Gene, stem and cellular therapies - Our impact

Waseem Qasim

Professor Waseem Qasim, deputy lead of the Gene, stem and cellular therapies, has developed and delivered over a dozen new cell and gene therapies for ‘first in human’ trials for patients at GOSH and elsewhere. Gene therapy techniques at GOSH have evolved from retroviral vectors, to lentiviral gene-addition and mRNA delivery of TALENS for genome editing. Emerging applications of CRISPR/Cas9 and base editing are now being applied for the next generation of treatments for leukaemia and inherited blood/immune disorders.

Recent publications include:

  1. 2020, UCART19 Group. Genome-edited, donor-derived allogeneic anti-CD19 chimeric antigen receptor T cells in paediatric and adult B-cell acute lymphoblastic leukaemia. Lancet. 2020 Dec 12;396(10266):1885-1894.
  2. 2017, Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells. Science Translational Medicine 9, 374
  3. 2016, Automated manufacturing of chimeric antigen receptor T cells for adoptive immunotherapy. Cytotherapy 18: 1002-11
  4. 2018, TCR/CD3 disruption enables CD3-specific antileukemic T cell immunotherapy. JCI Insight 3 (13).
  5. 2019 Safety and early efficacy outcomes for lentiviral fibroblast gene therapy in recessive dystrophic epidermolysis bullosa. JCI Insight. 2019 Jun6;4(11)
  6. 2018, Long Terminal Repeat CRISPR-CAR-Coupled "Universal" T Cells Mediate Potent Anti-leukemic Effects. Mol Ther 26, 1215-27