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ESGCT Expert Interview - Claire Booth

  • Apr 20
  • 3 min read

1.    What advice do you wish you had received at both the start and the conclusion of your PhD journey?

 

  • Be open to change — your original plan might evolve, but the learning along the way is just as valuable.

  • Expect setbacks — it’s is a safe space to make mistakes, so treat them as part of the process, not as failures.

  • The experience is as much about training and developing your skills as it is about producing results.

  • Start writing up before you finish!

 

2. What qualities do you value most in a prospective early-career researcher seeking to join your lab?

Curiosity, integrity, ambition, resilience

 

3.    What developments or trends in your research field excite you the most?

I think the shift toward in vivo gene therapy will be a major game-changer, especially if we can achieve precise targeting at the cell and organ level. Combined with newer gene editing techniques that enable truly personalized treatments and corrections, this could completely transform the field — and most importantly, lead to real impact for patients across a wide range of conditions.

 

4. Which manuscript published in the last two years has most profoundly influenced your thinking?

Cell. 2023 Mar 30;186(7):1398-1416.e23.

doi: 10.1016/j.cell.2023.02.027. Epub 2023 Mar 20.

Human T cell generation is restored in CD3δ severe combined immunodeficiency through adenine base editing

PMID: 36944331

 

There’s so much exciting work out there, but I am biased — I get especially energised by research in my own field: inherited immune and blood disorders, particularly when there’s clear translational potential.


Don Kohn’s group has spent decades advancing HSC-targeted gene therapies for immunodeficiencies, always staying at the forefront by integrating new technologies.  They published in 2023 a paper I really liked focused on an ultra-rare form of severe combined immunodeficiency — CD3δ SCID — a condition that disproportionately affects populations where finding a suitable bone marrow donor can be especially challenging. They developed a highly efficient base editing approach that corrected the immune abnormalities both in vitro and in vivo. It was beautifully done and lays solid groundwork for translating this to clinic.


This kind of work strengthens my belief that gene therapies can offer safer, gentler, more targeted cures for ultra-rare diseases. Seeing correction and a molecular and cellular level that has the potential to directly impact patients is incredibly motivating — it’s a powerful reminder of why this work matters and why I’m passionate about being part of it.


Claire Booth Biography



Claire is a Professor in Gene Therapy and Paediatric Immunology at UCL Great Ormond Street Institute of Child Health and Honorary Consultant in Paediatric Immunology and Gene Therapy at Great Ormond Street Hospital, London. She graduated from Guy’s, King’s and St. Thomas’ School of Medicine in 2001 and then trained in Paediatrics, subspecialising in Paediatric Immunology and Immunodeficiency. She undertook a PhD at UCL developing haematopoietic stem cell gene therapy to treat XLP. 


She now works as a clinical academic leading an expanding number of gene therapy clinical trials at Great Ormond Street Hospital which treats patients with immune deficiencies, haematological and metabolic disorders. Her pre-clinical research focuses on the development of gene therapy strategies to treat XLP-1 and XIAP deficiency using a conventional lentiviral mediated corrective strategy alongside exploration of novel gene editing techniques to facilitate targeted gene addition in both haematopoietic stem cells and T cells. As an attending physician she oversees the clinical management of patients with PID, including hematopoietic stem cell transplantation and maintains a strong interest in HLH disorders. She also holds an honorary position at Boston Children’s Hospital/Dana Farber Cancer Institute and Harvard Medical School. 

 
 
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