Biblio

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Journal Article

Fox T, Bueren J, Candotti F, Fischer A, Aiuti A, Lankester A, Booth C. Access to gene therapy for rare diseases when commercialization is not fit for purpose. Nat Med. 2023.

Panchal N, Houghton BChristophe, Vassalou E, Thrasher AJ, Booth C. Allosteric Inhibition of SHP2 rescues functional T-cell abnormalities in SAP deficiency. J Allergy Clin Immunol. 2022.

Mudde A, Booth C. Gene therapy for inborn error of immunity - current status and future perspectives. Curr Opin Allergy Clin Immunol. 2023;23(1):51-62.

Bahal S, Zinicola M, Moula SE, Whittaker TE, Schejtman A, Naseem A, Blanco E, Vetharoy W, Hu Y-T, Rai R, et al. Hematopoietic stem cell gene editing rescues B cell development in X-linked agammaglobulinemia. J Allergy Clin Immunol. 2024.

Ferrua F, Galimberti S, Courteille V, Slatter MAnne, Booth C, Moshous D, Neven B, Blanche S, Laberko A, Shcherbina A, et al. Hematopoietic stem cell transplantation for CD40 ligand deficiency: results from an EBMT/ESID-IEWP-SCETIDE-PIDTC Study. J Allergy Clin Immunol. 2019.

Fischer M, Olbrich P, Hadjadj J, Aumann V, Bakhtiar S, Barlogis V, von Bismarck P, Bloomfield MPhDMarké, Booth C, Buddingh EP, et al. JAK-inhibitor treatment for inborn errors of JAK/STAT signaling: An ESID and EBMT IEWP retrospective study. J Allergy Clin Immunol. 2023.

Houghton BC, Booth C, Thrasher AJ. Lentivirus technologies for modulation of the immune system. Curr Opin Pharmacol. 2015;24:119-127.

Elfeky R, Lucchini G, Lum S-H, Ottaviano G, Builes N, Nademi Z, Battersby A, Flood T, Owens S, Cant AJ, et al. New insights into risk factors for transplant-associated thrombotic microangiopathy in pediatric HSCT. Blood Adv. 2020;4(11):2418-2429.

Golwala ZM, Bhat NGireesh, Xu-Bayford J, Stankova T, Adams S, Morris EC, Qasim W, Booth C, Worth A, Kusters MA, et al. Non-osteopenic Bone Pathology After Allo-hematopoietic Stem Cell Transplantation in Patients with Inborn Errors of Immunity. J Clin Immunol. 2023.

Meinhardt A, Reilly L, Kaliakatsos M, Abdel-Aziz K, Alsharidah S, Bodi I, Booth C, Chetty K, Evans J, Ferreras-Antolín L, et al. Novel antivirals for severe enterovirus infection in immunocompromised hosts; a case series. J Infect. 2024:106142.

Yang L, Booth C, Speckmann C, Seidel MG, Worth AJj, Kindle G, Lankester AC, B G, Gennery AR, Seppänen MRj, et al. Phenotype, genotype, treatment, and survival outcomes in patients with X-linked inhibitor of apoptosis deficiency. J Allergy Clin Immunol. 2021.

Uhlig HH, Booth C, Cho J, Dubinsky M, Griffiths AM, Grimbacher B, Hambleton S, Huang Y, Jones K, Kammermeier J, et al. Precision medicine in monogenic inflammatory bowel disease: proposed mIBD REPORT standards. Nat Rev Gastroenterol Hepatol. 2023.

Tam PKwong Hang, Wong KKak Yuen, Atala A, Giobbe GGiuseppe, Booth C, Gruber PJ, Monone M, Rafii S, Rando TA, Vacanti J, et al. Regenerative medicine: postnatal approaches. Lancet Child Adolesc Health. 2022;6(9):654-666.

Burns H, Collins A, Marsden P, Flood TJ, Slatter MA, Booth C, Xu-Bayford J, Leahy TRonan. Severe Combined Immunodeficiency (SCID)-the Irish Experience. J Clin Immunol. 2021.

Panchal N, Ghosh S, Booth C. T cell gene therapy to treat immunodeficiency. Br J Haematol. 2020.

Fox TAndrew, Houghton BChristophe, Petersone L, Waters E, Edner NMona, McKenna A, Preham O, Hinze C, Williams C, de Albuquerque ASilva, et al. Therapeutic gene editing of T cells to correct CTLA-4 insufficiency. Sci Transl Med. 2022;14(668):eabn5811.

Booth C, H Gaspar B, Thrasher AJ. Treating Immunodeficiency through HSC Gene Therapy. Trends Mol Med. 2016.

Mudde ACa, Kuo CY, Kohn DB, Booth C. What a Clinician Needs to Know about Genome Editing: Status and Opportunities for Inborn Errors of Immunity. J Allergy Clin Immunol Pract. 2024.