Gene Editing
Rho 1-2 Meganuclease, an Allele-Specific Gene Editing Therapy, Rejuvenates Rod Photoreceptor Structure and Function in a Pig Model of adRP
Jalligampala, A. et al. Association for Research in Vision & Ophthalmology (ARVO) 2021 Meeting.
Translation of an AAV-Delivered Gene Editing Approach for Transthyretin Amyloidosis in Animal Models
Greig, J.A. et al. American Society of Gene and Cell Therapy (ASGCT) 2021 Annual Meeting.
A Gene Editing Approach to Eliminate Hepatitis B Virus Using ARCUS Meganucleases
Gorsuch, C. et al. American Society of Gene and Cell Therapy (ASGCT) 2020 Annual Meeting.
Engineering a Self-inactivating Adeno-associated Virus (AAV) Vector for ARCUS Nuclease Delivery
Li, H. et al. American Society of Gene and Cell Therapy (ASGCT) 2020 Annual Meeting.
ARCUS Gene Editing to Eliminate MELAS-Associated m.3243A>G Mutant Mitochondrial DNA
Shoop W., et al. American Society of Gene and Cell Therapy (ASGCT) 2022 Annual Meeting.
Optimization of Hydroxyacidoxidase 1 (HAO1) Targeting ARCUS Nucleases for the Treatment of Primary Hyperoxaluria Type 1 (PH1)
Owens G., et al. American Society of Gene and Cell Therapy (ASGCT) 2022 Annual Meeting.
Targeting the Hepatitis B cccDNA with a Sequence-Specific ARCUS Nuclease to Eliminate Hepatitis B Virus In Vivo
Gorsuch, C. et al. HEP DART 2021 Annual Conference.