Browse by UCL people
Group by: Type | Date
Number of items: 61.
Article
Aydin, SE;
Freeman, AF;
Al-Herz, W;
Al-Mousa, HA;
Arnaout, RK;
Aydin, RC;
Barlogis, V;
... Inborn Errors Working Party of the European Group for Blood and; + view all
(2019)
Hematopoietic Stem Cell Transplantation as Treatment for Patients with DOCK8 Deficiency.
Journal of Allergy and Clinical Immunology: In Practice
, 7
(3)
pp. 848-855.
10.1016/j.jaip.2018.10.035.
|
|
Bacchelli, C;
Moretti, FA;
Carmo, M;
Adams, S;
Stanescu, HC;
Pearce, K;
Madkaikar, M;
... Gaspar, HB; + view all
(2017)
Mutations in linker for activation of T cells (LAT) lead to a novel form of severe combined immunodeficiency.
Journal of Allergy and Clinical Immunology
, 139
(2)
pp. 634-642.
10.1016/j.jaci.2016.05.036.
|
|
Booth, C;
Gaspar, HB;
(2009)
Pegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID).
Biologics
, 3
349 -358.
10.2147/BTT.S3103.
|
 |
|
Booth, C;
Gaspar, HB;
Thrasher, AJ;
(2016)
Treating Immunodeficiency through HSC Gene Therapy.
Trends In Molecular Medicine
, 22
(4)
pp. 317-327.
10.1016/j.molmed.2016.02.002.
|
|
Bousfiha, A;
Jeddane, L;
Picard, C;
Ailal, F;
Gaspar, HB;
Al-Herz, W;
Chatila, T;
... Sullivan, KE; + view all
(2018)
The 2017 IUIS Phenotypic Classification for Primary Immunodeficiencies.
Journal of Clinical Immunology
, 38
(1)
pp. 129-143.
10.1007/s10875-017-0465-8.
|
 |
|
Bradford, KL;
Moretti, FA;
Carbonaro-Sarracino, DA;
Gaspar, HB;
Kohn, DB;
(2017)
Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID): Molecular Pathogenesis and Clinical Manifestations.
Journal of Clinical Immunology
, 37
(7)
pp. 626-637.
10.1007/s10875-017-0433-3.
|
|
Brendel, C;
Rothe, M;
Santilli, G;
Charrier, S;
Stein, S;
Kunkel, H;
Abriss, D;
... Grez, M; + view all
(2018)
Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease.
Human Gene Therapy Clinical Development
, 29
(2)
pp. 69-79.
10.1089/humc.2017.245.
|
|
Carmo, M;
Montiel-Equihua, CA;
Alonso-Ferrero, ME;
Blundell, MP;
Thrasher, AJ;
Gaspar, HB;
Risma, KA;
... Baum, C; + view all
(2015)
Perforin gene transfer into hematopoietic stem cells improves immune dysregulation in murine models of perforin deficiency.
Molecular Therapy
, 23
(4)
737 - 745.
10.1038/mt.2014.242.
|
|
|
Chapel, H;
Prevot, J;
Gaspar, HB;
Español, T;
Bonilla, FA;
Solis, L;
Drabwell, J;
(2014)
Primary immune deficiencies - principles of care.
Front Immunol
, 5
, Article 627. 10.3389/fimmu.2014.00627.
|
|
|
Critchley, Bethan J;
Gaspar, H Bobby;
Benedetti, Sara;
(2023)
Targeting the Central Nervous System in Lysosomal Storage Diseases: Strategies to Deliver Therapeutics Across the Blood-Brain Barrier.
Molecular Therapy
, 31
(3)
pp. 657-675.
10.1016/j.ymthe.2022.11.015.
|
|
Day, James W;
Elfeky, Reem;
Nicholson, Bethany;
Goodman, Rupert;
Pearce, Rachel;
Fox, Thomas A;
Worth, Austen;
... Morris, Emma C; + view all
(2022)
Retrospective, Landmark Analysis of Long-term Adult Morbidity Following Allogeneic HSCT for Inborn Errors of Immunity in Infancy and Childhood.
Journal of Clinical Immunology
10.1007/s10875-022-01278-6.
(In press).
|
|
Debnath, S;
Jaako, P;
Siva, K;
Rothe, M;
Chen, J;
Dahl, M;
Gaspar, HB;
... Karlsson, S; + view all
(2017)
Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia.
Molecular Therapy
, 25
(8)
pp. 1805-1814.
10.1016/j.ymthe.2017.04.002.
|
|
Duncan, L;
Yilmaz, Z;
Gaspar, H;
Walters, R;
Goldstein, J;
Anttila, V;
Bulik-Sullivan, B;
... Bulik, CM; + view all
(2017)
Significant Locus and Metabolic Genetic Correlations Revealed in Genome-Wide Association Study of Anorexia Nervosa.
American Journal of Psychiatry
, 174
(9)
pp. 850-858.
10.1176/appi.ajp.2017.16121402.
|
|
|
Eichler, F;
Duncan, C;
Musolino, PL;
Orchard, PJ;
De Oliveira, S;
Thrasher, AJ;
Armant, M;
... Williams, DA; + view all
(2017)
Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.
The New England Journal of Medicine
, 377
pp. 1630-1638.
10.1056/NEJMoa1700554.
|
|
Elfeky, RA;
Furtado-Silva, JM;
Chiesa, R;
Rao, K;
Lucchini, G;
Amrolia, P;
Worth, A;
... Veys, P; + view all
(2018)
Umbilical cord blood transplantation without in vivo T-cell depletion for children with MHC class II deficiency.
Journal of Allergy and Clinical Immunology
, 141
(6)
2279-2282.e2.
10.1016/j.jaci.2017.10.051.
|
|
Ellison, SM;
Liao, A;
Wood, S;
Taylor, J;
Youshani, AS;
Rowlston, S;
Parker, H;
... Bigger, BW; + view all
(2019)
Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA.
Molecular Therapy: Methods & Clinical Development
, 13
pp. 399-413.
10.1016/j.omtm.2019.04.001.
|
|
|
Garcia-Perez, L;
Van Eggermond, M;
Van Roon, L;
Vloemans, SA;
Cordes, M;
Schambach, A;
Rothe, M;
... Pike-Overzet, K; + view all
(2020)
Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID.
Molecular Therapy: Methods & Clinical Development
, 17
pp. 666-682.
10.1016/j.omtm.2020.03.016.
|
|
|
Gaspar, HB;
(2017)
A Practical Guide to Implementing Population Newborn Screening (NBS) for Severe Combined Immunodeficiency (SCID).
International Journal of Neonatal Screening
, 3
(4)
, Article 29. 10.3390/ijns3040029.
|
|
|
Ghosh, S;
Carmo, M;
Calero-Garcia, M;
Ricciardelli, I;
Bustamante Ogando, JC;
Blundell, MP;
Schambach, A;
... Gaspar, HB; + view all
(2018)
T cell gene therapy for perforin deficiency corrects cytotoxicity defects and prevents Haemophagocytic Lymphohistiocytosis manifestations.
Journal of Allergy and Clinical Immunology
, 142
(3)
904-913.e3.
10.1016/j.jaci.2017.11.050.
|
|
Ghosh, S;
Gaspar, HB;
(2017)
Gene Therapy Approaches to Immunodeficiency.
Hematology/Oncology Clinics of North America
, 31
(5)
pp. 823-834.
10.1016/j.hoc.2017.05.003.
|
|
Hassan, A;
Lee, P;
Maggina, P;
Xu, JH;
Moreira, D;
Slatter, M;
Nademi, Z;
... Qasim, W; + view all
(2014)
Host natural killer immunity is a key indicator of permissiveness for donor cell engraftment in patients with severe combined immunodeficiency.
Journal of Allergy and Clinical Immunology
, 133
(6)
pp. 1660-1666.
10.1016/j.jaci.2014.02.042.
|
|
|
Hoenig, M;
Lagresle-Peyrou, C;
Pannicke, U;
Notarangelo, LD;
Porta, F;
Gennery, AR;
Slatter, M;
... Cavazzana, M; + view all
(2017)
Reticular dysgenesis: international survey on clinical presentation, transplantation, and outcome.
Blood
, 129
(21)
pp. 2928-2938.
10.1182/blood-2016-11-745638.
|
|
Hoenig, M;
Pannicke, U;
Gaspar, HB;
Schwarz, K;
(2017)
Recent advances in understanding the pathogenesis and management of reticular dysgenesis.
British Journal of Haematology
, 180
(5)
pp. 644-653.
10.1111/bjh.15045.
|
|
Ip, W;
Silva, JMF;
Gaspar, H;
Mitra, A;
Patel, S;
Rao, K;
Chiesa, R;
... Qasim, W; + view all
(2018)
Multicenter phase 1/2 application of adenovirus-specific T cells in high-risk pediatric patients after allogeneic stem cell transplantation.
Cytotherapy
, 20
(6)
pp. 830-838.
10.1016/j.jcyt.2018.03.040.
|
|
Izotova, N;
Rivat, C;
Baricordi, C;
Blanco, E;
Pellin, D;
Watt, E;
Gkazi, AS;
... Biasco, L; + view all
(2021)
Long-term lymphoid progenitors independently sustain naïve T and NK cell production in humans.
Nature Communications
, 12
, Article 1622. 10.1038/s41467-021-21834-9.
|
|
|
Kohn, DB;
Booth, C;
Shaw, KL;
Xu-Bayford, J;
Garabedian, E;
Trevisan, V;
Carbonaro-Sarracino, DA;
... Gaspar, HB; + view all
(2021)
Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.
New England Journal of Medicine
, 384
pp. 2002-2013.
10.1056/NEJMoa2027675.
|
|
Kohn, DB;
Booth, C;
Kang, EM;
Pai, S-Y;
Shaw, KL;
Santilli, G;
Armant, M;
... the Net4CGD consortium; + view all
(2020)
Lentiviral gene therapy for X-linked chronic granulomatous disease.
Nature Medicine
, 26
pp. 200-206.
10.1038/s41591-019-0735-5.
|
|
Kohn, DB;
Gaspar, HB;
(2017)
How We Manage Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA SCID).
Journal of Clinical Immunology
, 37
(4)
pp. 351-356.
10.1007/s10875-017-0373-y.
|
|
Kohn, DB;
Hershfield, MS;
Puck, JM;
Aiuti, A;
Blincoe, A;
Gaspar, HB;
Notarangelo, LD;
(2019)
Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency.
Journal of Allergy and Clinical Immunology
, 143
(3)
pp. 852-863.
10.1016/j.jaci.2018.08.024.
|
|
Kreins, AY;
Velasco, HF;
Cheong, K-N;
Rao, K;
Veys, P;
Worth, A;
Gaspar, HB;
(2021)
Long-Term Immune Recovery After Hematopoietic Stem Cell Transplantation for ADA Deficiency: a Single-Center Experience.
Journal of Clinical Immunology
10.1007/s10875-021-01145-w.
(In press).
|
|
|
Lorenzini, T;
Fliegauf, M;
Klammer, N;
Frede, N;
Proietti, M;
Bulashevska, A;
Camacho-Ordonez, N;
... Grimbacher, B; + view all
(2020)
Characterization of the clinical and immunologic phenotype and management of 157 individuals with 56 distinct heterozygous NFKB1 mutations.
Journal of Allergy and Clinical Immunology
, 146
(4)
pp. 901-911.
10.1016/j.jaci.2019.11.051.
|
|
Moretti, FA;
Giardino, G;
Attenborough, TCH;
Gkazi, AS;
Margetts, BK;
la Marca, G;
Fairbanks, L;
... Gaspar, HB; + view all
(2021)
Metabolite and thymocyte development defects in ADA-SCID mice receiving enzyme replacement therapy.
Scientific Reports
, 11
, Article 23221. 10.1038/s41598-021-02572-w.
|
|
|
Morris, EC;
Fox, T;
Chakraverty, R;
Tendeiro, R;
Snell, K;
Rivat, C;
Grace, S;
... Thrasher, AJ; + view all
(2017)
Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult.
Blood
, 130
pp. 1327-1335.
10.1182/blood-2017-04-777136.
|
|
Palendira, U;
Low, C;
Chan, A;
Hislop, AD;
Ho, E;
Phan, TG;
Deenick, E;
... Tangye, SG; + view all
(2011)
Molecular Pathogenesis of EBV Susceptibility in XLP as Revealed by Analysis of Female Carriers with Heterozygous Expression of SAP.
PLOS BIOLOGY
, 9
(11)
, Article e1001187. 10.1371/journal.pbio.1001187.
|
|
|
Panchal, N;
Houghton, B;
Diez, B;
Ghosh, S;
Ricciardelli, I;
Thrasher, AJ;
Gaspar, HB;
(2018)
Transfer of gene corrected T cells corrects humoral and cytotoxic defects in X-linked lymphoproliferative disease (XLP1).
Journal of Allergy and Clinical Immunology
, 142
(1)
235-245.e6.
10.1016/j.jaci.2018.02.053.
|
|
|
Passweg, JR;
Baldomero, H;
Bregni, M;
Cesaro, S;
Dreger, P;
Duarte, RF;
Falkenburg, JHF;
... Transpla, EGBM; + view all
(2013)
Hematopoietic SCT in Europe: data and trends in 2011.
Bone Marrow Transplantation
, 48
(9)
1161 -1167.
10.1038/bmt.2013.51.
|
|
|
Picard, C;
Bobby Gaspar, H;
Al-Herz, W;
Bousfiha, A;
Casanova, JL;
Chatila, T;
Crow, YJ;
... Sullivan, KE; + view all
(2018)
International Union of Immunological Societies: 2017 Primary Immunodeficiency Diseases Committee Report on Inborn Errors of Immunity.
Journal of Clinical Immunology
, 38
(1)
pp. 96-128.
10.1007/s10875-017-0464-9.
|
|
|
Piras, G;
Montiel-Equihua, C;
Chan, Y-KA;
Wantuch, S;
Stuckey, D;
Burke, D;
Prunty, H;
... Gaspar, HB; + view all
(2020)
Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease.
Molecular Therapy - Methods and Clinical Development
, 18
pp. 558-570.
10.1016/j.omtm.2020.07.001.
|
|
|
Poletti, V;
Charrier, S;
Corre, G;
Gjata, B;
Vignaud, A;
Zhang, F;
Rothe, M;
... Mavilio, F; + view all
(2018)
Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency.
Molecular Therapy - Methods and Clinical Development
, 9
pp. 257-269.
10.1016/j.omtm.2018.03.002.
|
|
|
Schejtman, A;
Vetharoy, W;
Choi, U;
Rivat, C;
Theobald, N;
Piras, G;
Leon-Rico, D;
... Santilli, G; + view all
(2021)
Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47phox-Deficient Chronic Granulomatous Disease.
Human Gene Therapy
, 32
(17-18)
pp. 949-958.
10.1089/hum.2020.276.
|
|
Schejtman, A;
Aragão-Filho, WC;
Clare, S;
Zinicola, M;
Weisser, M;
Burns, SO;
Booth, C;
... Santilli, G; + view all
(2020)
Lentiviral gene therapy rescues p47phox chronic granulomatous disease and the ability to fight Salmonella infection in mice.
Gene Therapy
10.1038/s41434-020-0164-6.
(In press).
|
|
|
Schwarzer, A;
Talbot, SR;
Selich, A;
Morgan, M;
Schott, JW;
Dittrich-Breiholz, O;
Bastone, AL;
... Rothe, M; + view all
(2021)
Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning.
Molecular Therapy
10.1016/j.ymthe.2021.06.017.
(In press).
|
|
|
Schwarzwaelder, K;
Howe, SJ;
Schmidt, M;
Brugman, MH;
Deichmann, A;
Glimm, H;
Schmidt, S;
... von Kalle, C; + view all
(2007)
Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo.
J CLIN INVEST
, 117
(8)
2241 - 2249.
10.1172/JCI31661.
|
|
|
Slack, J;
Albert, MH;
Balashov, D;
Belohradsky, BH;
Bertaina, A;
Bleesing, J;
Booth, C;
... Gennery, AR; + view all
(2018)
Outcome of hematopoietic cell transplantation for DNA double-strand break repair disorders.
Journal of Allergy and Clinical Immunology
, 141
(1)
322-328.e10.
10.1016/j.jaci.2017.02.036.
|
|
Slatter, MA;
Rao, K;
Abd Hamid, IJ;
Nademi, Z;
Chiesa, R;
Elfeky, R;
Pearce, MS;
... Veys, P; + view all
(2018)
Treosulfan, Fludarabine Conditioning for HSCT in Children with Primary Immunodeficiency: UK Experience.
Biology of Blood and Marrow Transplantation
, 24
(3)
pp. 529-536.
10.1016/j.bbmt.2017.11.009.
|
|
Speckmann, C;
Doerken, S;
Aiuti, A;
Albert, MH;
Al-Herz, W;
Allende, LM;
Scarselli, A;
... P-CID study of the Inborn Errors Working Party of the EBMT, .; + view all
(2017)
A prospective study on the natural history of patients with profound combined immunodeficiency: An interim analysis.
Journal of Allergy and Clinical Immunology
, 139
(4)
1302-1310.e4.
10.1016/j.jaci.2016.07.040.
|
|
|
Steele, CL;
Dore, M;
Ammann, S;
Loughrey, M;
Montero, A;
Burns, SO;
Morris, EC;
... Edgar, DM; + view all
(2016)
X-linked Inhibitor of Apoptosis Complicated by Granulomatous Lymphocytic Interstitial Lung Disease (GLILD) and Granulomatous Hepatitis.
Journal of Clinical Immunology
, 36
(7)
pp. 733-738.
10.1007/s10875-016-0320-3.
|
|
|
Thaventhiran, JED;
Lango Allen, H;
Burren, OS;
Rae, W;
Greene, D;
Staples, E;
Zhang, Z;
... Smith, KGC; + view all
(2020)
Whole-genome sequencing of a sporadic primary immunodeficiency cohort.
Nature
, 583
pp. 90-95.
10.1038/s41586-020-2265-1.
|
|
Titman, P;
Allwood, Z;
Gilmour, C;
Malcolmson, C;
Duran-Persson, C;
Cale, C;
Davies, G;
... Jones, A; + view all
(2014)
Quality of life in children with primary antibody deficiency.
J Clin Immunol
, 34
(7)
pp. 844-852.
10.1007/s10875-014-0072-x.
|
|
Topal, Joseph;
Panchal, Neelam;
Barroeta, Amairelys;
Roppelt, Anna;
Mudde, Annelotte;
Gaspar, H Bobby;
Thrasher, Adrian J;
... Booth, Claire; + view all
(2023)
Lentiviral Gene Transfer Corrects Immune Abnormalities in XIAP Deficiency.
Journal of Clinical Immunology
, 43
(2)
pp. 440-451.
10.1007/s10875-022-01389-0.
|
|
Tuijnenburg, P;
Lango Allen, H;
Burns, SO;
Greene, D;
Jansen, MH;
Staples, E;
Stephens, J;
... NIHR-BioResource – Rare Diseases Consortium, .; + view all
(2018)
Loss of function NFKB1 variants are the most common monogenic cause of CVID in Europeans.
Journal of Allergy and Clinical Immunology
, 142
(4)
pp. 1285-1296.
10.1016/j.jaci.2018.01.039.
|
|
|
Unni, MNM;
Elfeky, R;
Rao, K;
Nademi, Z;
Chiesa, R;
Amrolia, P;
Skinner, R;
... Slatter, MA; + view all
(2018)
Non-posttransplant lymphoproliferative disorder malignancy after hematopoietic stem cell transplantation in patients with primary immunodeficiency: UK experience.
Journal of Allergy and Clinical Immunology
, 141
(6)
2319-2321.e1.
10.1016/j.jaci.2018.02.038.
|
|
van der Burg, M;
Mahlaoui, N;
Gaspar, HB;
Pai, S-Y;
(2019)
Universal Newborn Screening for Severe Combined Immunodeficiency (SCID).
Frontiers in Pediatrics
, 7
, Article 373. 10.3389/fped.2019.00373.
|
|
|
Whitmore, KV;
Gaspar, HB;
(2016)
Adenosine Deaminase Deficiency - More Than Just an Immunodeficiency.
Frontiers in Immunology
, 7
(ARTN 314)
10.3389/fimmu.2016.00314.
|
|
Yeung, Jenny;
Liao, Aiyin;
Shaw, Matthew;
Silva, Soraia;
Vetharoy, Winston;
Rico, Diego Leon;
Kirby, Ian;
... Amrolia, Persis J; + view all
(2024)
Anti-CD45 PBD-based antibody-drug conjugates are effective targeted conditioning agents for gene therapy and stem cell transplant.
Molecular Therapy
10.1016/j.ymthe.2024.03.032.
(In press).
|
|
Zhan, H;
Gilmour, K;
Chan, L;
Farzaneh, F;
McNicol, AM;
Xu, JH;
Adams, S;
... Qasim, W; + view all
(2013)
Production and First-in-Man Use of T Cells Engineered to Express a HSVTK-CD34 Sort-Suicide Gene.
PLoS One
, 8
(10)
, Article e77106. 10.1371/journal.pone.0077106.
|
|
Conference item
|
Aragao-Filho, WC;
Shejtman, A;
Zinicola, M;
Siler, U;
Reichenbach, J;
Gaspar, HB;
Thrasher, A;
... Condino-Neto, A; + view all
(2017)
Development of a pCCLChim Lentiviral Vector for Gene Therapy of Patients with Chronic Granulomatous Disease (CGD) due to p47-phox Deficiency.
Presented at: Annual Meeting of the American Academy of Allergy, Asthma and Immunology (AAAAI), Atlanta, Georgia, USA.
|
|
Panchal, N;
Hougton, B;
Diez, B;
Thrasher, AJ;
Gaspar, HB;
Booth, C;
(2017)
T Cell Gene Therapy Corrects Humoral and Cytotoxic Defects in X-Linked Lymphoproliferative Disease (XLP).
Presented at: 20th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), Washington, DC, United States.
|
|
Poletti, V;
Charrier, S;
Corre, G;
Martin, S;
Gjata, B;
Vignaud, A;
Zhang, F;
... Mavilio, F; + view all
(2017)
Preclinical Development of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1).
Presented at: 20th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), Washington, DC, United States.
|
|
Shejtman, A;
Aragao Filho, WC;
Weisser, M;
Zinicola, M;
Booth, C;
Burns, SO;
Siler, U;
... Santilli, G; + view all
(2017)
Lentiviral Gene Therapy for p47(phox) Deficient Chronic Granulomatous Disease.
Presented at: 20th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), Washington, DC, United States.
|
Thesis
|
Calero, M;
(2017)
Novel gene therapy strategies for ADA deficiency using AAV vectors or gene editing.
Doctoral thesis , UCL (University College London).
|
