38 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. CRISPR/Cas9-Mediated Genome Engineering of Primary Human B Cells
10. Supplementary Data from Implication of ZNF217 in Accelerating Tumor Development and Therapeutically Targeting ZNF217-Induced PI3K–AKT Signaling for the Treatment of Metastatic Osteosarcoma
11. Data from Implication of ZNF217 in Accelerating Tumor Development and Therapeutically Targeting ZNF217-Induced PI3K–AKT Signaling for the Treatment of Metastatic Osteosarcoma
12. Table S2 from Implication of ZNF217 in Accelerating Tumor Development and Therapeutically Targeting ZNF217-Induced PI3K–AKT Signaling for the Treatment of Metastatic Osteosarcoma
13. Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS After the Development of Cognitive Dysfunction
14. Engineering of Primary Human B cells with CRISPR/Cas9 Targeted Nuclease
15. A Pan-RNase Inhibitor Enabling CRISPR-mRNA Platforms for Engineering of Primary Human Monocytes
16. Correction of Fanconi Anemia Mutations Using Digital Genome Engineering
17. Multiplex Prime Editing and PASSIGE TM for Non-Viral Generation of an Allogeneic CAR-T Cell Product
18. Implication of ZNF217 in Accelerating Tumor Development and Therapeutically Targeting ZNF217-Induced PI3K–AKT Signaling for the Treatment of Metastatic Osteosarcoma
19. Genome Engineering of Primary Human B Cells Using CRISPR/Cas9
20. Engineering of Primary Human B cells with CRISPR/Cas9 Targeted Nuclease
21. ZFN-mediated in vivo genome editing of hepatocytes results in phenotypic correction in murine MPS I and MPS II models
22. Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System–Directed, AAV9-Mediated Iduronate Sulfatase Gene Transfer
23. ZFN-mediated in vivo genome editing results in phenotypic correction in murine MPS I and MPS II models
24. Neurologic improvement in a mouse model of Hunter syndrome (mucopolysaccharidosis type II) by treatment with AAV9 vector after the development of cognitive dysfunction
25. Relative effectiveness of different routes of AAV administration for gene therapy of mucopolysaccharidosis
26. 485. ZFN-Mediated Liver-Targeting Gene Therapy Corrects Systemic and Neurological Disease of Mucopolysaccharidosis Type I
27. 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
28. 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)
29. AAV9 mediated correction of iduronate-2-sulfatase deficiency in the central nervous system of mucopolysaccharidosis type II mice
30. ZFN-mediated in vivo genome editing results in supraphysiological levels of human iduronate 2-sulfatase and phenotypic correction in a murine MPS II model
31. ZFN-mediated correction of murine MPS I model by expression of the human IDUA cDNA from the albumin “safe harbor” locus
32. Multiplex Prime Editing and PASSIGE TMfor Non-Viral Generation of an Allogeneic CAR-T Cell Product
33. 369. AAV9 Mediated Correction of Iduronate-2-Sulfatase Deficiency in the Central Nervous System of Mucopolysaccharidosis Type II Mice
34. 479. ZFN-Mediated In Vivo Genome Editing Results in Supraphysiological Levels of Lysosomal Enzymes Deficient in Hunter and Hurler Syndrome and Gaucher Disease
35. ZFN-Mediated In VivoGenome Editing Corrects Murine Hurler Syndrome
36. Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In VivoGenome Editing
37. Engineering Memory T Cells as a platform for Long-Term Enzyme Replacement Therapy in Lysosomal Storage Disorders.
38. Precision Enhancement of CAR-NK Cells through Non-Viral Engineering and Highly Multiplexed Base Editing.
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