Phase 2 Publications

Publication Title	Journal	Authors	Pub Date	PubMed	Project PIs
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
Validated assays for the quantification of C9orf72 human pathology	Scientific Reports	Salomonsson et al.	1/8/24	38191789	Doudna
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	Tsi
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
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