UCL Great Ormond Street Institute of Child Health


Great Ormond Street Institute of Child Health


MSc Cell & Gene Therapy Dissertations


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



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



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