A5003327249- HIV Research and Treatment
- Genetics and Neurodevelopmental Disorders
- CRISPR and Genetic Engineering
- Mast cells and histamine
- RNA Interference and Gene Delivery
- Biomedical Ethics and Regulation
- HIV/AIDS drug development and treatment
- Virus-based gene therapy research
- Autism Spectrum Disorder Research
- CAR-T cell therapy research
- Hormonal Regulation and Hypertension
- Pituitary Gland Disorders and Treatments
- Adrenal and Paraganglionic Tumors
- Genomic variations and chromosomal abnormalities
- Click Chemistry and Applications
- Eosinophilic Disorders and Syndromes
- Pluripotent Stem Cells Research
- Immune Cell Function and Interaction
- Medical Device Sterilization and Disinfection
- Cell Adhesion Molecules Research
- RNA and protein synthesis mechanisms
- Mesenchymal stem cell research
- Blood properties and coagulation
- Monoclonal and Polyclonal Antibodies Research
- Prenatal Screening and Diagnostics
Christchurch Hospital
2024-2025
The University of Sydney
2014-2019
Royal Prince Alfred Hospital
2011-2019
Centenary Institute
2019
Johnson & Johnson (United States)
2004-2014
Wessex Regional Genetics Laboratory
1994-2010
Salisbury District Hospital
1996-2010
Johnson & Johnson (Australia)
1994-2009
Antrim Area Hospital
2006
Stanford University
2004
Retroviral vectors have been used in successful gene therapies. However, some patients, insertional mutagenesis led to leukemia or myelodysplasia. Both the strong promoter/enhancer elements long terminal repeats (LTRs) of murine virus (MLV)-based and vector-specific integration site preferences played an important role these adverse clinical events. MLV is known prefer regions near transcription start sites (TSS). Recently, BET family proteins were shown be major cellular responsible for...
A phase I gene transfer clinical study was undertaken to examine the ability introduce a potential anti-human immunodeficiency virus (HIV) therapeutic into hematopoietic progenitor cells (HPC), thereby contributing multilineage engraftment. The effect of genetically modifying HPC with protective genes in HIV-infected adults depends part on presence adult thymic activity and myeloid capacity setting HIV replication. Herein we report expression retroviral vector encoding an anti-HIV-1 ribozyme...
The fragile X mutation can now be recognised by a variety of molecular techniques. We report pilot screening survey population children with mental impairment in which we used Southern blotting methods to detect the mutation, augmented cytogenetic studies on whose phenotype suggested possible chromosome abnormality. There were 873 special educational needs our and 310 fulfilled criteria for testing. A sample was obtained from 254, whom four found have full fra(X) (delta L) none premutation....
Abstract Background An anti‐HIV‐1 tat ribozyme, termed Rz2, has been shown to inhibit HIV‐1 infection/replication and decrease HIV‐1‐induced pathogenicity in T‐lymphocyte cell lines normal peripheral blood T‐lymphocytes. We report here the results of a phase I gene transfer clinical trial using Rz2. Methods Apheresis was used obtain population from each four HIV‐negative donors. After enrichment for CD4+ T‐lymphocytes, ex vivo expansion genetic manipulation (approximately equal aliquots...
Postoperative outcomes of patients with normotensive pheochromocytomas are poorly documented. We aimed to evaluate the impact preoperative hypertension on post-operative following adrenalectomy for pheochromocytoma. An international retrospective study undergoing pheochromocytoma in 46 centers between 2012-2022 was performed. Hypertensive and were defined respectively by presence or absence history before at time diagnosis. To differences postoperative hypertensive patients, propensity score...
Human peripheral blood lymphocytes (PBLs) were transduced with a number of recombinant retroviruses including RRz2, an LNL6-based virus ribozyme targeted to the human immunodeficiency (HIV) tat gene transcript inserted within 3' region neomycin-resistance gene; RASH5, and LNHL-based containing antisense sequence 5' leader HIV-1 downstream cytomegalovirus promoter; R20TAR, LXSN-based 20 tandem copies trans-activation response element driven by Moloney murine leukemia long terminal repeat....
Several hammerhead ribozymes targeted to different sites within the retroviral packaging (psi) sequences of Moloney murine leukemia virus (Mo-MLV) and human immunodeficiency type 1 (HIV-1) were designed shown cleave target RNA in vitro at chosen sites. The engineered ribozymes, as well antisense sequence complementary Mo-MLV psi region, cloned into 3' untranslated region neomycin-resistance gene (neo). This was coupled simian 40 early promoter pSV2neo vector. For against site, constructs...
Gene therapy for individuals infected with HIV has the potential to provide a once-only treatment that will act reduce viral load, preserve immune system, and mitigate cumulative toxicities associated highly active antiretroviral (HAART). The authors have been involved in two clinical trials (phase I phase II) using gene-modified adult hematopoietic stem cells (HSCs), these are discussed as prototypic within general field of HSC gene HIV. Taken group shown (i) safety both procedure anti-HIV...
An n-allele model is developed for the FMR1 locus, which causes fragile X syndrome, where n number of triplet repeats in first exon. Frequencies general population and index families are used to generate an + delta transition matrix that predicts specific risks satisfactory agreement with observation. However, until sequencing distinguishes between stable unstable alleles same value n, it premature infer whether allelic frequencies at locus equilibrium or, as some have suggested, evolving...
Abstract Background Gene therapy represents a new treatment paradigm for HIV that is potentially delivered by safe, once‐only therapeutic intervention. Methods Using mathematical modelling, we assessed the possible impact of autologous haematopoietic stem cell (HSC) delivered, anti‐HIV gene therapy. The comprises ribozyme construct (OZ1) directed to conserved region HIV‐1 transduced HSC (OZ1+HSC). OZ1+HSC contributes CD4+ T lymphocyte and monocyte/macrophage pools preferentially expand under...
Antiretroviral drug therapy can effectively reduce the viral load, and is associated with a degree of immune reconstitution in human immunodeficiency virus (HIV)-infected patients. However, presence latent reservoir, development resistance, toxicity, compliance problems are obstacles that impede full eradication HIV through therapy. The cellular introduction genetic elements capable inhibiting replication conceptually appealing as potential new treatment paradigm for aquired syndrome (AIDS)....
Celiac disease (gluten-sensitive enteropathy [GSE]) is a disorder characterized by small intestinal mucosal injury caused dietary exposure to wheat gluten and similar proteins. There evidence that the immunologically mediated there an inflammatory infiltrate present in mucosa. It postulated release of lipid-derived mediators may be involved pathogenesis injury. Jejunal biopsy samples from patients with GSE group who were subsequently shown have normal jejunal mucosa incubated tritiated...