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Number of items: 113.

A

Acosta-Saltos, C; Gonitel, R; Acosta-Saltos, A; Chavda, S; Thrasher, A; Anderson, P; Raivich, G; (2012) Lentiviral GMCSF is neuroinflammatory and proregenerative. In: HUMAN GENE THERAPY. (pp. A17 - A18).

Aitchison, KL; Rahim, AA; Hughes, D; Kinnon, C; Waddington, SN; Howe, SJ; (2012) Developing lentiviral vectors for gene therapy of Type I Gaucher Disease. In: HUMAN GENE THERAPY. (pp. A15 - A16).

Alonso-Ferrero, ME; Bartolovic, K; Kinnon, C; Mata, M; Van Til, NP; Howe, SJ; (2012) Transient manipulation of haematopoietic stem cells with integrative deficient lentiviral vectors for improved cell expansion, survival and engraftment. In: HUMAN GENE THERAPY. (pp. A14 - A14).

Alonso-Ferrero, ME; Langford-Smith, A; Burke, D; Heales, S; Bigger, BW; Thrasher, A; Gaspar, B; (2012) Gene Therapy for Lysosomal Storage Diseases using an enhanced lentiviral vector delivery system. In: HUMAN GENE THERAPY. (pp. A14 - A14).

Anderson, J; Gustafsson, K; Himoudi, N; (2012) Licensing of killer dendritic cells in mouse and humans: functional similarities between IKDC and human blood γδ T-lymphocytes. J Immunotoxicol , 9 (3) 259 - 266. 10.3109/1547691X.2012.685528.

Andreu, N; Thomas, D; Saraiva, L; Ward, N; Gustafsson, K; Jayasinghe, SN; Robertson, BD; (2012) In vitro and in vivo interrogation of bio-sprayed cells. Small , 8 (16) 2495 - 2500. 10.1002/smll.201200138.

B

Bardelli, M; Zhang, F; Linden, RM; Thrasher, AJ; Henckaerts, E; (2012) AAV-Mediated Targeted Gene Addition in Patient-Specific SCID-X1 Induced Pluripotent Stem Cells. In: MOLECULAR THERAPY. (pp. S152 - S152).

Binny, C; McIntosh, J; Della Peruta, M; Kymalainen, H; Tuddenham, EGD; Buckley, SMK; ... Nathwani, AC; + view all (2012) AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage. BLOOD , 119 (4) 957 - 966. 10.1182/blood-2011-09-377630.

Booth, C; Algar, VE; Xu-Bayford, J; Fairbanks, L; Owens, C; Gaspar, HB; (2012) Non-infectious lung disease in patients with adenosine deaminase deficient severe combined immunodeficiency. J Clin Immunol , 32 (3) 449 - 453. 10.1007/s10875-012-9658-3.

Booth, C; Rivat, C; Blundell, M; Alonso-Ferrera, M; Thrasher, A; Gaspar, B; (2012) LENTIVIRAL VECTOR MEDIATED GENE THERAPY FOR X-LINKED LYMPHOPROLIFERATIVE DISEASE RESTORES HUMORAL AND CELLULAR FUNCTIONS. In: JOURNAL OF CLINICAL IMMUNOLOGY. (pp. 130 - 130).

Bouma, G; Carter, NA; Recher, M; Notarangelo, LD; Burns, SO; Mauri, C; Thrasher, AJ; (2012) DEFICIENCY OF REGULATORY B CELLS IN WISKOTT-ALDRICH SYNDROME PROTEIN DEFICIENT MICE LEADS TO EXACERBATED AUTOIMMUNE ARTHRITIS. In: JOURNAL OF CLINICAL IMMUNOLOGY. (pp. 12 - 12).

Bouma, G; Lang, PA; Shaabani, N; Borkens, S; Scheu, S; Booth, S; ... Lang, KS; + view all (2012) REDUCED TYPE I INTERFERON PRODUCTION BY DENDRITIC CELLS AND WEAKENED ANTIVIRAL IMMUNITY IN WISKOTT-ALDRICH SYNDROME PROTEIN DEFICIENCY. In: JOURNAL OF CLINICAL IMMUNOLOGY. (pp. 46 - 46).

Burns, S; Zenner, H; Plagnol, V; Curtis, J; Mok, K; Eisenhut, M; ... Nejentsev, S; + view all (2012) EXOME SEQUENCINGIDENTIFIES LRBA GENE DELETION IN A PATIENT PRESENTING WITH AUTOIMMUNITY WITHOUT HYPOGAMMAGLOBULINEMIA. In: JOURNAL OF CLINICAL IMMUNOLOGY. (pp. 398 - 398).

Burns, SO; Thrasher, AJ; Zenner, HL; Curtis, J; Nejentsev, S; Plagnol, V; ... Doffinger, R; + view all (2012) LRBA gene deletion in a patient presenting with autoimmunity without hypogammaglobulinemia. Journal of Allergy and Clinical Immunology , 130 (6) 1428 - 1432. 10.1016/j.jaci.2012.07.035.

Burns, SO; Zenner, HL; Plagnol, V; Curtis, J; Mok, K; Eisenhut, M; ... Nejentsev, S; + view all (2012) LRBA gene deletion in a patient presenting with autoimmunity without hypogammaglobulinemia. J Allergy Clin Immunol , 130 (6) 1428 - 1432. 10.1016/j.jaci.2012.07.035.

C

Carbonaro, DA; Jin, X; Geiger, S; Senadheera, S; Montiel-Equihua, C; Gaspar, B; ... Kohn, DB; + view all (2012) Development of a Lentiviral Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immunodeficiency (ADA-SCID). In: MOLECULAR THERAPY. (pp. S162 - S162).

Carmo, M; Arumugam, P; Montiel-Equiha, C; Alonso-Ferrero, M; Schambach, A; Baum, C; ... Gaspar, B; + view all (2012) DEVELOPMENT OF GENE THERAPY FOR HAEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS (HLH) DUE TO PERFORIN DEFICIENCY. In: JOURNAL OF CLINICAL IMMUNOLOGY. (pp. 131 - 131).

Carmo, M; Arumugam, P; Montiel-Equihua, CA; Alonso-Ferrero, ME; Schambach, A; Baum, C; ... Gaspar, B; + view all (2012) Development of gene therapy for hemophagocytic lymphohistiocytosis (HLH) due to perforin deficiency. In: HUMAN GENE THERAPY. (pp. A3 - A3).

Carmo, M; Arumugam, P; Montiel-Equihua, CA; Alonso-Ferrero, ME; Tiwari, S; Schambach, A; ... Gaspar, B; + view all (2012) Development of Gene Therapy for Hemophagocytic Lymphohistiocytosis (HLH) Due to Perforin Deficiency. In: MOLECULAR THERAPY. (pp. S125 - S126).

Carmo, M; Arumugam, P; Tiwari, S; Thrasher, A; Risma, K; Malik, P; ... Jordan, M; + view all (2012) GENE THERAPY FOR HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS (HLH): FIXING A CRITICAL 'CIRCUIT BREAKER' IN THE IMMUNE SYSTEM. In: BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION. (pp. S201 - S201).

Carpenter, B; Ghorashian, S; Nicholson, E; Griffin, J; Ahmadi, M; Zhang, L; ... Chakraverty, R; + view all (2012) Targeting therapeutic T cells to tumour niches. In: IMMUNOLOGY. (pp. 53 - 54).

Carpenter, B; Ghorashian, S; Nicholson, E; Griffin, JE; Ahmadi, M; Holler, A; ... Chakraverty, R; + view all (2012) Targeting Therapeutic T Cells to Tumour Niches. In: BLOOD.

Chan, E; (2012) Lentiviral gene therapy for HIV using TRIM-cyclophilin restriction factors. Doctoral thesis, UCL (University College London). Green open access
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Chan, E; Schaller, T; Eddaoudi, A; Zhan, H; Tan, CP; Jacobsen, M; ... Qasim, W; + view all (2012) Lentiviral Gene Therapy Against Human Immunodeficiency Virus Type 1, Using a Novel Human TRIM21-Cyclophilin A Restriction Factor. HUMAN GENE THERAPY , 23 (11) 1176 - 1185. 10.1089/hum.2012.083.

Chan, E; Schaller, T; Eddaoudi, A; Zhan, H; Tan, CP; Jacobsen, M; ... Qasim, W; + view all (2012) Lentiviral gene therapy against human immunodeficiency virus type 1, using a novel human TRIM21-cyclophilin A restriction factor. In: Hum Gene Ther. (pp. 1176 - 1185). Green open access
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Charrier, S; Blundell, M; Cedrone, G; Louache, F; Vainchenker, W; Thrasher, A; Galy, A; (2012) Efficient mobilization and transduction of WASP-deficient haematopoietic cells in a murine model. In: HUMAN GENE THERAPY. (pp. A111 - A111).

Chick, HE; Nowrouzi, A; Fronza, R; McDonald, RA; Kane, NM; Alba, R; ... Baker, AH; + view all (2012) Integrase-Deficient Lentiviral Vectors Mediate Efficient Gene Transfer to Human Vascular Smooth Muscle Cells with Minimal Genotoxic Risk. HUMAN GENE THERAPY , 23 (12) 1247 - 1257. 10.1089/hum.2012.042.

Chiesa, R; Gilmour, K; Qasim, W; Adams, S; Worth, AJ; Zhan, H; ... Veys, P; + view all (2012) Omission of in vivo T-cell depletion promotes rapid expansion of naïve CD4+ cord blood lymphocytes and restores adaptive immunity within 2 months after unrelated cord blood transplant. Br J Haematol , 156 (5) 656 - 666. 10.1111/j.1365-2141.2011.08994.x.

Chiesa, R; Gilmour, K; Qasim, W; Adams, S; Worth, AJJ; Zhan, H; ... Veys, P; + view all (2012) Omission of in vivo T-cell depletion promotes rapid expansion of naive CD4(+) cord blood lymphocytes and restores adaptive immunity within 2 months after unrelated cord blood transplant. BRITISH JOURNAL OF HAEMATOLOGY , 156 (5) 656 - 666. 10.1111/j.1365-2141.2011.08994.x.

Chiew, M-L; Ballard, S; Stauss, H; Thrasher, A; Longhurst, H; Workman, S; (2012) WORLD IMMUNOLOGY DAY 2012 EDUCATIONAL SYMPOSIUM FOR CLINICIANS AND PATIENTS. In: JOURNAL OF CLINICAL IMMUNOLOGY. (pp. 26 - 26).

Cole, TS; Jones, LKR; McGrogan, P; Pearce, MS; Flood, TJ; Cant, AJ; ... Titman, P; + view all (2012) Emotional and behavioural difficulties in chronic granulomatous disease. ARCHIVES OF DISEASE IN CHILDHOOD , 97 (1) 87 - 87. 10.1136/archdischild-2011-300780.

Cooper, N; Rao, K; Goulden, N; Amrolia, P; Veys, P; (2012) Alpha interferon augments the graft-versus-leukaemia effect of second stem cell transplants and donor lymphocyte infusions in high-risk paediatric leukaemias. Br J Haematol , 156 (4) 550 - 552. 10.1111/j.1365-2141.2011.08889.x.

Cooray, S; Howe, SJ; Thrasher, AJ; (2012) Retrovirus and lentivirus vector design and methods of cell conditioning. Methods Enzymol , 507 29 - 57. 10.1016/B978-0-12-386509-0.00003-X.

D

Davies, EG; Gilmour, KC; Parsley, K; Curry, J; Sebire, N; Poliani, L; ... Thrasher, AJ; + view all (2012) THYMUS TRANSPLANTATION FOR COMPLETE DIGEORGE SYNDROME: THE LONDON EXPERIENCE. In: JOURNAL OF CLINICAL IMMUNOLOGY. (pp. 12 - 13).

Di, WL; Semenova, E; Larcher, F; Del Rio, M; Harper, JI; Thrasher, AJ; Qasim, W; (2012) Human involucrin promoter mediates repression-resistant and compartment-specific LEKTI expression. Hum Gene Ther , 23 (1) 83 - 90. 10.1089/hum.2011.091.

Dignan, FL; Clark, A; Amrolia, P; Cornish, J; Jackson, G; Mahendra, P; ... British Society for Blood and Marrow Transplantation,; + view all (2012) Diagnosis and management of acute graft-versus-host disease. Br J Haematol , 158 (1) 30 - 45. 10.1111/j.1365-2141.2012.09129.x.

Dignan, FL; Greenblatt, D; Scarisbrick, JJ; Potter, MN; Shaw, BE; Cox, M; ... Medd, P; + view all (2012) Efficacy of bimonthly extracorporeal photopheresis in refractory chronic mucocutaneous GVHD. Bone Marrow Transplantation , 47 (6) 824 - 830. 10.1038/bmt.2011.186.

Dignan, FL; Potter, MN; Shaw, BE; Scarisbrick, JJ; Cornish, J; Clark, A; ... Hadzic, N; + view all (2012) Organ-specific management and supportive care in chronic graft-versus-host disease. British Journal of Haematology , 158 (1) 62 - 78. 10.1111/j.1365-2141.2012.09131.x.

Dignan, FL; Shaw, BE; Potter, MN; Amrolia, P; Clark, A; Cornish, J; ... Taylor, PC; + view all (2012) Diagnosis and management of chronic graft-versus-host disease. British Journal of Haematology , 158 (1) 46 - 61. 10.1111/j.1365-2141.2012.09128.x.

E

Engelhardt, KR; Gertz, EM; Keles, S; Schaeffer, AA; Ceja, R; Sassi, A; ... Grimbacher, B; + view all (2012) DOCK8 DEFICIENCY AND A DIAGNOSTIC SCORE TO DIFFERENTIATE IT FROM OTHER HYPER-IGE SYNDROMES. In: JOURNAL OF CLINICAL IMMUNOLOGY. (pp. 332 - 333).

F

Field, A-C; Stauss, HJ; Morris, EC; Qasim, W; (2012) Comparison on lentiviral and Sleeping Beauty transposon-mediated gene expression of T cell receptor genes. In: HUMAN GENE THERAPY. (pp. A10 - A11).

G

Gaspar, B; Carmo, M; Arumugam, P; Alonso-Ferrero, M; Schambach, A; Baum, C; ... Thrasher, A; + view all (2012) Development of Gene therapy for HLH due to perforin deficiency and for XLP1. In: HUMAN GENE THERAPY. (pp. A9 - A9).

Gaspar, HB; (2012) Gene therapy for ADA-SCID: defining the factors for successful outcome. Blood , 120 (18) 3628 - 3629. 10.1182/blood-2012-08-446559.

Georgiadis, C; Semenova, K; McGrath, J; Di, W; Qasim, W; (2012) Development of lentiviral gene therapy for type VII collagen deficient epidermolysis bullosa. In: HUMAN GENE THERAPY. (pp. A9 - A9).

Georgiadis, C; Thrasher, A; South, AP; McGrath, J; Qasim, W; Di, W; (2012) Development of lentiviral gene therapy for type VII collagen deficient epidermolysis bullosa. In: JOURNAL OF INVESTIGATIVE DERMATOLOGY. (pp. S96 - S96).

Ghani, S; Di, W-L; Semenova, E; Chan, L; Farzaneh, F; Qasim, W; (2012) Clinical Grade Lentiviral Vector Production for the Inherited Skin Disease, Netherton Syndrome. In: MOLECULAR THERAPY. (pp. S164 - S164).

Ghorashian, S; Carpenter, B; Holler, A; Nicholson, E; Ahmadi, M; Zech, M; ... Chakraverty, R; + view all (2012) CD4 Cells Engineered to Express an MHC Class I Restricted TCR Can Rescue CD8 Cells Tolerized to Tumour-Associated Antigens. In: BLOOD.

Ghorashian, S; Holler, A; Carpenter, B; Ahmadi, M; Nicholson, E; Zech, M; ... Chakraverty, R; + view all (2012) CD4 cells expressing an MHC class I restricted TCR can rescue CD8 cells tolerized to tumour-associated antigen. In: IMMUNOLOGY. (pp. 707 - 707).

Ghorashian, S; Holler, A; Flutter, B; Xue, S-A; Fallah-Arani, F; Sivakumaran, J; ... Chakraverty, R; + view all (2012) CD4 help can overcome tolerance developing in adoptively-transferred T cells transduced with a self-specific TCR. In: HUMAN GENE THERAPY. (pp. A6 - A6).

Guasti, L; Prasongchean, W; Kleftouris, G; Mukherjee, S; Thrasher, AJ; Bulstrode, NW; Ferretti, P; (2012) High plasticity of pediatric adipose tissue-derived stem cells: too much for selective skeletogenic differentiation? Stem Cells Transl Med , 1 (5) 384 - 395. 10.5966/sctm.2012-0009.

H

Hassan, A; Booth, C; Brightwell, A; Allwood, Z; Veys, P; Rao, K; ... Inborn Errors Working Party of the European Group for Blood and Marrow Transplantation and European Society for Immunodeficiency,; + view all (2012) Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency. Blood , 120 (17) 3615 - 3624. 10.1182/blood-2011-12-396879.

Himoudi, N; Morgenstern, DA; Yan, M; Vernay, B; Saraiva, L; Wu, Y; ... Anderson, J; + view all (2012) Human γδ T lymphocytes are licensed for professional antigen presentation by interaction with opsonized target cells. J Immunol , 188 (4) 1708 - 1716. 10.4049/jimmunol.1102654.

Himoudi, N; Wallace, R; Parsley, KL; Gilmour, K; Barrie, AU; Howe, K; ... Anderson, J; + view all (2012) Lack of T-cell responses following autologous tumour lysate pulsed dendritic cell vaccination, in patients with relapsed osteosarcoma. Clin Transl Oncol , 14 (4) 271 - 279. 10.1007/s12094-012-0795-1.

Howe, SJ; Chandrashekran, A; (2012) Vector systems for prenatal gene therapy: principles of retrovirus vector design and production. Methods Mol Biol , 891 85 - 107. 10.1007/978-1-61779-873-3_5.

I

Ip, W; Gaspar, H; Davies, EG; Veys, P; Qasim, W; (2012) ALLOGENEIC HAEMATOPOIETIC STEM CELL TRANSPLANTATION FOR IMMUNODEFICIENCY DUE TO DEFECTIVE RIBONUCLEASE MITOCHONDRIAL RNA PROCESSING. In: JOURNAL OF CLINICAL IMMUNOLOGY. (pp. 326 - 326).

J

Jonuschies, J; Boldrin, L; Thrasher, A; Muntoni, F; Morgan, J; (2012) Assessment of potential promoters for lentiviral gene therapy in DMD. In: NEUROMUSCULAR DISORDERS. (pp. S8 - S8).

K

Kallinikou, K; Anjos-Afonso, F; Blundell, MP; Ings, SJ; Watts, MJ; Thrasher, AJ; ... Yong, KL; + view all (2012) Engraftment defect of cytokine-cultured adult human mobilized CD34(+) cells is related to reduced adhesion to bone marrow niche elements. Br J Haematol , 158 (6) 778 - 787. 10.1111/j.1365-2141.2012.09219.x.

Kenny, GD; Villegas-Llerena, C; Tagalakis, AD; Campbell, F; Welser, K; Botta, M; ... Hart, SL; + view all (2012) Multifunctional receptor-targeted nanocomplexes for magnetic resonance imaging and transfection of tumours. Biomaterials , 33 (29) 7241 - 7250. 10.1016/j.biomaterials.2012.06.042. Green and gold open access
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Knight, S; Zhang, F; Mueller-Kuller, U; Bokhoven, M; Gupta, A; Broughton, T; ... Takeuchi, Y; + view all (2012) Safer, silencing-resistant lentiviral vectors: optimization of the ubiquitous chromatin-opening element through elimination of aberrant splicing. J Virol , 86 (17) 9088 - 9095. 10.1128/JVI.00485-12. Gold open access

L

Langford-Smith, A; Wilkinson, FL; Langford-Smith, KJ; Holley, RJ; Sergijenko, A; Howe, SJ; ... Bigger, BW; + view all (2012) Hematopoietic Stem Cell and Gene Therapy Corrects Primary Neuropathology and Behavior in Mucopolysaccharidosis IIIA Mice. MOLECULAR THERAPY , 20 (8) 1610 - 1621. 10.1038/mt.2012.82.

Leon-Rico, D; Aldea, M; Sanchez, R; Segovia, JC; Santilli, G; Thrasher, AJ; ... Almarza, E; + view all (2012) Itgb2 hypomorphic mice: a novel approach for ex vivo LAD gene therapy. In: HUMAN GENE THERAPY. (pp. A112 - A112).

Levin, MC; Lidberg, U; Jirholt, P; Adiels, M; Wramstedt, A; Gustafsson, K; ... Gjertsson, I; + view all (2012) Evaluation of macrophage-specific promoters using lentiviral delivery in mice. Gene Ther , 19 (11) 1041 - 1047. 10.1038/gt.2011.195.

Lowdell, MW; Birchall, M; Thrasher, AJ; (2012) Use of compassionate-case ATMP in preclinical data for clinical trial applications. Lancet , 379 (9834) 2341 - ?. 10.1016/S0140-6736(12)61018-6.

Lugthart, G; Albon, SJ; Ricciardelli, I; Kester, MG; Meij, P; Lankester, AC; Amrolia, PJ; (2012) Simultaneous generation of multivirus-specific and regulatory T cells for adoptive immunotherapy. J Immunother , 35 (1) 42 - 53. 10.1097/CJI.0b013e31823569e2.

M

Macpherson, L; Monypenny, J; Blundell, MP; Cory, GO; Tomé-García, J; Thrasher, AJ; ... Calle, Y; + view all (2012) Tyrosine phosphorylation of WASP promotes calpain-mediated podosome disassembly. Haematologica , 97 (5) 687 - 691. 10.3324/haematol.2011.048868.

Moeendarbary, E; Valon, L; Harris, A; Moulding, D; Thrasher, A; Stride, E; ... Charras, G; + view all (2012) Cellular Rheology and Hydraulics. BIOPHYSICAL JOURNAL , 102 (3) 563A - 563A.

Mohanty, ST; Cairney, CJ; Chantry, AD; Madan, S; Fernandes, JA; Howe, SJ; ... Bellantuono, I; + view all (2012) A Small Molecule Modulator of Prion Protein Increases Human Mesenchymal Stem Cell Lifespan, Ex Vivo Expansion, and Engraftment to Bone Marrow in NOD/SCID Mice. STEM CELLS , 30 (6) 1134 - 1143. 10.1002/stem.1065.

Montiel-Equihua, CA; Thrasher, AJ; Gaspar, HB; (2012) Gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency. Curr Gene Ther , 12 (1) 57 - 65.

Montiel-Equihua, CA; Zhang, L; Carmo, M; Alonso-Ferrero, ME; Blundell, MP; Monkeviciute, A; ... Fairbanks, L; + view all (2012) 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. Molecular Therapy , 20 (7) 1400 - 1409. 10.1038/mt.2012.50.

Montiel-Equihua, CA; Zhang, L; Knight, S; Saadeh, H; Scholz, S; Carmo, M; ... Gaspar, HB; + view all (2012) Development of a gene expression system for enhanced erythroid expression. In: HUMAN GENE THERAPY. (pp. A11 - A11).

Montiel-Equihua, CA; Zhang, L; Knight, S; Saadeh, H; Scholz, S; Carmo, M; ... Gaspar, HB; + view all (2012) 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. Mol Ther , 20 (7) 1400 - 1409. 10.1038/mt.2012.50.

Moschidou, D; Jones, GN; Abdulrazzak, H; Phoolchund, A; Lay, K; Ramasamy, TS; ... Atala, A; + view all (2012) Valproic acid confers functional pluripotency to human amniotic fluid stem cells in a transgene-free approach. Molecular Therapy , 20 (10) 1953 - 1967. 10.1038/mt.2012.117.

Moschidou, D; Mukherjee, S; Blundell, MP; Drews, K; Jones, GN; Abdulrazzak, H; ... Guillot, PV; + view all (2012) Valproic acid confers functional pluripotency to human amniotic fluid stem cells in a transgene-free approach. Mol Ther , 20 (10) 1953 - 1967. 10.1038/mt.2012.117.

Moulding, DA; Moeendarbary, E; Valon, L; Record, J; Charras, GT; Thrasher, AJ; (2012) Excess F-actin mechanically impedes mitosis leading to cytokinesis failure in X-linked neutropenia by exceeding Aurora B kinase error correction capacity. Blood , 120 (18) 3803 - 3811. 10.1182/blood-2012-03-419663.

Mukherjee, S; Pipino, C; Sung, P; DeCoppi, P; Thrasher, A; (2012) Investigating perturbed haematopoiesis in Down Syndrome patients: an induced pluripotent stem cell approach. In: HUMAN GENE THERAPY. (pp. A8 - A8).

Munye, MM; Howe, SJ; Hart, SL; (2012) Towards gene therapy for primary ciliary dyskinesia. In: HUMAN GENE THERAPY. (pp. A11 - A11).

N

Nicholson, E; Ghorashian, S; Stauss, H; (2012) Improving TCR Gene Therapy for Treatment of Haematological Malignancies. Adv Hematol , 2012 404081 - ?. 10.1155/2012/404081. Gold open access

Nicholson, EK; Ahmadi, M; Holler, A; Pike, R; Carpenter, B; Thomas, S; ... Morris, E; + view all (2012) Enhancing the Efficacy of T Cell Receptor (TCR) Gene Therapy by Co-Transfer of TCR and Additional CD3 Molecules Into CD4+T Cells. In: BLOOD.

Nicholson, EK; Ahmadi, M; Holler, A; Pike, R; Carpenter, B; Thomas, S; ... Morris, E; + view all (2012) Enhancing the Efficacy of T Cell Receptor (TCR) Gene Therapy by Co-Transfer of TCR and Additional CD3 Molecules Into CD4+T Cells. In: BLOOD.

P

Pallant, C; Cathomen, T; Dannemann, N; Qasim, W; Naldini, L; Kinnon, C; ... Thrasher, A; + view all (2012) Targeting models of severe combined immunodeficiency for correction by zinc finger nucleases. In: HUMAN GENE THERAPY. (pp. A7 - A7).

Pantoglou, J; Abdul-Razak, HH; Rocca, C; Howe, SJ; Gregory, PD; Holmes, MC; ... Yanez-Munoz, RJ; + view all (2012) Specificity of a zinc-finger nuclease targeting mouse Prkdc: preparation of samples for analysis of off-target cutting. In: HUMAN GENE THERAPY. (pp. A18 - A18).

Pearson, K; Taylor, C; Basu, P; Mussai, F; Santilli, G; Sala, A; Anderson, J; (2012) FUNCTIONALLY ACTIVE MYELOID DERIVED SUPPRESSOR CELLS (MDSCS) ARE FOUND WITHIN THE BLOOD OF PATIENTS WITH NEUROBLASTOMA. PEDIATRIC BLOOD & CANCER , 59 (6) 1055 - 1055.

Pike-Overzet, K; Rodijk, M; Ng, YY; Baert, M; Lagresle-Peyrou, C; Schambach, A; ... Staal, F; + view all (2012) SELF-INACTIVATING LENTIVIRAL VECTORS FOR CORRECTION OF RAG1 SEVERE COMBINED IMMUNODEFICIENCY. In: EXPERIMENTAL HEMATOLOGY. (pp. S46 - S47).

Pike-Overzet, K; Rodijk, M; Ng, YY; Baert, MRM; Lagresle-Peyrou, C; Schambach, A; ... Staal, FJT; + view all (2012) Self-Inactivating Lentiviral Vectors for Correction of Rag1 Severe Combined Immunodeficiency. In: HUMAN GENE THERAPY. (pp. A42 - A42).

Plagnol, V; Curtis, J; Epstein, M; Mok, K; Stebbings, E; Grigoriadou, S; ... Nejentsev, S; + view all (2012) NEW POWERFUL SOFTWARE FOR COPY NUMBER ANALYSIS OF EXOME SEQUENCE DATA, EXOMEDEPTH, DISCOVERS NOVEL MUTATIONS IN PRIMARY IMMUNODEFICIENCY PATIENTS. In: JOURNAL OF CLINICAL IMMUNOLOGY. (pp. 216 - 216).

Plagnol, V; Curtis, J; Epstein, M; Mok, KY; Stebbings, E; Grigoriadou, S; ... Nejentsev, S; + view all (2012) A robust model for read count data in exome sequencing experiments and implications for copy number variant calling. Bioinformatics , 28 (21) 2747 - 2754. 10.1093/bioinformatics/bts526. Gold open access
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Pugh, JAT; Cox, AG; McLeod, CW; Bunch, J; Writer, MJ; Hart, SL; ... Bell, J; + view all (2012) Elemental imaging of MRI contrast agents: benchmarking of LA-ICP-MS to MRI. ANALYTICAL AND BIOANALYTICAL CHEMISTRY , 403 (6) 1641 - 1649. 10.1007/s00216-012-5973-z.

R

Recher, M; Burns, SO; de la Fuente, MA; Volpi, S; Dahlberg, C; Walter, JE; ... Notarangelo, LD; + view all (2012) B cell-intrinsic deficiency of the Wiskott-Aldrich syndrome protein (WASp) causes severe abnormalities of the peripheral B-cell compartment in mice. Blood , 119 (12) 2819 - 2828. 10.1182/blood-2011-09-379412.

Rivat, C; Booth, C; Blundell, M; Thrasher, AJ; Gaspar, HB; (2012) Lentiviral vector mediated gene therapy for X-linked lymphoproliferative disease (XLP1) restores humoral and cellular functions. In: HUMAN GENE THERAPY. (pp. A8 - A8).

Rivat, C; Booth, C; Blundell, M; Thrasher, AJ; Gaspar, HB; (2012) Lentiviral Vector Mediated Gene Therapy for X-Linked Lymphoproliferative Disease Restores Humoral and Cellular Functions. In: MOLECULAR THERAPY. (pp. S87 - S88).

Rivat, C; Santilli, G; Gaspar, HB; Thrasher, AJ; (2012) Gene therapy for primary immunodeficiencies. Hum Gene Ther , 23 (7) 668 - 675. 10.1089/hum.2012.116. Gold open access

S

Samarasinghe, S; Steward, C; Hiwarkar, P; Saif, MA; Hough, R; Webb, D; ... Veys, P; + view all (2012) Excellent outcome of matched unrelated donor transplantation in paediatric aplastic anaemia following failure with immunosuppressive therapy: a United Kingdom multicentre retrospective experience. Br J Haematol , 157 (3) 339 - 346. 10.1111/j.1365-2141.2012.09066.x.

Semenova, E; Ghani, S; Chan, L; Farzaneh, F; Larcher, F; Harper, J; ... Di, WL; + view all (2012) Clinical grade lentiviral vector production for inherited skin disease. In: BRITISH JOURNAL OF DERMATOLOGY. (pp. e26 - e26).

Seymour, LW; Thrasher, AJ; (2012) Gene therapy matures in the clinic. NATURE BIOTECHNOLOGY , 30 (7) 588 - 593. 10.1038/nbt.2290.

Shaw, SWS; David, AL; Blundell, M; Howe, S; Pipino, C; Maghsoudlou, P; ... De Coppi, P; + view all (2012) CD34+cells derived from sheep amniotic fluid could engraft in NOD-SCID gamma mice and in lambs after prenatal autologous transplantation. Presented at: UNSPECIFIED.

Slatter, M; Rao, K; Chiesa, R; Amrolia, P; Nademi, Z; Flood, T; ... Veys, P; + view all (2012) TREOSULFAN, FLUDARABINE AND ALEMTUZUMAB CONDITIONING FOR HEMATOPOIETIC STEM CELL TRANSPLANTATION IN CHILDREN WITH PRIMARY IMMUNODEFICIENCY: UK EXPERIENCE. In: BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION. (pp. S234 - S234).

Sung, P; Mukherjee, S; Thrasher, AJ; (2012) Lineage-specific haematopoietic differentiation of human induced pluripotent stem cells. In: HUMAN GENE THERAPY. (pp. A5 - A5).

T

Tagalakis, AD; Kenny, G; Saraiva, L; McCarthy, D; Hart, SL; (2012) Development of Anionic Nanocomplexes for Targeted SiRNA and Plasmid DNA Delivery. In: MOLECULAR THERAPY. (pp. S248 - S248).

Tolar, J; Becker, PS; Clapp, DW; Hanenberg, H; Diaz de Heredia, C; Kiem, H-P; ... Bueren, J; + view all (2012) Gene Therapy for Fanconi Anemia: One Step Closer to the Clinic. HUMAN GENE THERAPY , 23 (2) 141 - 144. 10.1089/hum.2011.237.

U

Uttenthal, BJ; Carpenter, B; Ghorashian, S; Wright, GP; Flutter, B; Xue, SA; ... Morris, EC; + view all (2012) Adoptive therapy with redirected MHC class I-restricted antigen-specific regulatory T cells is a potential novel cellular therapy for graft-versus-host disease. In: BRITISH JOURNAL OF HAEMATOLOGY. (pp. 8 - 8).

V

Van Der Loo, JCM; Grassman, E; Terwilliger, A; Higashimoto, T; Nordling, DL; Malik, P; ... Reeves, L; + view all (2012) Scale-up and manufacturing of clinical-grade self-inactivating γ-retroviral vectors by transient transfection. Gene Therapy , 19 (3) 246 - 254. 10.1038/gt.2011.102.

Van Der Loo, JCM; Grassman, E; Terwilliger, A; Higashimoto, T; Nordling, DL; Malik, P; ... Reeves, L; + view all (2012) Critical Variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency. Gene Therapy , 19 (8) 872 - 876. 10.1038/gt.2012.37.

van der Loo, JCM; Swaney, WP; Grassman, E; Terwilliger, A; Higashimoto, T; Schambach, A; ... Malik, P; + view all (2012) Scale-up and manufacturing of clinical-grade self-inactivating gamma-retroviral vectors by transient transfection. GENE THERAPY , 19 (3) 246 - 254. 10.1038/gt.2011.102.

van der Loo, JCM; Swaney, WP; Grassman, E; Terwilliger, A; Higashimoto, T; Schambach, A; ... Malik, P; + view all (2012) Critical Variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency. GENE THERAPY , 19 (8) 872 - 876. 10.1038/gt.2012.37.

van Til, NP; de Boer, H; Huston, M; Fontana, E; Poliani, PL; Cassani, B; ... Wagemaker, G; + view all (2012) Correction of Immunity in Murine Rag2 Severe Combined Immunodeficiency by Lentiviral Gene Therapy Using a Codon-Optimized RAG2 Therapeutic Transgene. In: MOLECULAR THERAPY. (pp. S90 - S91).

van Til, NP; de Boer, H; Mashamba, N; Wabik, A; Huston, M; Visser, TP; ... Wagemaker, G; + view all (2012) Correction of Murine Rag2 Severe Combined Immunodeficiency by Lentiviral Gene Therapy Using a Codon-optimized RAG2 Therapeutic Transgene. MOLECULAR THERAPY , 20 (10) 1968 - 1980. 10.1038/mt.2012.110.

Vetrie, D; Vořechovský, I; Sideras, P; Holland, J; Davies, A; Flinter, F; ... Bentley, DR; + view all (2012) The gene involved in X-linked agammaglobulinaemia is a member of the Src family of protein-tyrosine kinases. 1993. J Immunol , 188 (7) 2948 - 2955.

Vink, CA; Howe, SJ; (2012) Epigenetics of integration-deficient lentiviral vectors. In: HUMAN GENE THERAPY. (pp. A13 - A13).

W

Ward, N; Buckley, S; Rahim, A; McVey, J; Thrasher, A; Waddington, S; (2012) Codon optimised FVIII constructs for treatment of haemophilia A. In: HUMAN GENE THERAPY. (pp. A17 - A17).

White, K; Kane, NM; Milligan, G; Baker, AH; (2012) The role of miRNA in stem cell pluripotency and commitment to the vascular endothelial lineage. Microcirculation , 19 (3) 196 - 207. 10.1111/j.1549-8719.2012.00161.x.

Wootten, V; Goulden, N; Veys, P; Qasim, W; (2012) A double cure: Omenn syndrome and β thalassaemia successfully treated with mismatched unrelated donor transplantation. Bone Marrow Transplant , 47 (4) 589 - 590. 10.1038/bmt.2011.115.

Writer, MJ; Kyrtatos, PG; Bienemann, AS; Pugh, JA; Lowe, AS; Villegas-Llerena, C; ... Hart, SL; + view all (2012) Lipid peptide nanocomplexes for gene delivery and magnetic resonance imaging in the brain. Journal of Controlled Release , 162 (2) 340 - 348. 10.1016/j.jconrel.2012.07.002. Green and gold open access
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Z

Zhang, F; Knight, S; Collins, M; Takeuchi, Y; Antoniou, M; Thrasher, AJ; (2012) Improved safety of Lentiviral vectors containing silencing resistant A2UCOE regulatory elements. In: HUMAN GENE THERAPY. (pp. A9 - A9).

This list was generated on Sun Jul 20 04:21:26 2014 BST.