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Number of items: 104.

A

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

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

Alonso-Ferrero, ME and Bartolovic, K and Kinnon, C and Mata, M and Van Til, NP and 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 and Langford-Smith, A and Burke, D and Heales, S and Bigger, BW and Thrasher, A and 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 and Gustafsson, K and 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 and Thomas, D and Saraiva, L and Ward, N and Gustafsson, K and Jayasinghe, SN and 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 and Zhang, F and Linden, RM and Thrasher, AJ and 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 and McIntosh, J and Della Peruta, M and Kymalainen, H and Tuddenham, EGD and Buckley, SMK and Waddington, SN and McVey, JH and Spence, Y and Morton, CL and Thrasher, AJ and Gray, JT and Castellino, FJ and Tarantal, AF and Davidoff, AM and Nathwani, AC (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 and Algar, VE and Xu-Bayford, J and Fairbanks, L and Owens, C and 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 and Rivat, C and Blundell, M and Alonso-Ferrera, M and Thrasher, A and 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 and Carter, NA and Recher, M and Notarangelo, LD and Burns, SO and Mauri, C and 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 and Lang, PA and Shaabani, N and Borkens, S and Scheu, S and Booth, S and Honke, N and Brenner, D and Meryk, A and Recher, M and Mak, TW and Ohashi, PS and Hausinger, D and Griffiths, GM and Thrasher, AJ and Lang, KS (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 and Zenner, H and Plagnol, V and Curtis, J and Mok, K and Eisenhut, M and Kumararatne, D and Doffinger, R and Thrasher, A and Nejentsev, S (2012) EXOME SEQUENCINGIDENTIFIES LRBA GENE DELETION IN A PATIENT PRESENTING WITH AUTOIMMUNITY WITHOUT HYPOGAMMAGLOBULINEMIA. In: JOURNAL OF CLINICAL IMMUNOLOGY. (pp. 398 - 398).

Burns, SO and Zenner, HL and Plagnol, V and Curtis, J and Mok, K and Eisenhut, M and Kumararatne, D and Doffinger, R and Thrasher, AJ and Nejentsev, S (2012) LRBA gene deletion in a patient presenting with autoimmunity without hypogammaglobulinemia. Journal of Allergy and Clinical Immunology , 130 (6) 1428 - 1432.

Burns, SO and Zenner, HL and Plagnol, V and Curtis, J and Mok, K and Eisenhut, M and Kumararatne, D and Doffinger, R and Thrasher, AJ and Nejentsev, S (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 and Jin, X and Geiger, S and Senadheera, S and Montiel-Equihua, C and Gaspar, B and Thrasher, A and Hollis, RP and Kohn, DB (2012) Development of a Lentiviral Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immunodeficiency (ADA-SCID). In: MOLECULAR THERAPY. (pp. S162 - S162).

Carmo, M and Arumugam, P and Montiel-Equiha, C and Alonso-Ferrero, M and Schambach, A and Baum, C and Risma, K and Malik, P and Thrasher, A and Jordan, M and Gaspar, B (2012) DEVELOPMENT OF GENE THERAPY FOR HAEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS (HLH) DUE TO PERFORIN DEFICIENCY. In: JOURNAL OF CLINICAL IMMUNOLOGY. (pp. 131 - 131).

Carmo, M and Arumugam, P and Montiel-Equihua, CA and Alonso-Ferrero, ME and Schambach, A and Baum, C and Risma, K and Malik, P and Thrasher, AJ and Jordan, MB and Gaspar, B (2012) Development of gene therapy for hemophagocytic lymphohistiocytosis (HLH) due to perforin deficiency. In: HUMAN GENE THERAPY. (pp. A3 - A3).

Carmo, M and Arumugam, P and Montiel-Equihua, CA and Alonso-Ferrero, ME and Tiwari, S and Schambach, A and Baum, C and Risma, K and Malik, P and Thrasher, AJ and Jordan, MB and Gaspar, B (2012) Development of Gene Therapy for Hemophagocytic Lymphohistiocytosis (HLH) Due to Perforin Deficiency. In: MOLECULAR THERAPY. (pp. S125 - S126).

Carmo, M and Arumugam, P and Tiwari, S and Thrasher, A and Risma, K and Malik, P and Gaspar, B and Jordan, M (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).

Chan, E (2012) Lentiviral gene therapy for HIV using TRIM-cyclophilin restriction factors. Doctoral thesis, UCL (University College London).
An open access version is available from UCL Discovery
 file

Chan, E and Schaller, T and Eddaoudi, A and Zhan, H and Tan, CP and Jacobsen, M and Thrasher, AJ and Towers, GJ and Qasim, W (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).
An open access version is available from UCL Discovery
 file

Charrier, S and Blundell, M and Cedrone, G and Louache, F and Vainchenker, W and Thrasher, A and 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 and Nowrouzi, A and Fronza, R and McDonald, RA and Kane, NM and Alba, R and Delles, C and Sessa, WC and Schmidt, M and Thrasher, AJ and Baker, AH (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 and Gilmour, K and Qasim, W and Adams, S and Worth, AJ and Zhan, H and Montiel-Equihua, CA and Derniame, S and Cale, C and Rao, K and Hiwarkar, P and Hough, R and Saudemont, A and Fahrenkrog, CS and Goulden, N and Amrolia, PJ and Veys, P (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 and Gilmour, K and Qasim, W and Adams, S and Worth, AJJ and Zhan, H and Montiel-Equihua, CA and Derniame, S and Cale, C and Rao, K and Hiwarkar, P and Hough, R and Saudemont, A and Fahrenkrog, CS and Goulden, N and Amrolia, PJ and Veys, P (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 and Ballard, S and Stauss, H and Thrasher, A and Longhurst, H and Workman, S (2012) WORLD IMMUNOLOGY DAY 2012 EDUCATIONAL SYMPOSIUM FOR CLINICIANS AND PATIENTS. In: JOURNAL OF CLINICAL IMMUNOLOGY. (pp. 26 - 26).

Cole, TS and Jones, LKR and McGrogan, P and Pearce, MS and Flood, TJ and Cant, AJ and Goldblatt, D and Thrasher, AJ and Gennery, AR and McKendrick, F and Titman, P (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 and Rao, K and Goulden, N and Amrolia, P and 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 and Howe, SJ and 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 and Gilmour, KC and Parsley, K and Curry, J and Sebire, N and Poliani, L and McCarthy, EA and Devlin, B and Markert, ML and Thrasher, AJ (2012) THYMUS TRANSPLANTATION FOR COMPLETE DIGEORGE SYNDROME: THE LONDON EXPERIENCE. In: JOURNAL OF CLINICAL IMMUNOLOGY. (pp. 12 - 13).

Di, WL and Semenova, E and Larcher, F and Del Rio, M and Harper, JI and Thrasher, AJ and 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 and Amrolia, P and Clark, A and Cornish, J and Jackson, G and Mahendra, P and Scarisbrick, JJ and Taylor, PC and Shaw, BE and Potter, MN (2012) Diagnosis and management of chronic graft-versus-host disease. British Journal of Haematology , 158 (1) 46 - 61.

Dignan, FL and Clark, A and Amrolia, P and Cornish, J and Jackson, G and Mahendra, P and Scarisbrick, JJ and Taylor, PC and Hadzic, N and Shaw, BE and Potter, MN and Haemato-oncology Task Force of British Committee for Standards in Haematology, and British Society for Blood and Marrow Transplantation, (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 and Greenblatt, D and Cox, M and Cavenagh, J and Oakervee, H and Apperley, JF and Fielding, AK and Pagliuca, A and Mufti, G and Raj, K and Marks, DI and Amrolia, P and Peniket, A and Medd, P and Potter, MN and Shaw, BE and Scarisbrick, JJ (2012) Efficacy of bimonthly extracorporeal photopheresis in refractory chronic mucocutaneous GVHD. Bone Marrow Transplantation , 47 (6) 824 - 830.

Dignan, FL and Scarisbrick, JJ and Cornish, J and Clark, A and Amrolia, P and Jackson, G and Mahendra, P and Taylor, PC and Shah, P and Lightman, S and Fortune, F and Kibbler, C and Andreyev, J and Albanese, A and Hadzic, N and Potter, MN and Shaw, BE (2012) Organ-specific management and supportive care in chronic graft-versus-host disease. British Journal of Haematology , 158 (1) 62 - 78.

E

Engelhardt, KR and Gertz, EM and Keles, S and Schaeffer, AA and Ceja, R and Sassi, A and Massaad, MJ and Mellouli, F and Benmustapha, I and Khemiri, M and Kilic, SS and Etzioni, A and Freeman, AF and Thiel, J and Schulze, I and Al-Herz, W and Metin, A and Sanal, O and Yeganeh, M and Niehues, T and Siepermann, K and Unal, E and Patiroglu, T and Dasouki, M and Yilmaz, M and Genel, F and Aytekin, C and Kutukculer, N and Somer, A and Kilic, M and Reisli, I and Camcioglu, Y and Gennery, AR and Cant, AJ and Jones, A and Gaspar, HB and Arkwright, PD and Pietrogrande, MC and Baz, Z and Al-Tamemi, S and Lougaris, V and Lefranc, G and Megarbane, A and Boutros, J and Galal, N and Bejaoui, M and Barbouche, R and Geha, RS and Chatila, TA and Grimbacher, B (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 and Stauss, HJ and Morris, EC and 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 and Carmo, M and Arumugam, P and Alonso-Ferrero, M and Schambach, A and Baum, C and Risma, K and Malik, P and Jordan, M and Rivat, C and Booth, C and Thrasher, A (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 and Semenova, K and McGrath, J and Di, W and Qasim, W (2012) Development of lentiviral gene therapy for type VII collagen deficient epidermolysis bullosa. In: HUMAN GENE THERAPY. (pp. A9 - A9).

Georgiadis, C and Thrasher, A and South, AP and McGrath, J and Qasim, W and 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 and Di, W-L and Semenova, E and Chan, L and Farzaneh, F and Qasim, W (2012) Clinical Grade Lentiviral Vector Production for the Inherited Skin Disease, Netherton Syndrome. In: MOLECULAR THERAPY. (pp. S164 - S164).

Guasti, L and Prasongchean, W and Kleftouris, G and Mukherjee, S and Thrasher, AJ and Bulstrode, NW and 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 and Booth, C and Brightwell, A and Allwood, Z and Veys, P and Rao, K and Hönig, M and Friedrich, W and Gennery, A and Slatter, M and Bredius, R and Finocchi, A and Cancrini, C and Aiuti, A and Porta, F and Lanfranchi, A and Ridella, M and Steward, C and Filipovich, A and Marsh, R and Bordon, V and Al-Muhsen, S and Al-Mousa, H and Alsum, Z and Al-Dhekri, H and Al Ghonaium, A and Speckmann, C and Fischer, A and Mahlaoui, N and Nichols, KE and Grunebaum, E and Al Zahrani, D and Roifman, CM and Boelens, J and Davies, EG and Cavazzana-Calvo, M and Notarangelo, L and Gaspar, HB and Inborn Errors Working Party of the European Group for Blood and Marrow Transplantation and European Society for Immunodeficiency, (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 and Morgenstern, DA and Yan, M and Vernay, B and Saraiva, L and Wu, Y and Cohen, CJ and Gustafsson, K and Anderson, J (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 and Wallace, R and Parsley, KL and Gilmour, K and Barrie, AU and Howe, K and Dong, R and Sebire, NJ and Michalski, A and Thrasher, AJ and Anderson, J (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.

Hiwarkar, P and Gaspar, HB and Gilmour, K and Jagani, M and Chiesa, R and Bennett-Rees, N and Breuer, J and Rao, K and Cale, C and Goulden, N and Davies, G and Amrolia, P and Veys, P and Qasim, W (2012) Impact of viral reactivations in the era of pre-emptive antiviral drug therapy following allogeneic haematopoietic SCT in paediatric recipients. Bone Marrow Transplant 10.1038/bmt.2012.221.

Howe, SJ and 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 and Gaspar, H and Davies, EG and Veys, P and 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 and Boldrin, L and Thrasher, A and Muntoni, F and Morgan, J (2012) Assessment of potential promoters for lentiviral gene therapy in DMD. In: NEUROMUSCULAR DISORDERS. (pp. S8 - S8).

K

Kallinikou, K and Anjos-Afonso, F and Blundell, MP and Ings, SJ and Watts, MJ and Thrasher, AJ and Linch, DC and Bonnet, D and Yong, KL (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 and Villegas-Llerena, C and Tagalakis, AD and Campbell, F and Welser, K and Botta, M and Tabor, AB and Hailes, HC and Lythgoe, MF and Hart, SL (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.

Knight, S and Zhang, F and Mueller-Kuller, U and Bokhoven, M and Gupta, A and Broughton, T and Sha, S and Antoniou, MN and Brendel, C and Grez, M and Thrasher, AJ and Collins, M and Takeuchi, Y (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.
An open access publication

L

Langford-Smith, A and Wilkinson, FL and Langford-Smith, KJ and Holley, RJ and Sergijenko, A and Howe, SJ and Bennett, WR and Jones, SA and Wraith, JE and Merry, CLR and Wynn, RF and Bigger, BW (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 and Aldea, M and Sanchez, R and Segovia, JC and Santilli, G and Thrasher, AJ and Bueren, JA and Almarza, E (2012) Itgb2 hypomorphic mice: a novel approach for ex vivo LAD gene therapy. In: HUMAN GENE THERAPY. (pp. A112 - A112).

Levin, MC and Lidberg, U and Jirholt, P and Adiels, M and Wramstedt, A and Gustafsson, K and Greaves, DR and Li, S and Fazio, S and Linton, MF and Olofsson, SO and Borén, J and Gjertsson, I (2012) Evaluation of macrophage-specific promoters using lentiviral delivery in mice. Gene Ther , 19 (11) 1041 - 1047. 10.1038/gt.2011.195.

Lowdell, MW and Birchall, M and 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 and Albon, SJ and Ricciardelli, I and Kester, MG and Meij, P and Lankester, AC and 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 and Monypenny, J and Blundell, MP and Cory, GO and Tomé-García, J and Thrasher, AJ and Jones, GE and Calle, Y (2012) Tyrosine phosphorylation of WASP promotes calpain-mediated podosome disassembly. Haematologica , 97 (5) 687 - 691. 10.3324/haematol.2011.048868.

Mohanty, ST and Cairney, CJ and Chantry, AD and Madan, S and Fernandes, JA and Howe, SJ and Moore, HD and Thompson, MJ and Chen, B and Thrasher, A and Keith, WN and Bellantuono, I (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 and Thrasher, AJ and Gaspar, HB (2012) Gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency. Curr Gene Ther , 12 (1) 57 - 65.

Montiel-Equihua, CA and Zhang, L and Knight, S and Saadeh, H and Scholz, S and Carmo, M and Alonso-Ferrero, ME and Blundell, MP and Monkeviciute, A and Schulz, R and Collins, M and Takeuchi, Y and Schmidt, M and Fairbanks, L and Antoniou, M and Thrasher, AJ and Bobby Gaspar, H (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.

Montiel-Equihua, CA and Zhang, L and Knight, S and Saadeh, H and Scholz, S and Carmo, M and Alonso-Ferrero, ME and Blundell, MP and Monkeviciute, A and Schulz, R and Collins, M and Takeuchi, Y and Schmidt, M and Fairbanks, L and Antoniou, M and Thrasher, AJ and Gaspar, HB (2012) Development of a gene expression system for enhanced erythroid expression. In: HUMAN GENE THERAPY. (pp. A11 - A11).

Montiel-Equihua, CA and Zhang, L and Knight, S and Saadeh, H and Scholz, S and Carmo, M and Alonso-Ferrero, ME and Blundell, MP and Monkeviciute, A and Schulz, R and Collins, M and Takeuchi, Y and Schmidt, M and Fairbanks, L and Antoniou, M and Thrasher, AJ and Gaspar, HB (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 and Mukherjee, S and Blundell, MP and Drews, K and Jones, GN and Abdulrazzak, H and Nowakowska, B and Phoolchund, A and Lay, K and Ramasamy, TS and Cananzi, M and Nettersheim, D and Sullivan, M and Frost, J and Moore, G and Vermeesch, JR and Fisk, NM and Thrasher, AJ and Atala, A and Adjaye, J and Schorle, H and De Coppi, P and Guillot, PV (2012) Valproic acid confers functional pluripotency to human amniotic fluid stem cells in a transgene-free approach. Molecular Therapy , 20 (10) 1953 - 1967.

Moschidou, D and Mukherjee, S and Blundell, MP and Drews, K and Jones, GN and Abdulrazzak, H and Nowakowska, B and Phoolchund, A and Lay, K and Ramasamy, TS and Cananzi, M and Nettersheim, D and Sullivan, M and Frost, J and Moore, G and Vermeesch, JR and Fisk, NM and Thrasher, AJ and Atala, A and Adjaye, J and Schorle, H and De Coppi, P and Guillot, PV (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 and Moeendarbary, E and Valon, L and Record, J and Charras, GT and 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 and Pipino, C and Sung, P and DeCoppi, P and 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 and Howe, SJ and Hart, SL (2012) Towards gene therapy for primary ciliary dyskinesia. In: HUMAN GENE THERAPY. (pp. A11 - A11).

P

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

Pantoglou, J and Abdul-Razak, HH and Rocca, C and Howe, SJ and Gregory, PD and Holmes, MC and Kinnon, C and Thrasher, AJ and Yanez-Munoz, RJ (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 and Taylor, C and Basu, P and Mussai, F and Santilli, G and Sala, A and 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 and Rodijk, M and Ng, YY and Baert, M and Lagresle-Peyrou, C and Schambach, A and Zhang, F and Hoeben, R and Hacein-Bey-Abina, S and Lankester, A and Bredius, R and Driessen, G and Thrasher, A and Baum, C and Cavazzana-Calvo, M and van Dongen, J and Staal, F (2012) SELF-INACTIVATING LENTIVIRAL VECTORS FOR CORRECTION OF RAG1 SEVERE COMBINED IMMUNODEFICIENCY. In: EXPERIMENTAL HEMATOLOGY. (pp. S46 - S47).

Pike-Overzet, K and Rodijk, M and Ng, YY and Baert, MRM and Lagresle-Peyrou, C and Schambach, A and Zhang, F and Hoeben, RC and Hacein-Bey-Abina, S and Lankester, AC and Bredius, RGM and Driessen, GJA and Thrasher, AJ and Baum, C and Cavazzana-Calvo, M and van Dongen, JJM and Staal, FJT (2012) Self-Inactivating Lentiviral Vectors for Correction of Rag1 Severe Combined Immunodeficiency. In: HUMAN GENE THERAPY. (pp. A42 - A42).

Plagnol, V and Curtis, J and Epstein, M and Mok, K and Stebbings, E and Grigoriadou, S and Wood, N and Hambleton, S and Burns, S and Thrasher, A and Kumararatne, D and Doffinger, R and Nejentsev, S (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 and Curtis, J and Epstein, M and Mok, KY and Stebbings, E and Grigoriadou, S and Wood, NW and Hambleton, S and Burns, SO and Thrasher, AJ and Kumararatne, D and Doffinger, R and Nejentsev, S (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.
An open access publication

Pugh, JAT and Cox, AG and McLeod, CW and Bunch, J and Writer, MJ and Hart, SL and Bienemann, A and White, E and Bell, J (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 and Burns, SO and de la Fuente, MA and Volpi, S and Dahlberg, C and Walter, JE and Moffitt, K and Mathew, D and Honke, N and Lang, PA and Patrizi, L and Falet, H and Keszei, M and Mizui, M and Csizmadia, E and Candotti, F and Nadeau, K and Bouma, G and Delmonte, OM and Frugoni, F and Fomin, AB and Buchbinder, D and Lundequist, EM and Massaad, MJ and Tsokos, GC and Hartwig, J and Manis, J and Terhorst, C and Geha, RS and Snapper, S and Lang, KS and Malley, R and Westerberg, L and Thrasher, AJ and Notarangelo, LD (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 and Booth, C and Blundell, M and Thrasher, AJ and 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 and Booth, C and Blundell, M and Thrasher, AJ and 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 and Santilli, G and Gaspar, HB and Thrasher, AJ (2012) Gene therapy for primary immunodeficiencies. Hum Gene Ther , 23 (7) 668 - 675. 10.1089/hum.2012.116.
An open access publication

S

Samarasinghe, S and Steward, C and Hiwarkar, P and Saif, MA and Hough, R and Webb, D and Norton, A and Lawson, S and Qureshi, A and Connor, P and Carey, P and Skinner, R and Vora, A and Pelidis, M and Gibson, B and Stewart, G and Keogh, S and Goulden, N and Bonney, D and Stubbs, M and Amrolia, P and Rao, K and Meyer, S and Wynn, R and Veys, P (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 and Ghani, S and Chan, L and Farzaneh, F and Larcher, F and Harper, J and Thrasher, A and Qasim, W and Di, WL (2012) Clinical grade lentiviral vector production for inherited skin disease. In: BRITISH JOURNAL OF DERMATOLOGY. (pp. e26 - e26).

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

Shaw, SWS and David, AL and Blundell, M and Howe, S and Pipino, C and Maghsoudlou, P and Lin, J and Porada, CD and Thrasher, A and De Coppi, P (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 and Rao, K and Chiesa, R and Amrolia, P and Nademi, Z and Flood, T and Abinun, M and Cant, A and Hambleton, J and Goulden, N and Davies, G and Qasim, W and Gaspar, HB and Gennay, A and Veys, P (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 and Mukherjee, S and Thrasher, AJ (2012) Lineage-specific haematopoietic differentiation of human induced pluripotent stem cells. In: HUMAN GENE THERAPY. (pp. A5 - A5).

T

Tagalakis, AD and Attwood, M and Manunta, MDI and Munye, M and McAnulty, RJ and Hart, SL (2012) Targeted Nanocomplex-Delivered ENaC siRNA as a Therapeutic Strategy for Cystic Fibrosis. In: MOLECULAR THERAPY. (pp. S243 - S244).

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

Tolar, J and Becker, PS and Clapp, DW and Hanenberg, H and Diaz de Heredia, C and Kiem, H-P and Navarro, S and Qasba, P and Rio, P and Schmidt, M and Sevilla, J and Verhoeyen, E and Thrasher, AJ and Bueren, J (2012) Gene Therapy for Fanconi Anemia: One Step Closer to the Clinic. HUMAN GENE THERAPY , 23 (2) 141 - 144. 10.1089/hum.2011.237.

V

van der Loo, JCM and Swaney, WP and Grassman, E and Terwilliger, A and Higashimoto, T and Schambach, A and Baum, C and Thrasher, AJ and Williams, DA and Nordling, DL and Reeves, L and Malik, P (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 and Swaney, WP and Grassman, E and Terwilliger, A and Higashimoto, T and Schambach, A and Baum, C and Thrasher, AJ and Williams, DA and Nordling, DL and Reeves, L and Malik, P (2012) Scale-up and manufacturing of clinical-grade self-inactivating γ-retroviral vectors by transient transfection. Gene Therapy , 19 (3) 246 - 254.

Van Der Loo, JCM and Swaney, WP and Grassman, E and Terwilliger, A and Higashimoto, T and Schambach, A and Hacein-Bey-Abina, S and Nordling, DL and Cavazzana-Calvo, M and Thrasher, AJ and Williams, DA and Reeves, L and Malik, P (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.

van der Loo, JCM and Swaney, WP and Grassman, E and Terwilliger, A and Higashimoto, T and Schambach, A and Hacein-Bey-Abina, S and Nordling, DL and Cavazzana-Calvo, M and Thrasher, AJ and Williams, DA and Reeves, L and Malik, P (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 and de Boer, H and Huston, M and Fontana, E and Poliani, PL and Cassani, B and Antoniou, MN and Zhang, F and Thrasher, AJ and Villa, A and Wagemaker, G (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 and de Boer, H and Mashamba, N and Wabik, A and Huston, M and Visser, TP and Fontana, E and Poliani, PL and Cassani, B and Zhang, F and Thrasher, AJ and Villa, A and Wagemaker, G (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 and Vořechovský, I and Sideras, P and Holland, J and Davies, A and Flinter, F and Hammarström, L and Kinnon, C and Levinsky, R and Bobrow, M and Smith, CI and Bentley, DR (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 and Howe, SJ (2012) Epigenetics of integration-deficient lentiviral vectors. In: HUMAN GENE THERAPY. (pp. A13 - A13).

W

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

White, K and Kane, NM and Milligan, G and 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 and Goulden, N and Veys, P and 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 and Kyrtatos, PG and Bienemann, AS and Pugh, JA and Lowe, AS and Villegas-Llerena, C and Kenny, GD and White, EA and Gill, SS and McLeod, CW and Lythgoe, MF and Hart, SL (2012) Lipid peptide nanocomplexes for gene delivery and magnetic resonance imaging in the brain. J Control Release , 162 (2) 340 - 348. 10.1016/j.jconrel.2012.07.002.

Z

Zhang, F and Knight, S and Collins, M and Takeuchi, Y and Antoniou, M and 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 Wed May 22 07:45:23 2013 BST.