Phase 2 Publications

Publication Title	Journal	Authors	Pub Date	PubMed	Project PIs
Zfp106 binds to G-quadruplex RNAs and inhibits RAN translation and formation of RNA foci caused by G4C2 repeats	Proceedings of the National Academy of Sciences of the United States of America	Celona et al.	7/23/24	39042693	Doudna
Interferon-γ induces combined pyroptotic angiopathy and APOL1 expression in human kidney disease	Cell Reports	Juliar et al.	6/25/24	38838223	Freedman
Selective haematological cancer eradication with preserved haematopoiesis	Nature	Garaude et al.	5/22/24	38778101	Tsai
Recommendations on fit-for-purpose criteria to establish quality management for microphysiological systems and for monitoring their reproducibility	Stem cell reports	Pamies et al.	5/14/24	38670111	Freedman
Rapid measurement of hemoglobin-oxygen dissociation by leveraging Bohr effect and Soret band bathochromic shift	The Analyst	Sekyonda et al.	4/29/24	38501195	Giannikopoulos, Gurkan
Reversal of C9orf72 mutation-induced transcriptional dysregulation and pathology in cultured human neurons by allele-specific excision	Proceedings of the National Academy of Sciences of the United States of America	Sachdev et al.	4/15/24	38621131	Doudna
Genetics of cystogenesis in base-edited human organoids reveal therapeutic strategies for polycystic kidney disease	Cell Stem Cell	Vishy et al.	4/4/24	38579684	Freedman
Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy	Nature Biomedical Engineering	Alves et al.	3/8/24	38057426	Tsai
Building CRISPR Gene Therapies for the Central Nervous System: A Review	JAMA Neurology	Salomonsson & Clelland	3/1/24	38285472	Doudna
Phage-assisted evolution of highly active cytosine base editors with enhanced selectivity and minimal sequence context preference	Nature Communications	Zhang et al.	2/24/24	38402281	Lutz, Arbab, Grey, Liu, Mouro Pinto
Validated assays for the quantification of C9orf72 human pathology	Scientific Reports	Salomonsson et al.	1/8/24	38191789	Doudna
Efficient in vivo prime editing corrects the most frequent phenylketonuria variant, associated with high unmet medical need	American Journal of Human Genetics	Brooks et al.	11/3/23	37924808	Peranteau, Musunuru
A base editing strategy using mRNA-LNPs for in vivo correction of the most frequent phenylketonuria variant	HGG Advances	Brooks et al.	10/31/23	37922902	Peranteau, Musunuru
Lipid Nanoparticles Deliver mRNA to the Brain after an Intracerebral Injection	Biochemistry	Tuma et al.	9/20/23	37729550	Doudna | Bankiewicz, Murthy
Rapid and definitive treatment of phenylketonuria in variant-humanized mice with corrective editing	Nature Communications	Brooks et al.	6/10/23	37301931	Peranteau, Musunuru