Long-term HSC gene therapy in mucopolysaccharidosis type IIIB mice corrects disease with no evidence of insertional mutagenesis despite high vector copy numbersMOLECULAR GENETICS AND METABOLISMIF3.52025-02-010PRE
Systemic immune challenge exacerbates neurodegeneration in a model of neurological lysosomal diseaseEMBO MOLECULAR MEDICINEIF8.32024-06-180OAAI
Systemic inflammation leads to neuronal loss and exacerbates behavioural deficits in a mouse model of MPS IIIAMOLECULAR GENETICS AND METABOLISMIF3.52024-02-010PRE
Clinical outcomes and sustained biochemical engraftment following ex-vivo autologous stem cell gene therapy for mucopolysaccharidosis type IIIAMOLECULAR GENETICS AND METABOLISMIF3.52024-02-013PRE
Sustained biochemical engraftment and early clinical outcomes following ex-vivo autologous stem cell gene therapy for mucopolysaccharidosis type IIIAMOLECULAR GENETICS AND METABOLISMIF3.52023-02-012PRE
Identification of novel peptide sequences to fuse to iduronate-2-sulfatase for the treatment of Hunter syndromeMOLECULAR GENETICS AND METABOLISMIF3.52023-02-010PRE
Validation of a GMP stem cell gene therapy manufacturing process for mucopolysaccharidosis type II (MPS II) in preparation for an approved phase I/II clinical trialMOLECULAR GENETICS AND METABOLISMIF3.52023-02-010PRE
Enhanced transduction and immunophenotyping demonstrates preclinical safety and efficacy of hematopoietic stem cell gene therapy for mucopolysaccharidosis II using an IDS.ApoEII brain targeted therapyMOLECULAR GENETICS AND METABOLISMIF3.52022-02-010PRE
Ex-vivo autologous stem cell gene therapy for MPS II (Hunter syndrome)MOLECULAR GENETICS AND METABOLISMIF3.52022-02-010PRE
Ex-vivo autologous stem cell gene therapy clinical trial for mucopolysaccharidosis type IIIA: Update on phase I/II clinical trialMOLECULAR GENETICS AND METABOLISMIF3.52021-02-012PREAI
Actinomycin D downregulates Sox2 and improves survival in preclinical models of recurrent glioblastoma放线菌素d在复发性胶质母细胞瘤的临床前模型中下调Sox2并改善生存率NEURO-ONCOLOGYIF13.42020-03-3025OAAI
Haematopoietic stem cell gene therapy with IL-1Ra rescues cognitive loss in mucopolysaccharidosis IIIAEMBO MOLECULAR MEDICINEIF8.32020-02-1439OAAI
Interleukin-1 plays a central role in behaviour abnormalities in mucopolysaccharidosis type III (MPS HI)MOLECULAR GENETICS AND METABOLISMIF3.52018-02-012PREAI
Pre-clinical safety and efficacy evaluation of GMP lentiviral vector in preparation for a clinical trial of hematopoietic stem cell gene therapy in MPS IIIAMOLECULAR GENETICS AND METABOLISMIF3.52018-02-011PREAI
Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapyBRAINIF11.72017-11-2768OAAI
Neurological correction of mucopolysaccharidosis type IIIB mice by haematopoietic stem cell gene therapyMOLECULAR GENETICS AND METABOLISMIF3.52017-01-010PREAI
Neurological Correction of Mucopolysaccharidosis IIIB Mice by Haematopoietic Stem Cell Gene TherapyMOLECULAR THERAPYIF122016-05-010OAAI
Pre-clinical workup of lentiviral mediated stem cell gene therapy for mucopolysaccharidosis type IIIAMOLECULAR GENETICS AND METABOLISMIF3.52015-02-010PREAI
Specific Retrograde Transduction of Spinal Motor Neurons using Lentiviral Vectors Targeted to Presynaptic NMJ ReceptorsMOLECULAR THERAPYIF122014-07-0119OAAI
Dose-dependent Neuroprotection of VEGF165 in Huntington's Disease StriatumMOLECULAR THERAPYIF122013-10-0120OA
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