Aiyin Liao
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View article: Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW mice
Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW mice Open
Adenosine deaminase type 2 deficiency (DADA2) is caused by bi-allelic loss-of-function mutations in ADA2 . While anti-TNF therapy is effective for the autoinflamatory and vasculitic components of the disease it does not correct marrow fail…
View article: Sustained long-term disease correction in a murine model of MPSII following stem cell gene therapy
Sustained long-term disease correction in a murine model of MPSII following stem cell gene therapy Open
View article: Haematopoietic stem cell gene therapy with <scp>IL</scp> ‐1Ra rescues cognitive loss in mucopolysaccharidosis <scp>IIIA</scp>
Haematopoietic stem cell gene therapy with <span>IL</span> ‐1Ra rescues cognitive loss in mucopolysaccharidosis <span>IIIA</span> Open
Mucopolysaccharidosis IIIA is a neuronopathic lysosomal storage disease, characterised by heparan sulphate and other substrates accumulating in the brain. Patients develop behavioural disturbances and cognitive decline, a possible conseque…
View article: Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA
Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA Open
View article: Strategies for the Induction of Immune Tolerance to Enzyme Replacement Therapy in Mucopolysaccharidosis Type I
Strategies for the Induction of Immune Tolerance to Enzyme Replacement Therapy in Mucopolysaccharidosis Type I Open
Enzyme replacement therapy with laronidase is an established treatment for Mucopolysaccharidosis type I (MPS I), but its efficacy may be limited by the development of anti-drug antibodies, which inhibit cellular uptake of the enzyme. In a …
View article: Non-myeloablative busulfan chimeric mouse models are less pro-inflammatory than head-shielded irradiation for studying immune cell interactions in brain tumours
Non-myeloablative busulfan chimeric mouse models are less pro-inflammatory than head-shielded irradiation for studying immune cell interactions in brain tumours Open
Our data suggest that non-myeloablative conditioning generates a more homeostatic peripheral inflammatory environment than head-shielded irradiation to allow a more consistent evaluation of immune cells in glioblastoma and can be used to i…
View article: Identifying tumour associated macrophages and microglia in an experimental glioblastoma model
Identifying tumour associated macrophages and microglia in an experimental glioblastoma model Open
Glioblastoma is the most aggressive primary brain cancer, with 14.6 months median survival. Tumour associated macrophages and microglia (TAMM) populate upto 40% of the tumour bulk and represent potential immunotherapy targets. Characterisi…
View article: IMMU-50. A NOVEL CHIMERIC MODEL TO ACCURATELY IDENTIFY TAMMs IN GLIOBLASTOMA
IMMU-50. A NOVEL CHIMERIC MODEL TO ACCURATELY IDENTIFY TAMMs IN GLIOBLASTOMA Open
Glioblastoma is the most aggressive primary brain cancer with poor survival, but treatment strategies to improve prognosis have failed to materialise in over 30-years. Immune cells, in particular Tumour associated macrophages and microglia…
View article: Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy
Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy Open
Mucopolysaccharidosis IIIB is a paediatric lysosomal storage disease caused by deficiency of the enzyme α-N-acetylglucosaminidase (NAGLU), involved in the degradation of the glycosaminoglycan heparan sulphate. Absence of NAGLU leads to acc…