MSc Cell & Gene Therapy Dissertations
2015
CRISPR/Cas9 mediated knock-out of ENaC in cystic fibrosis
Identification of endogenous SAP regulatory elements for the improvement of XLP gene therapy
Immune complex uptake by expanded gamma delta T-cells
In vitro differentiation of first trimester chorionic stem cells into podocytes - implications for cell therapy in Alport syndrome
Lentiviral configurations for T-cell transduction
Modified mRNAs for gene therapy of cystic fibrosis: a novel alternative treatment strategy
Optimisation of adeno-associated viral vector for use in gene therapy
A perinatal gene therapy approach for a mouse model of cerebral palsy using an adeno-associated virus vector
Utilizing induced pluripotent stem cell technology to advance human in vitro skeletal muscle models: preliminary results on myogenic and vasculogenic cell co-culture compatibility
2014
CAR array therapy for acute myeloid leukaemia
CRISPR/Cas system applications: generation of genetically modified mouse ES cells and knockout rat
Receptor-targeted nanocomplexes for gene delivery into epidermal keratinocytes - implications for gene therapy for skin diseases
TALEN-mediated TRIM5CypA construct genetically engineered as a means to confer human T lymphocytes with resistance to HIV-1 infection
Validation of the hyperactive piggyBac transposon system expressing full-length dystrophin in dystrophic myoblastic cell lines
2013
Developing lentiviral vectors for gene therapy of Fabry disease
Development of a recombinogenic lentivirus system for the transfer of full-length human dystrophin cDNA
Development of gene therapy for HLH due to perforin deficiency - transplant of corrected effector cells
Generating a lentiviral vector for use in early studies to investigate the potential of gene therapy for the treatment of hereditary angioedema
Lineage restricted SAP transgene expression using the SAP endogenous promoter
Nanoparticle-delivered ENaC siRNA as a therapeutic strategy for cystic fibrosis