J. Appleby

Gene therapy in rare diseases: the benefits and challenges of developing a patient-centric registry for Strimvelis in ADA-SCID

A. Aiuti, S. Zancan, Pauline Ng, 2018, Orphanet Journal of Rare Diseases.

Good Laboratory Practice Preclinical Safety Studies for GSK2696273 (MLV Vector-Based Ex Vivo Gene Therapy for Adenosine Deaminase Deficiency Severe Combined Immunodeficiency) in NSG Mice.

E. Draghici, A. Aiuti, P. Cristofori, 2017, Human gene therapy. Clinical development.

A detailed study of loss of heterozygosity on chromosome 17 in tumours from Li – Fraumeni patients carrying a mutation to the TP53 gene

D. Evans, J. Varley, J. Birch, 1997, Oncogene.

Abstracts of the 7th International Workshop on AT, November, 1997, Clermont-Ferrand, France

T. Stankovic, A. Taylor, J. Varley, 2002, Disease markers.

The effects of the TRPV1 antagonist SB-705498 on TRPV1 receptor-mediated activity and inflammatory hyperalgesia in humans

B. Chizh, A. Napolitano, R. Lai, 2007, Pain.

Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety

M. Roncarolo, A. Aiuti, E. D. De Boever, 2018, Molecular therapy : the journal of the American Society of Gene Therapy.

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency.

J. Puck, I. Tezcan, M. Roncarolo, 2016, Blood.

Absence of mutations in the ATM gene in breast cancer patients with severe responses to radiotherapy.

T. Stankovic, A. Taylor, J. Varley, 1997, British Journal of Cancer.

The effects of the TRPV1 receptor antagonist SB-705498 on trigeminovascular sensitisation and neurotransmission

G. A. Lambert, B. Chizh, J. Appleby, 2009, Naunyn-Schmiedeberg's Archives of Pharmacology.

702 A RANDOMISED, TWO‐PERIOD CROSS‐OVER STUDY TO INVESTIGATE THE EFFICACY OF THE TRPV1 ANTAGONIST SB‐705498 IN ACUTE MIGRAINE

B. Chizh, J. Palmer, J. Appleby, 2009 .