H. Gaspar

A. Gennery, M. Pearce, P. Veys, 2017, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

A Practical Guide to Implementing Population Newborn Screening (NBS) for Severe Combined Immunodeficiency (SCID)

H. Gaspar, 2017 .

The Case for Mandatory Newborn Screening for Severe Combined Immunodeficiency (SCID)

L. Hammarström, N. Mahlaoui, S. Borte, 2014, Journal of Clinical Immunology.

Screening of Neonatal UK Dried Blood Spots Using a Duplex TREC Screening Assay

K. Harvey, H. Gaspar, Tharindu Premachandra, 2014, Journal of Clinical Immunology.

How I treat severe combined immunodeficiency.

P. Veys, P. Amrolia, W. Qasim, 2013, Blood.

Neonatal diagnosis of severe combined immunodeficiency leads to significantly improved survival outcome: the case for newborn screening.

A. Gennery, P. Veys, E. Davies, 2011, Blood.

Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study

A. Fischer, A. Gennery, E. Haddad, 2014, The Lancet.

reconstitution after haploidentical stem cell transplant Adoptive immunotherapy with allodepleted donor T-cells improves immune

H. Heslop, C. Rooney, M. Cobbold, 2013 .

Adoptive immunotherapy with allodepleted donor T-cells improves immune reconstitution after haploidentical stem cell transplantation.

R. Krance, H. Heslop, C. Rooney, 2006, Blood.

T-cell gene therapy for perforin deficiency corrects cytotoxicity defects and prevents hemophagocytic lymphohistiocytosis manifestations

A. Schambach, S. Ehl, P. Ashton‐Rickardt, 2018, The Journal of allergy and clinical immunology.

Whole genome sequencing of a sporadic primary immunodeficiency cohort

William J. Astle, Sri V. V. Deevi, Kenneth G. C. Smith, 2018, Nature.

X-linked lymphoproliferative disease due to SAP/SH2D1A deficiency: a multicenter study on the manifestations, management and outcome of the disease.

A. Fischer, E. Cirillo, A. Plebani, 2011, Blood.

Pathology of astrovirus associated diarrhoea in a paediatric bone marrow transplant recipient

N. Sebire, H. Gaspar, G. Anderson, 2004, Journal of Clinical Pathology.

Gene therapy for primary immunodeficiencies.

A. Thrasher, H. Gaspar, G. Santilli, 2012, Human gene therapy.

Nonmyeloablative stem cell transplantation for congenital immunodeficiencies.

G. Mieli-Vergani, P. Veys, P. Amrolia, 2000, Blood.

SAP mediates specific cytotoxic T-cell functions in X-linked lymphoproliferative disease.

A. Thrasher, C. Kinnon, K. Gilmour, 2004, Blood.

Clinical and immunological features in a cohort of patients with partial DiGeorge syndrome followed at a single center.

G. Giardino, A. Worth, Winnie Ip, 2019, Blood.

Hematopoietic Stem Cell Transplantation Outcomes for 11 Patients with Dedicator of Cytokinesis 8 (DOCK8) Deficiency

L. Notarangelo, J. Chou, W. Al-Herz, 2017 .

Thymus transplantation for complete DiGeorge syndrome: European experience

Kathryn L. Parsley, N. Sebire, A. Worth, 2017, Journal of Allergy and Clinical Immunology.

Combined immunodeficiency due to JAK3 mutation in a child presenting with skin granuloma.

C. Cancrini, F. Locatelli, A. Aiuti, 2016, The Journal of allergy and clinical immunology.

Hematopoietic stem cell transplantation outcomes for 11 patients with dedicator of cytokinesis 8 deficiency.

Douglas R. McDonald, L. Notarangelo, R. Geha, 2016, The Journal of allergy and clinical immunology.

Immunodeficiency: Long-term persistence of a polyclonal t cell repertoire after gene therapy for X-linked severe combined immunodeficiency

Kathryn L. Parsley, S. Cooray, A. Thrasher, 2011 .

Long-Term Persistence of a Polyclonal T Cell Repertoire After Gene Therapy for X-Linked Severe Combined Immunodeficiency

Kathryn L. Parsley, S. Cooray, A. Thrasher, 2011, Science Translational Medicine.

Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA

F. Farzaneh, A. Biffi, B. Bigger, 2019, Molecular therapy. Methods & clinical development.

Immunodeficiency and disseminated mycobacterial infection associated with homozygous nonsense mutation of IKKβ☆

S. Nejentsev, V. Plagnol, S. Burns, 2014, The Journal of allergy and clinical immunology.

T cell suicide gene therapy to aid haematopoietic stem cell transplantation.

A. Thrasher, W. Qasim, H. Gaspar, 2005, Current gene therapy.

The impact of retroviral suicide gene transduction procedures on T cells

S. Verfuerth, A. Thrasher, C. Kinnon, 2003, British journal of haematology.

Corrigendum: Primary Immunodeficiency Diseases: An Update on the Classification from the International Union of Immunological Societies Expert Committee for Primary Immunodeficiency

J. Casanova, J. Franco, A. Bousfiha, 2014, Front. Immunol..

Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency.

David A. Williams, A. Schambach, N. Sebire, 2008, Molecular therapy : the journal of the American Society of Gene Therapy.

Gene therapy progress and prospects: gene therapy for severe combined immunodeficiency

A. Thrasher, S. Howe, H. Gaspar, 2003, Gene Therapy.

Intrinsic defects of B cell function in X‐linked severe combined immunodeficiency

P. Veys, A. Thrasher, A. Thrasher, 2000, European journal of immunology.

Multicenter phase 1/2 application of adenovirus-specific T cells in high-risk pediatric patients after allogeneic stem cell transplantation.

A. Gennery, P. Veys, P. Amrolia, 2018, Cytotherapy.

Molecular Pathogenesis of EBV Susceptibility in XLP as Revealed by Analysis of Female Carriers with Heterozygous Expression of SAP

S. Choo, S. Tangye, T. Phan, 2011, PLoS biology.

Adenosine Deaminase Deficient Severe Combined Immunodeficiency Presenting as Atypical Haemolytic Uraemic Syndrome

Joanne M. Smart, A. Worth, E. Davies, 2015, Journal of Clinical Immunology.

Busulfan Pharmacokinetics in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Gene Therapy

M. Krajinovic, M. Ansari, D. Kohn, 2020 .

Busulfan Pharmacokinetics in ADA SCID Gene Therapy.

M. Krajinovic, M. Ansari, D. Kohn, 2020, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

Outcomes of splenectomy in patients with common variable immunodeficiency (CVID): a survey of 45 patients

R. Schmidt, A. Plebani, J. Litzman, 2013, Clinical and experimental immunology.

The C76R transmembrane activator and calcium modulator cyclophilin ligand interactor mutation disrupts antibody production and B-cell homeostasis in heterozygous and homozygous mice.

C. Bacchelli, P. Schneider, A. Thrasher, 2011, The Journal of allergy and clinical immunology.

Gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency.

A. Thrasher, H. Gaspar, C. Montiel-Equihua, 2012, Current gene therapy.

Universal Newborn Screening for Severe Combined Immunodeficiency (SCID)

M. van der Burg, S. Pai, N. Mahlaoui, 2019, Front. Pediatr..

"Darwinian" tumor-suppression model unsupported in clinical experience.

A. Thrasher, W. Qasim, H. Gaspar, 2014, Molecular therapy : the journal of the American Society of Gene Therapy.

Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease

S. Benedetti, Daniel W. Stuckey, A. Thrasher, 2020, Molecular therapy. Methods & clinical development.

The β-globin locus control region in combination with the EF1α short promoter allows enhanced lentiviral vector-mediated erythroid gene expression with conserved multilineage activity.

Y. Takeuchi, Heba Saadeh, A. Thrasher, 2012, Molecular therapy : the journal of the American Society of Gene Therapy.

Characterization of the clinical and immunological phenotype and management of 157 individuals with 56 distinct heterozygous NFKB1 mutations.

William J. Astle, Sri V. V. Deevi, Kenneth G. C. Smith, 2020, The Journal of allergy and clinical immunology.

Quality of Life in Children with Primary Antibody Deficiency

C. Cale, H. Gaspar, G. Davies, 2014, Journal of Clinical Immunology.

Metabolite and thymocyte development defects in ADA-SCID mice receiving enzyme replacement therapy

G. Giardino, F. Moretti, G. la Marca, 2021, Scientific Reports.

IMMUNODEFICIENCY REVIEWEarly B cell defects

M. Conley, H. Gaspar, M. E. Conley, 2000, Clinical and experimental immunology.

Production and First-in-Man Use of T Cells Engineered to Express a HSVTK-CD34 Sort-Suicide Gene

P. Veys, F. Farzaneh, B. Fehse, 2013, PloS one.

Allogeneic stem cell transplantation in X-linked lymphoproliferative disease: two cases in one family and review of the literature

R. Egeler, A. Lankester, H. Gaspar, 2005, Bone Marrow Transplantation.

Primary Immune Deficiencies – Principles of Care

F. Bonilla, H. Chapel, H. Gaspar, 2014, Front. Immunol..

Primary immune - principles of care.

F. Bonilla, H. Chapel, H. Gaspar, 2014 .

Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome

E. Macintyre, F. Adam, A. Kauskot, 2022, Nature medicine.

X‐linked lymphoproliferative disease: clinical, diagnostic and molecular perspective

A. Thrasher, K. Gilmour, H. Gaspar, 2002, British journal of haematology.

Successful RAG1-SCID gene therapy depends on the level of RAG1 expression.

A. Schambach, J. V. van Dongen, G. Driessen, 2014, The Journal of allergy and clinical immunology.

Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood?

A. Fischer, A. Schulz, W. Friedrich, 2012, Blood.

TITLE Improved Survival after Unrelated Donor Bone Marrow Transplant in Children with Primary Immunodeficiency using a Reduced Intensity Conditioning Regimen

P. Veys, P. Amrolia, C. Cale, 2004 .

Non-myeloablative stem cell transplantation for congenital immunodeficiencies.

P. Veys, P. Amrolia, N. Hadžić, 2002, Recent results in cancer research. Fortschritte der Krebsforschung. Progres dans les recherches sur le cancer.

Short title: Outcome of related and unrelated HSCT for SCID

A. Fischer, A. Schulz, W. Friedrich, 2017 .

A. Fischer, A. Schulz, W. Friedrich, 2014 .

Cognitive and behavioral abnormalities in children after hematopoietic stem cell transplantation for severe congenital immunodeﬁciencies

P. Veys, A. Thrasher, E. Davies, 2008 .

Severe combined immunodeficiency—molecular pathogenesis and diagnosis

K. Gilmour, H. Gaspar, A. Jones, 2001, Archives of disease in childhood.

Adenosine Deaminase Deficient Severe Combined Immunodeficiency Presenting as Atypical Haemolytic Uraemic Syndrome

Joanne M. Smart, A. Worth, E. Davies, 2015, Journal of Clinical Immunology.

Improved outcome for children with disseminated adenoviral infection following allogeneic stem cell transplantation

P. Veys, S. Samarasinghe, P. Amrolia, 2005, British journal of haematology.

Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting.

David A. Williams, C. von Kalle, J. Wagner, 2011, Molecular therapy : the journal of the American Society of Gene Therapy.

Impact of viral reactivations in the era of pre-emptive antiviral drug therapy following allogeneic haematopoietic SCT in paediatric recipients

P. Veys, P. Amrolia, R. Chiesa, 2013, Bone Marrow Transplantation.

Host natural killer immunity is a key indicator of permissiveness for donor cell engraftment in patients with severe combined immunodeficiency☆

A. Gennery, P. Veys, A. Worth, 2014, The Journal of allergy and clinical immunology.

Tearing RAGs apart.

H. Gaspar, H. Bobby Gaspar, 2014, Blood.

Kingdom experience transplantation in children with primary immunodeficiency: United Treosulfan-based conditioning regimens for hematopoietic stem cell

P. Veys, P. Amrolia, W. Qasim, 2012 .

Treosulfan-based conditioning regimens for hematopoietic stem cell transplantation in children with primary immunodeficiency: United Kingdom experience.

A. Gennery, P. Veys, P. Amrolia, 2011, Blood.

SAP gene transfer restores cellular and humoral immune function in a murine model of X-linked lymphoproliferative disease.

N. Sebire, A. Thrasher, H. Gaspar, 2013, Blood.

X‐Linked lymphoproliferative disease: three atypical cases

K. Gilmour, E. Davies, H. Gaspar, 2001, Clinical and experimental immunology.

Successful treatment of lymphoproliferative disease complicating primary immunodeficiency/immunodysregulatory disorders with reduced-intensity allogeneic stem-cell transplantation.

N. Sebire, P. Veys, P. Amrolia, 2007, Blood.

Diagnosis of X‐linked lymphoproliferative disease by analysis of SLAM‐associated protein expression

K. Nichols, D. Goldblatt, A. Thrasher, 2000, European journal of immunology.

Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo.

Christine Kinnon, Christof von Kalle, Dick de Ridder, 2007, The Journal of clinical investigation.

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients.

Christine Kinnon, Christof von Kalle, Michael Hubank, 2008, The Journal of clinical investigation.

A modified γ-retrovirus vector for X-linked severe combined immunodeficiency.

Christopher Baum, David A. Williams, Gregory Hopkins, 2014, The New England journal of medicine.

Long-term lymphoid progenitors independently sustain naïve T and NK cell production in humans

Elena Blanco, L. Biasco, A. Thrasher, 2021, Nature Communications.

Treating Immunodeficiency through HSC Gene Therapy.

A. Thrasher, H. Gaspar, C. Booth, 2016, Trends in molecular medicine.

Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome.

Frederic D Bushman, Fulvio Mavilio, Anne Galy, 2015, JAMA.

Immune deficiencies , infection , and systemic immune disorders Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe : Entering a new century , do we do better ?

A. Fischer, W. Friedrich, L. Notarangelo, 2010 .

Mutations in TNFRSF13B Encoding TACI Are Associated With Common Variable Immunodeficiency in Humans

Christine Kinnon, Manfred Schmidt, R. Levinsky, 2006, Pediatrics.

Primary Immunodeficiency Diseases: an Update on the Classification from the International Union of Immunological Societies Expert Committee for Primary Immunodeficiency 2015

J. Casanova, J. Franco, A. Bousfiha, 2015, Journal of Clinical Immunology.

Lentiviral gene therapy rescues p47phox chronic granulomatous disease and the ability to fight Salmonella infection in mice

S. Clare, S. Burns, A. Thrasher, 2020, Gene Therapy.

Lentiviral gene therapy for X-linked chronic granulomatous disease

Luca Biasco, Adrian J. Thrasher, David A. Williams, 2020, Nature Medicine.

Perforin Gene Transfer Into Hematopoietic Stem Cells Improves Immune Dysregulation in Murine Models of Perforin Deficiency

A. Schambach, P. Malik, A. Thrasher, 2014, Molecular therapy : the journal of the American Society of Gene Therapy.

Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency.

Michael L Kaufman, Aaron R Cooper, K. Cornetta, 2014, Molecular therapy : the journal of the American Society of Gene Therapy.

Gene therapy for primary immunodeficiency

A. Thrasher, H. Gaspar, C. Booth, 2011, Current opinion in pediatrics.

Hematopoietic Stem Cell Gene Therapy for Adenosine Deaminase–Deficient Severe Combined Immunodeficiency Leads to Long-Term Immunological Recovery and Metabolic Correction

Kathryn L. Parsley, P. Veys, S. Cooray, 2011, Science Translational Medicine.

Comprehensive genomic access to vector integration in clinical gene therapy

Luca Biasco, Alessandro Aiuti, Richard Gabriel, 2009, Nature Medicine.

Improving cellular therapy for primary immune deficiency diseases: recognition, diagnosis, and management.

L. Notarangelo, H. Ochs, J. Puck, 2009, The Journal of allergy and clinical immunology.

Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells

A. Schambach, A. Thrasher, M. Antoniou, 2007, Blood.

DOCK8 Deficiency: Clinical and Immunological Phenotype and Treatment Options - a Review of 136 Patients

J. Orange, A. Schulz, R. Geha, 2015, Journal of Clinical Immunology.

Primary Immunodeficiency Diseases: an Update on the Classification from the International Union of Immunological Societies Expert Committee for Primary Immunodeficiency 2015

Steven M. Holland, Christoph Klein, Shigeaki Nonoyama, 2009, Front. Immun..

The 2015 IUIS Phenotypic Classification for Primary Immunodeficiencies

J. Casanova, J. Franco, A. Bousfiha, 2015, Journal of Clinical Immunology.

Treatment of intractable ulcerating enterocolitis of infancy by allogeneic bone marrow transplantation.

M. Ashworth, P. Veys, P. Milla, 2008, Clinical gastroenterology and hepatology : the official clinical practice journal of the American Gastroenterological Association.

Gene therapy for severe combined immune deficiency

A. Thrasher, W. Qasim, H. Gaspar, 2004, Expert Reviews in Molecular Medicine.

Site- and allele-specific polycomb dysregulation in T-cell leukaemia

B. Nadel, M. Gut, I. Gut, 2015, Nature Communications.

Prevalence of SAP gene defects in male patients diagnosed with common variable immunodeficiency

H. Chapel, H. Gaspar, K. Gilmour, 2004, Clinical and experimental immunology.

Induced pluripotent stem cells and primary immunodeficiencies: a new frontier reached, a new world beyond?

H. Gaspar, 2011, The Journal of allergy and clinical immunology.

Whole-genome sequencing of a sporadic primary immunodeficiency cohort

William J. Astle, Sri V. V. Deevi, Kenneth G. C. Smith, 2020, Nature.

The impact of telomere erosion on memory CD8+ T cells in patients with X-linked lymphoproliferative syndrome

M. Salmon, N. Khan, A. Akbar, 2005, Mechanisms of Ageing and Development.

Lentiviral vectors for T-cell suicide gene therapy: preservation of T-cell effector function after cytokine-mediated transduction.

A. Thrasher, C. Kinnon, W. Qasim, 2007, Molecular therapy : the journal of the American Society of Gene Therapy.

Cognitive and behavioral abnormalities in children after hematopoietic stem cell transplantation for severe congenital immunodeficiencies.

T. Cole, P. Veys, A. Thrasher, 2008, Blood.

Progress and prospects: gene therapy clinical trials (part 1) (vol 14, pg 1439, 2007)

R. Ali, J. Bainbridge, T. Flotte, 2007 .

Development of gene therapy: potential in severe combined immunodeficiency due to adenosine deaminase deficiency

H. Bobby Gaspar, A. Thrasher, H. Gaspar, 2009, Stem cells and cloning : advances and applications.

Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult.

F. Bushman, S. Grace, A. Galy, 2017, Blood.

Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency.

A. Fischer, W. Friedrich, L. Notarangelo, 2012, Blood.

"Special exemptions": should they be put on trial?

A. Thrasher, H. Gaspar, Sue Swift, 2013, Molecular therapy : the journal of the American Society of Gene Therapy.

Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID

A. Schambach, M. van der Burg, J. V. van Dongen, 2020, Molecular therapy. Methods & clinical development.

Biochemical and immunological status following gene therapy and PEG-ADA therapy for adenosine deaminase (ADA) deficiency.

H. Simmonds, V. V. van Beusechem, H. Gaspar, 1994, Advances in experimental medicine and biology.

Recent advances in understanding the pathogenesis and management of reticular dysgenesis

U. Pannicke, K. Schwarz, H. Gaspar, 2018, British journal of haematology.

Serial transplantation of mismatched donor hematopoietic cells between HLA-identical sibling pairs with congenital immunodeficiency: in vivo tolerance permits rapid immune reconstitution following T-replete transplantation without GVHD in the secondary recipient.

P. Veys, P. Amrolia, E. Davies, 2006, Blood.

Signal transducer and activator of transcription 5 tyrosine phosphorylation for the diagnosis and monitoring of patients with severe combined immunodeficiency.

A. Thrasher, K. Gilmour, C. Cale, 2009, The Journal of allergy and clinical immunology.

Third‐party virus‐specific T cells eradicate adenoviraemia but trigger bystander graft‐versus‐host disease

P. Veys, P. Amrolia, R. Chiesa, 2011, British journal of haematology.

Absence of expression of the Wiskott-Aldrich syndrome protein in peripheral blood cells of Wiskott-Aldrich syndrome patients.

C. Kinnon, H. Gaspar, A. Jones, 1998, Clinical immunology and immunopathology.

Progress and prospects: gene therapy for inherited immunodeficiencies

A. Thrasher, W. Qasim, H. Gaspar, 2009, Gene Therapy.

Gene therapy for haemophagocytic lymphohistiocytosis.

H. Gaspar, C. Booth, M. Carmo, 2014, Current gene therapy.

A novel Rab27a mutation binds melanophilin, but not Munc13-4, causing immunodeficiency without albinism.

H. Hakonarson, J. Orange, C. Klein, 2016, The Journal of allergy and clinical immunology.

DOCK8 Deficiency: Clinical and Immunological Phenotype and Treatment Options - a Review of 136 Patients

J. Orange, A. Schulz, R. Geha, 2015, Journal of Clinical Immunology.

How We Manage Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA SCID)

D. Kohn, H. Gaspar, 2017, Journal of Clinical Immunology.

Variable Phenotype of Severe Immunodeficiencies Associated with RMRP Gene Mutations

A. Gennery, P. Veys, C. Bacchelli, 2015, Journal of Clinical Immunology.

Protein assays for diagnosis of Wiskott–Aldrich syndrome and X‐linked thrombocytopenia

A. Finn, A. Thrasher, C. Kinnon, 2001, British journal of haematology.

Rapid protein‐based assays for the diagnosis of T–B+ severe combined immunodeficiency

C. Kinnon, K. Gilmour, E. Davies, 2001, British journal of haematology.

Lentiviral Gene Transfer Corrects Immune Abnormalities in XIAP Deficiency

A. Thrasher, H. Gaspar, C. Booth, 2022, Journal of Clinical Immunology.

The 2015 IUIS Phenotypic Classification for Primary Immunodeficiencies

J. Casanova, J. Franco, A. Bousfiha, 2015, Journal of Clinical Immunology.

Preimplantation Genetic Diagnosis and HLA-Typing: The Potential for Selection of Unaffected HLA Matched Siblings

H. Gaspar, L. Brown, 2014 .

X-Linked Agammaglobulinemia

C. Kinnon, H. Gaspar, Surjit Singh, 2016 .

Kinase mutant Btk results in atypical X‐linked agammaglobulinaemia phenotype

I. Caragol, C. Kinnon, H. Gaspar, 2000, Clinical and experimental immunology.

Bilateral sensorineural deafness in adenosine deaminase-deficient severe combined immunodeficiency.

H. Gaspar, Wendy Albuquerque, 2004, The Journal of pediatrics.

Expression of Bruton's tyrosine kinase protein within the B cell lineage

R. Callard, R. Lovering, R. Levinsky, 1994, European journal of immunology.

Non-posttransplant lymphoproliferative disorder malignancy after hematopoietic stem cell transplantation in patients with primary immunodeficiency: UK experience.

A. Gennery, P. Veys, A. Worth, 2018, The Journal of allergy and clinical immunology.

Sleeping beauty transposition from nonintegrating lentivirus.

C. Vink, R. McIvor, A. Thrasher, 2009, Molecular therapy : the journal of the American Society of Gene Therapy.

Retrospective, Landmark Analysis of Long-term Adult Morbidity Following Allogeneic HSCT for Inborn Errors of Immunity in Infancy and Childhood

A. Laurence, P. Veys, A. Worth, 2022, Journal of Clinical Immunology.

Hematopoietic Stem Cell Transplantation as Treatment for Patients with DOCK8 Deficiency.

J. Ziegler, A. Schulz, R. Geha, 2019, The journal of allergy and clinical immunology. In practice.

Cognitive and behavioural abnormalities in children following haematopoietic stem cell transplantation for severe congenital immunodeficiencies

P. Veys, A. Thrasher, E. Davies, 2008 .

Lentiviral gene therapy for X-linked chronic granulomatous disease

David A. Williams, Leo D. Wang, F. Bushman, 2020, Nature Medicine.

Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning

A. Schambach, F. Staal, A. Schwarzer, 2021, Molecular therapy : the journal of the American Society of Gene Therapy.

The Case for Mandatory Newborn Screening for Severe Combined Immunodeficiency (SCID)

L. Hammarström, N. Mahlaoui, S. Borte, 2014, Journal of Clinical Immunology.

Screening of Neonatal UK Dried Blood Spots Using a Duplex TREC Screening Assay

K. Harvey, H. Gaspar, Tharindu Premachandra, 2014, Journal of Clinical Immunology.

Analysis of Bruton's tyrosine kinase protein expression and activity in patients with X-linked agammaglobulinemia (XLA):the use of protein analysis as a diagnostic indicator of XLA

R. Levinsky, C. Kinnon, H. Gaspar, 1998 .

Non-infectious Lung Disease in Patients with Adenosine Deaminase Deficient Severe Combined Immunodeficiency

H. Gaspar, L. Fairbanks, C. Owens, 2012, Journal of Clinical Immunology.

Chronic Infection with Rotavirus Vaccine Strains in UK Children with Severe Combined Immunodeficiency.

A. Gennery, A. Worth, H. Gaspar, 2015, The Pediatric infectious disease journal.

Interferon‐γ capture T cell therapy for persistent Adenoviraemia following allogeneic haematopoietic stem cell transplantation

P. Veys, Winnie Ip, W. Qasim, 2013, British journal of haematology.

Reexamining the role of TACI coding variants in common variable immunodeficiency and selective IgA deficiency

C. Cunningham-Rundles, Likun Du, Q. Pan-Hammarström, 2007, Nature Genetics.

Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome

E. Macintyre, A. Kauskot, A. Toubert, 2022, Nature Medicine.

Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID): Molecular Pathogenesis and Clinical Manifestations

F. Moretti, D. Kohn, H. Gaspar, 2017, Journal of Clinical Immunology.

Long-Term Immune Recovery After Hematopoietic Stem Cell Transplantation for ADA Deficiency: a Single-Center Experience

P. Veys, A. Worth, H. Gaspar, 2021, Journal of clinical immunology.

Immunological and metabolic correction after lentiviral vector mediated haematopoietic stem cell gene therapy for ADA deficiency

K. Cornetta, D. Kohn, A. Thrasher, 2014 .

Social outcome in children treated by haematopoietic cell transplant for congenital immunodeficiency

H. Gaspar, E. Skucek, P. Titman, 2011, Bone Marrow Transplantation.

Quality of Life in Children with Primary Antibody Deficiency

C. Cale, H. Gaspar, G. Davies, 2014, Journal of Clinical Immunology.

Abnormal expression of only the CD34 part of a transgenic CD34/herpes simplex virus-thymidine kinase fusion protein is associated with ganciclovir resistance.

M. Deschamps, J. Rémy-Martin, P. Tiberghien, 2008, Human gene therapy.

Stem Cells and Cloning: Advances and Applications

H. Bobby Gaspar, A. Thrasher, H. Gaspar, 2009 .

Primary Immunodeficiency Diseases: An Update on the Classification from the International Union of Immunological Societies Expert Committee for Primary Immunodeficiency

A. Fischer, J. Casanova, L. Notarangelo, 2011, Front. Immun..

Somatic Gene Therapy for X-Linked Severe Combined Immunodeficiency Using a Self-Inactivating Modified Gammaretroviral Vector Results in An Improved Preclinical Safety Profile and Early Clinical Efficacy in a Human Patient

David A. Williams, Kathryn L. Parsley, F. Bushman, 2011 .

The 2017 IUIS Phenotypic Classification for Primary Immunodeficiencies

J. Casanova, J. Franco, A. Bousfiha, 2017, Journal of Clinical Immunology.

Severe combined immunodeficiency—molecular pathogenesis and diagnosis

H. Gaspar, K. Gilmour, A. M. Jones, 2000 .

Severe combined immunodeficiency—molecular pathogenesis and diagnosis

H. Gaspar, K. Gilmour, A. M. Jones, 2022 .

Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better?

A. Fischer, W. Friedrich, L. Notarangelo, 2010, The Journal of allergy and clinical immunology.

Spectrum of mutations in a cohort of UK patients with ADA deficient SCID: Segregation of genotypes with specific ethnicities.

H. Bobby Gaspar, M. Madkaikar, H. Gaspar, 2015, Clinical immunology.

Primary Immunodeficiency Diseases

H. Gaspar, H. B. Gaspar, 2021, Medical Immunology.

Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector

Kathryn L. Parsley, R. Levinsky, R. Ali, 2004, The Lancet.

Pegademase bovine ( PeG-ADA ) for the treatment of infants and children with severe combined immunodeficiency ( SCID )

H. Gaspar, C. Booth, 2009 .

Modeling and Increasing the Genome Accessibility of Viral Integration Sites

Alessandro Aiuti, Steven J. Howe, Robin R. Ali, 2009 .

Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood?

A. Schulz, W. Friedrich, A. Gennery, 2012, Blood.

Update on treatment of human SCID-X1 by somatic gene therapy using a pseudotyped gammaretroviral vector

Kathryn L. Parsley, A. Thrasher, S. Howe, 2004 .

transplantation without GVHD in the secondary recipient tolerance permits rapid immune reconstitution following T-replete HLA-identical sibling pairs with congenital immunodeficiency: in vivo Serial transplantation of mismatched donor hematopoietic cells between

P. Veys, P. Amrolia, E. Davies, 2013 .

Impaired Epstein-Barr virus-specific CD8+ T-cell function in X-linked lymphoproliferative disease is restricted to SLAM family-positive B-cell targets.

S. Tangye, P. Rohrlich, A. Rickinson, 2010, Blood.

How I treat severe combined immunodeficiency.

P. Veys, P. Amrolia, W. Qasim, 2013, Blood.

Outcomes of splenectomy in patients with common variable immunodeficiency (CVID): a survey of 45 patients

R. Schmidt, A. Plebani, J. Litzman, 2013, Clinical and experimental immunology.

Erratum: Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction (Science Translational Medicine (2013) 5 (168er1))

P. Veys, S. Cooray, A. Thrasher, 2013 .

Gene Therapy Approaches to Immunodeficiency.

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