Improving blood-brain barrier penetration in Hurler syndrome using an IDUA-ApoE fusion enzyme delivered via the PS Gene Editing SystemMOLECULAR GENETICS AND METABOLISMIF3.52025-02-010PRE
Penetrating the blood-brain barrier: Utilizing the PS gene editing system to encode a novel fusion β-galactosidase for the treatment of GM1 gangliosidosisMOLECULAR GENETICS AND METABOLISMIF3.52025-02-010PRE
PS Gene-editing system corrects the CNS with blood-brain barrier penetrant ApoE-enzymesMOLECULAR GENETICS AND METABOLISMIF3.52025-02-010PRE
Optimal stoichiometry of CRISPR-Cas9 components in the PS Gene-editing SystemMOLECULAR GENETICS AND METABOLISMIF3.52024-02-010PRE
Treating murine Hurler syndrome utilizing small-activating RNA following bone marrow transplantMOLECULAR GENETICS AND METABOLISMIF3.52023-02-010PRE
Drugging transcription factors with small activating RNAs: A novel approach for enhancing bone marrow therapy for monogenic rare diseasesMOLECULAR GENETICS AND METABOLISMIF3.52023-02-010PRE
Prevention of murine GM1-gangliosidosis following heterotopic insertion of Glb1 using gene editingMOLECULAR GENETICS AND METABOLISMIF3.52022-02-010PRE
PS gene editing with a novel HEXO construct to treat both Tay-Sachs and Sandhoff diseasesMOLECULAR GENETICS AND METABOLISMIF3.52021-02-010PREAI
Examination of a blood-brain barrier targeting β-galactosidase-monoclonal antibody fusion protein in a murine model of GM1-gangliosidosisMOLECULAR GENETICS AND METABOLISMIF3.52021-02-010PREAI
Immunogenicity, genotoxicity, and efficacy of PS gene editing in treating MPS I miceMOLECULAR GENETICS AND METABOLISMIF3.52021-02-010PREAI
A Highly Efficacious PS Gene Editing System Corrects Metabolic and Neurological Complications of Mucopolysaccharidosis Type IMOLECULAR THERAPYIF122020-06-0147OAAI
Correction of both Tay-Sachs and Sandhoff diseases with the PS gene editing systemMOLECULAR GENETICS AND METABOLISMIF3.52020-02-010PREAI
Gene editing to treat GM1-gangliosidosis by heterotopic insertion of GLB1 in a murine modelMOLECULAR GENETICS AND METABOLISMIF3.52020-02-010PREAI
SAAMP 3.0© : Computational biology to predict the phenotype of a missense mutation for lysosomal diseasesMOLECULAR GENETICS AND METABOLISMIF3.52020-02-010PREAI
Liver-targeting gene editing achieves significant neurological benefits in MPS I miceMOLECULAR GENETICS AND METABOLISMIF3.52020-02-010PREAI
Metabolomics profiling reveals profound metabolic impairments in mice and patients with Sandhoff diseaseMOLECULAR GENETICS AND METABOLISMIF3.52019-02-0115OAAI
Comprehensive behavioral and biochemical outcomes of novel murine models of GM1-gangliosidosis and Morquio syndrome type BMOLECULAR GENETICS AND METABOLISMIF3.52019-02-0129OAAI
ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler SyndromeMOLECULAR THERAPYIF122019-01-0172OAAI
Proteomics and interactomics analysis of brain samples from MPS mice and patientsMOLECULAR GENETICS AND METABOLISMIF3.52018-02-010PREAI
Fetal accumulation of glycosaminoglycan in a mouse model of Hurler syndromeMOLECULAR GENETICS AND METABOLISMIF3.52018-02-010PREAI
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