Author
Massachusetts General HospitalORCID 0009-0006-2228-6515Unclaimed ORCID profile · follow to get notified of new preprints
Mutual co-authors in cluster
4 papers
Recent·Most cited
Matthew Nolan, Sandeep Aryal, I. Sandra Ndayambaje…·Clotilde Lagier‐Tourenne
The paper shows statins and mevalonate pathway inhibition increase STMN2 and ATF3, promoting neuronal regeneration in neurodegenerative disease.
Christiano R. R. Alves, Sabyasachi Das, Vijai Krishnan…·Patricia Leonor Musolino
This paper develops a customized CRISPR-Cas9 base editor to correct the ACTA2 R179H mutation in a mouse model of MSMDS, improving survival and rescuing vascular and neurological phenotypes.
Paolo Pigini, Huilin Xu, Yan Ji…·Elisabetta Morini
The paper identifies 9,814 poison exons in the human genome, finds brain-specific splicing patterns, and links pathogenic variants to PE splicing affecting neurological disorders.
Shuqi Yun, Anil K. Chekuri, Jennifer Art…·Christiano R. R. Alves
This paper develops a CRISPR base editing strategy to correct the FD-causing mutation in human cells and mice, achieving up to 70% on-target correction and functional restoration.