21 results on '"Laoharawee, Kanut"'
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2. Development and testing of a versatile genome editing application reporter (V-GEAR) system
3. Generation and characterization of an immunodeficient mouse model of mucopolysaccharidosis type II
4. Non-invasive intravenous administration of AAV9 transducing iduronate sulfatase leads to global metabolic correction and prevention of neurologic deficits in a mouse model of Hunter syndrome
5. PLX3397 treatment inhibits constitutive CSF1R-induced oncogenic ERK signaling, reduces tumor growth, and metastatic burden in osteosarcoma
6. ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome
7. Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing
8. Primary B cell engineering for therapeutic research
9. Multiplex Prime Editing and PASSIGE TM for Non-Viral Generation of an Allogeneic CAR-T Cell Product
10. ZFN-mediated in vivo genome editing of hepatocytes results in phenotypic correction in murine MPS I and MPS II models
11. Neurologic improvement in a mouse model of Hunter syndrome (mucopolysaccharidosis type II) by treatment with AAV9 vector after the development of cognitive dysfunction
12. Relative effectiveness of different routes of AAV administration for gene therapy of mucopolysaccharidosis
13. ZFN-mediated in vivo genome editing results in phenotypic correction in murine MPS I and MPS II models
14. 484. In Vivo Zinc-Finger Nuclease Mediated Iduronate-2-Sulfatase (IDS) Target Gene Insertion and Correction of Metabolic Disease in a Mouse Model of Mucopolysaccharidosis Type II (MPS II)
15. 485. ZFN-Mediated Liver-Targeting Gene Therapy Corrects Systemic and Neurological Disease of Mucopolysaccharidosis Type I
16. 86. AAV9 Mediated Correction of Iduronate-2-Sulfatase Deficiency in the Central Nervous System to Prevent the Onset of Neurologic Deficits in a Murine Model of Mucopolysaccharidosis Type II
17. ZFN-mediated in vivo genome editing results in supraphysiological levels of human iduronate 2-sulfatase and phenotypic correction in a murine MPS II model
18. ZFN-mediated correction of murine MPS I model by expression of the human IDUA cDNA from the albumin “safe harbor” locus
19. AAV9 mediated correction of iduronate-2-sulfatase deficiency in the central nervous system of mucopolysaccharidosis type II mice
20. 479. ZFN-Mediated In Vivo Genome Editing Results in Supraphysiological Levels of Lysosomal Enzymes Deficient in Hunter and Hurler Syndrome and Gaucher Disease
21. 369. AAV9 Mediated Correction of Iduronate-2-Sulfatase Deficiency in the Central Nervous System of Mucopolysaccharidosis Type II Mice
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