) or worsen (AXL) FTD-disease features. CSF MERTK...

nature.com

Our team is developing a platform to use physiologically maintained deceased to accelerate the discovery of delivery vectors. We anticipate this will save years in creating new gene therapies. An overview and the ethical considerations is now published in Science. www.science.org/doi/10.1126/...

Discovery research in physiologically maintained deceased

Expanded research opportunities in deceased humans require ongoing ethical inquiry

science.org

We have created an end-to-end bioinformatics pipeline to make analyzing PacBio single molecule sequencing data easier. It handles WGS and pure target. We use it for our C9orf72 gene therapy development. Well done Tanya, the talent data scientist who lead this work! academic.oup.com/bioinformati...

nf-core/pacvar: a pipeline for analyzing long-read PacBio whole genome and repeat expansion sequencing data

AbstractMotivation. Pacific Biosciences (PacBio) single-molecule, long-read sequencing enables whole genome annotation and the characterization of 20 compl

academic.oup.com

Will Elon return the billions in US grants and contracts Tesla and SpaceX receives or only block funds for hard working Americans? Is it legal to let the fox in the hen house?

We stand with our colleagues at the NIH, FDA and CDC. Promoting health and keeping our medicines safe is essential to us all and the well being of our country.

We believe in science.

This beautiful and moving story tells the story of Linde's fight to change the future of those with genetic FTD. We are working to create a CRISPR MAPT-FTD therapy and a better future for Linde and all with genetic neurodegenerative conditions. Thanks for sharing your story Linde! shorturl.at/DOK1J

A Woman With a Rare Gene Mutation Fights to Avoid Her Mother’s Fate (Gift Article)

A mutant gene is coming to steal Linde Jacobs’s mind. Can she find a way to stop it?

nytimes.com