Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing

Kanut Laoharawee, Russell C. DeKelver, Kelly M. Podetz-Pedersen, +13

Molecular Therapy

Engineering of Primary Human B cells with CRISPR/Cas9 Targeted Nuclease

Matthew J. Johnson, Kanut Laoharawee, Walker S. Lahr, +2

Scientific Reports

ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

Li Ou, Russell C. DeKelver, Michelle Rohde, +15

Molecular Therapy

In Vivo Gene TherapyGeneticsImmunologyDisease Gene IdentificationGenetic Medicine +20

Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System–Directed, AAV9-Mediated Iduronate Sulfatase Gene Transfer

Kanut Laoharawee, Kelly M. Podetz-Pedersen, Tam T. T. N. Nguyen, +7

Human Gene Therapy

PLX3397 treatment inhibits constitutive CSF1R-induced oncogenic ERK signaling, reduces tumor growth, and metastatic burden in osteosarcoma

Branden A. Smeester, Nicholas J. Slipek, Emily J. Pomeroy, +15

Bone

OncologyReduces Tumor GrowthOncogenic AgentMedicinePathology +5

Precision enhancement of CAR-NK cells through non-viral engineering and highly multiplexed base editing

Minjing Wang, Joshua Krueger, Alexandria K. Gilkey, +15

Journal for ImmunoTherapy of Cancer

Implication of <i>ZNF217</i> in Accelerating Tumor Development and Therapeutically Targeting ZNF217-Induced PI3K–AKT Signaling for the Treatment of Metastatic Osteosarcoma

Branden A. Smeester, Garrett M. Draper, Nicholas J. Slipek, +16

Molecular Cancer Therapeutics

Genome Engineering of Primary Human B Cells Using CRISPR/Cas9

Kanut Laoharawee, Matthew J. Johnson, Walker S. Lahr, +3

Journal of Visualized Experiments

Multiplex Prime Editing and PASSIGE TM for Non-Viral Generation of an Allogeneic CAR-T Cell Product

Emily J. Pomeroy, Andrew V. Anzalone, Christopher J. Podracky, +10

Blood

Correction of Fanconi Anemia Mutations Using Digital Genome Engineering

Christopher J. Sipe, Mitchell G. Kluesner, Samuel P. Bingea, +14

International Journal of Molecular Sciences