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Number of items: 100.

A

Abeyewickreme, A and Thrasher, AJ and Kinnon, C (2011) Bone morphogenetic protein-4 (BMP4) up regulates key haematopoietic genes in differentiating embryonic stem cells treated with BMP4 short hairpin RNA. Br J Haematol , 155 (5) 638 - 641. 10.1111/j.1365-2141.2011.08759.x.

Acosta-Saltos, C and Gonitel, R and Chavda, S and Acosta-Saltos, A and Rahim, A and Thrasher, A and Anderson, P and Raivich, G (2011) Viral expression of GM-CSF: neuroinflammatory response and effects on regeneration. HUM GENE THER , 22 (10) A95 - A95.

Aitchison, KL and Rahim, AA and Hughes, DA and Kinnon, C and Waddington, SN and Howe, SJ (2011) Lentiviral vectors for gene therapy of Type I Gaucher Disease. HUM GENE THER , 22 (10) A44 - A44.

Ali-Boucetta, H and Al-Jamal, KT and Müller, KH and Li, S and Porter, AE and Eddaoudi, A and Prato, M and Bianco, A and Kostarelos, K (2011) Cellular uptake and cytotoxic impact of chemically functionalized and polymer-coated carbon nanotubes. Small , 7 (22) 3230 - 3238. 10.1002/smll.201101004.

Almarza, E and Zhang, F and Santilli, G and Blundell, MP and Howe, SJ and Thornhill, SI and Bueren, JA and Thrasher, AJ (2011) Correction of SCID-X1 Using an Enhancerless Vav Promoter. HUM GENE THER , 22 (3) 263 - 270. 10.1089/hum.2010.119.

Alonso-Ferrero, ME and Burke, D and Heales, S and Thrasher, A and Gaspar, HB (2011) Gene therapy for lysosomal storage diseases using an enhanced lentiviral vector delivery system. HUM GENE THER , 22 (10) A44 - A45.

Alvares, CL and Schenk, T and Hulkki, S and Min, T and Vijayaraghavan, G and Yeung, J and Gonzalez, D and So, CWE and Greaves, M and Titley, I and Bartolovic, K and Morgan, G (2011) Tyrosine kinase inhibitor insensitivity of non-cycling CD34+ human acute myeloid leukaemia cells with FMS-like tyrosine kinase 3 mutations. BRIT J HAEMATOL , 154 (4) 457 - 465. 10.1111/j.1365-2141.2011.08748.x.

Argyros, O and Wong, SP and Fedonidis, C and Tolmachov, O and Waddington, SN and Howe, SJ and Niceta, M and Coutelle, C and Harbottle, RP (2011) Development of S/MAR minicircles for enhanced and persistent transgene expression in the mouse liver. J Mol Med (Berl) , 89 (5) 515 - 529. 10.1007/s00109-010-0713-3.

B

Bacchelli, C and Buckland, KF and Buckridge, S and Salzer, U and Schneider, P and Thrasher, AJ and Gaspar, HB (2011) The C76R transmembrane activator and calcium modulator cyclophilin ligand interactor mutation disrupts antibody production and B-cell homeostasis in heterozygous and homozygous mice. J ALLERGY CLIN IMMUN , 127 (5) 1253 - U259. 10.1016/j.jaci.2011.02.037.

Bainbridge, J and Sundaram, V and Robbie, S and Smith, A and Stockman, A and Rubin, G and Holder, G and Thrasher, A and Moore, A and Ali, R (2011) Gene therapy for eye disease. In: JOURNAL OF MEDICAL GENETICS. (pp. S45 - S45). B M J PUBLISHING GROUP

Bartholomae, CC and Arens, A and Balaggan, KS and Yanez-Munoz, RJ and Montini, E and Howe, SJ and Paruzynski, A and Korn, B and Appelt, JU and MacNeil, A and Cesana, D and Abel, U and Glimm, H and Naldini, L and Ali, RR and Thrasher, AJ and von Kalle, C and Schmidt, M (2011) Lentiviral Vector Integration Profiles Differ in Rodent Postmitotic Tissues. MOL THER , 19 (4) 703 - 710. 10.1038/mt.2011.19.

Beilin, C and Choudhuri, K and Bouma, G and Thrasher, AJ and Dustin, ML and Burns, SO (2011) Dendritic cell common G-chain cytokine receptor is required for optimal IL-15 transpresentation to CD4+T cells at the immunological synapse. In: IMMUNOLOGY. (pp. 110 - 110).

Beilin, C. (2011) Common cytokine receptor γ chain deficiency in dendritic cells: implications for immunity. Doctoral thesis, UCL (University College London).

Binny, C and McIntosh, J and Peruta, M and Kymalainen, H and Tuddenham, E and Waddington, S and Buckley, S and Spence, Y and Morton, C and Thrasher, A and Gray, J and Tarantal, A and Davidoff, A and Nathwani, A (2011) AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous haemorrhage. HUMAN GENE THERAPY , 22 (10) A45 - A45.

Booth, C and Gaspar, HB and Thrasher, AJ (2011) Gene therapy for primary immunodeficiency. Curr Opin Pediatr , 23 (6) 659 - 666. 10.1097/MOP.0b013e32834cd67a.

Booth, C and Gilmour, KC and Veys, P and Gennery, AR and Slatter, MA and Chapel, H and Heath, PT and Steward, CG and Smith, O and O'Meara, A and Kerrigan, H and Mahlaoui, N and Cavazzana-Calvo, M and Fischer, A and Moshous, D and Blanche, S and Pachlopnik Schmid, J and Latour, S and de Saint-Basile, G and Albert, M and Notheis, G and Rieber, N and Strahm, B and Ritterbusch, H and Lankester, A and Hartwig, NG and Meyts, I and Plebani, A and Soresina, A and Finocchi, A and Pignata, C and Cirillo, E and Bonanomi, S and Peters, C and Kalwak, K and Pasic, S and Sedlacek, P and Jazbec, J and Kanegane, H and Nichols, KE and Hanson, IC and Kapoor, N and Haddad, E and Cowan, M and Choo, S and Smart, J and Arkwright, PD and Gaspar, HB (2011) X-linked lymphoproliferative disease due to SAP/SH2D1A deficiency: a multicenter study on the manifestations, management and outcome of the disease. Blood , 117 (1) 53 - 62. 10.1182/blood-2010-06-284935.

Bouma, G and Mendoza-Naranjo, A and Blundell, MP and de Falco, E and Parsley, KL and Burns, SO and Thrasher, AJ (2011) Cytoskeletal remodeling mediated by WASp in dendritic cells is necessary for normal immune synapse formation and T-cell priming. BLOOD , 118 (9) 2492 - 2501. 10.1182/blood-2011-03-340265.

Brown, L and Xu-Bayford, J and Allwood, Z and Slatter, M and Cant, A and Davies, EG and Veys, P and Gennery, AR and Gaspar, HB (2011) Neonatal diagnosis of severe combined immunodeficiency leads to significantly improved survival outcome: the case for newborn screening. BLOOD , 117 (11) 3243 - 3246. 10.1182/blood-2010-08-300384.

Brown, L and Xu-Bayford, J and Allwood, Z and Slatter, M and Cant, A and Davies, EG and Veys, P and Gennery, AR and Gaspar, HB (2011) Neonatal Diagnosis of Severe Combined Immunodeficiency Leads to Significantly Improved Survival Outcome: The Case for Newborn Screening EDITORIAL COMMENT. OBSTET GYNECOL SURV , 66 (7) 398 - 399. 10.1097/OGX.0b013e3182338432.

Buckland, KF and Ramaprakash, H and Murray, LA and Carpenter, KJ and Choi, ES and Kunkel, SL and Lukacs, NW and Xing, Z and Aoki, N and Hartl, D and Hogaboam, CM (2011) Triggering Receptor Expressed on Myeloid cells-1 (TREM-1) Modulates Immune Responses to Aspergillus fumigatus During Fungal Asthma in Mice. IMMUNOL INVEST , 40 (7-8) 692 - 722. 10.3109/08820139.2011.578270.

C

Carmo, M and Montiel-Equihua, CA and Schambach, A and Baum, C and Thrasher, AJ and Gaspar, HB (2011) Development of gene therapy for haemophagocytic lymphohistiocytosis (HLH) due to perforin deficiency. HUM GENE THER , 22 (10) A46 - A47.

Chan, E and Schaller, T and Thrasher, A and Towers, G and Qasim, W (2011) Lentivirus mediated protection against HIV-1 using a human TRIM21-Cyclophilin A fusion protein. HUM GENE THER , 22 (10) A68 - A68.

Cooray, S and Gilmour, KC and Parsley, KL and Adams, S and Howe, SJ and Al Ghonaium, A and Bayford, J and Brown, L and Davies, EG and Kinnon, C and Gasper, HB and Thrasher, AJ (2011) Persistence of a polyclonal T-cell repertoire in patients with SCID-X1 following gene therapy. HUM GENE THER , 22 (10) A42 - A42.

Cooray, S and Gilmour, KC and Parsley, KL and Zhang, F and Bjorkegren, E and Bayford, J and Brown, L and Davies, EG and Veys, P and Fairbanks, L and Bordon, V and Petropolou, T and Kinnon, C and Thrasher, AJ and Gaspar, HB (2011) Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction. HUM GENE THER , 22 (10) A40 - A40.

D

de Boer, J and Yeung, J and Ellu, J and Ramanujachar, R and Bornhauser, B and Solarska, O and Hubank, M and Williams, O and Brady, HJM (2011) The E2A-HLF oncogenic fusion protein acts through Lmo2 and Bcl-2 to immortalize hematopoietic progenitors. LEUKEMIA , 25 (2) 321 - 330. 10.1038/leu.2010.253.

Deichmann, A and Brugman, MH and Bartholomae, CC and Schwarzwaelder, K and Verstegen, MMA and Howe, SJ and Arens, A and Ott, MG and Hoelzer, D and Seger, R and Grez, M and Hacein-Bey-Abina, S and Cavazzana-Calvo, M and Fischer, A and Paruzynski, A and Gabriel, R and Glimm, H and Abel, U and Cattoglio, C and Mavilio, F and Cassani, B and Aiuti, A and Dunbar, CE and Baum, C and Gaspar, HB and Thrasher, AJ and von Kalle, C and Schmidt, M and Wagemaker, G (2011) Insertion Sites in Engrafted Cells Cluster Within a Limited Repertoire of Genomic Areas After Gammaretroviral Vector Gene Therapy. MOL THER , 19 (11) 2031 - 2039. 10.1038/mt.2011.178.

Di, WL and Larcher, F and Semenova, E and Talbot, GE and Harper, JI and Del Rio, M and Thrasher, AJ and Qasim, W (2011) Ex-vivo Gene Therapy Restores LEKTI Activity and Corrects the Architecture of Netherton Syndrome-derived Skin Grafts. MOL THER , 19 (2) 408 - 416. 10.1038/mt.2010.201.

Dong, R and Moulding, D and Himoudi, N and Adams, S and Bouma, G and Eddaoudi, A and Basu, BP and Derniame, S and Chana, P and Duncan, A and Anderson, J (2011) Cells with dendritic cell morphology and immunophenotype, binuclear morphology, and immunosuppressive function in dendritic cell cultures. Cellular Immunology , 272 (1) 1 - 10.

F

Farmer, S and Lorain, S and Thrasher, A and Garcia, L and Muntoni, F and Conti, F (2011) Correction of FKRP function via RNA trans-splicing. In: NEUROMUSCULAR DISORDERS. (pp. S8 - S8). PERGAMON-ELSEVIER SCIENCE LTD

Fassone, E and Duncan, AJ and Taanman, JW and Pagnamenta, AT and Sadowski, M and Holand, T and Qasim, W and Rutland, P and Calvo, SE and Mootha, VK and Bitner-Glindzicz, M and Rahman, S (2011) Mutations in the novel chaperone FOXRED1 cause mitochondrial complex I deficiency. In: NEUROMUSCULAR DISORDERS. (pp. S19 - S19). PERGAMON-ELSEVIER SCIENCE LTD

Fassone, E and Duncan, AJ and Taanman, JW and Pagnamenta, AT and Sadowski, MI and Holand, T and Qasim, W and Rutland, P and Calvo, SE and Mootha, VK and Bitner-Glindzicz, M and Rahman, S (2011) BIOCHEMICAL AND MOLECULAR ANALYSIS IN MITOCHONDRIAL COMPLEX I DEFICIENT CHILDREN. In: JOURNAL OF INHERITED METABOLIC DISEASE. (pp. S162 - S162).

Fassone, E and Duncan, AJ and Taanman, JW and Pagnamenta, AT and Sadowski, MI and Holand, T and Qasim, W and Rutland, P and Calvo, SE and Mootha, VK and Bitner-Glindzicz, M and Rahman, S (2011) FOXRED1 mutations are a novel cause of mitochondrial complex I deficiency. In: NEUROPATHOLOGY AND APPLIED NEUROBIOLOGY. (pp. 37 - 37). WILEY-BLACKWELL PUBLISHING, INC

Felgentreff, K and Perez-Becker, R and Speckmann, C and Schwarz, K and Kalwak, K and Markelj, G and Avcin, T and Qasim, W and Davies, EG and Niehues, T and Ehl, S (2011) Clinical and immunological manifestations of patients with atypical severe combined immunodeficiency. CLIN IMMUNOL , 141 (1) 73 - 82. 10.1016/j.clim.2011.05.007.

G

Galy, A and Thrasher, AJ (2011) Gene therapy for the Wiskott-Aldrich syndrome. CURR OPIN ALLERGY CL , 11 (6) 545 - 550. 10.1097/ACI.0b013e32834c230c.

Gaspar, HB (2011) Induced pluripotent stem cells and primary immunodeficiencies: A new frontier reached, a new world beyond? J ALLERGY CLIN IMMUN , 127 (6) 1408 - 1409. 10.1016/j.jaci.2011.02.038.

Gaspar, HB and Cooray, S and Gilmour, KC and Parsley, KL and Adams, S and Howe, SJ and Al Ghonaium, A and Bayford, J and Brown, L and Davies, EG and Kinnon, C and Thrasher, AJ (2011) Immunodeficiency: Long-term persistence of a polyclonal t cell repertoire after gene therapy for X-linked severe combined immunodeficiency. Science Translational Medicine , 3 (97)

Gaspar, HB and Cooray, S and Gilmour, KC and Parsley, KL and Adams, S and Howe, SJ and Al Ghonaium, A and Bayford, J and Brown, L and Davies, EG and Kinnon, C and Thrasher, AJ (2011) Long-Term Persistence of a Polyclonal T Cell Repertoire After Gene Therapy for X-Linked Severe Combined Immunodeficiency. SCI TRANSL MED , 3 (97) , Article 97ra79. 10.1126/scitranslmed.3002715.

Gaspar, HB and Cooray, S and Gilmour, KC and Parsley, KL and Zhang, F and Adams, S and Bjorkegren, E and Bayford, J and Brown, L and Davies, EG and Veys, P and Fairbanks, L and Bordon, V and Petropolou, T and Kinnon, C and Thrasher, AJ (2011) Hematopoietic Stem Cell Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Leads to Long-Term Immunological Recovery and Metabolic Correction. SCI TRANSL MED , 3 (97) , Article 97ra80. 10.1126/scitranslmed.3002716.

Gaspar, HB and Cooray, S and Gilmour, KC and Parsley, KL and Zhang, F and Adams, S and Bjorkegren, E and Bayford, J and Brown, L and Davies, EG and Veys, P and Fairbanks, L and Bordon, V and Petropolou, T and Kinnon, C and Thrasher, AJ (2011) Immunodeficiency: Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction. Science Translational Medicine , 3 (97)

Gholam, C and Grigoriadou, S and Gilmour, KC and Gaspar, HB (2011) Familial haemophagocytic lymphohistiocytosis: advances in the genetic basis, diagnosis and management. CLIN EXP IMMUNOL , 163 (3) 271 - 283. 10.1111/j.1365-2249.2010.04302.x.

Grez, M and Reichenbach, J and Schwable, J and Seger, R and Dinauer, MC and Thrasher, AJ (2011) Gene Therapy of Chronic Granulomatous Disease: The Engraftment Dilemma. MOL THER , 19 (1) 28 - 35. 10.1038/mt.2010.232.

H

Hacein-Bey-Abina, S and Caccaveli, L and Touzot, F and Dal-Cortivo, L and Heritier, S and Frange, P and Thrasher, A and Gaspar, B and Baum, C and Schambach, A and Malik, P and Van der Loo, H and Williams, D and Bushman, F and de Saint-Basile, G and Lim, A and Debre, M and Blanche, S and Fischer, A and Cavazzana-Calvo, M (2011) Efficacy of gene therapy for X-linked severe combined immunodeficiency: update on trial No.1 follow-up and preliminary results of multicentric collaborative trial No. 2 (Paris - Boston - London). HUMAN GENE THERAPY , 22 (10) A40 - A40.

Hiwarkar, P and Rao, A (2011) Revisiting Darwinism explains extinction of fetal erythroid progenitors in a leukaemogenic model of a paediatric myeloproliferative neoplasm. Br J Haematol , 155 (1) 2 - ?. 10.1111/j.1365-2141.2011.08731.x.

Hiwarkar, P and Shaw, BE and Tredger, JM and Brown, NW and Kulkarni, S and Saso, R and Evans, S and Treleaven, J and Davies, FE and Ethell, ME and Morgan, GJ and Potter, MN (2011) Mycophenolic acid trough level monitoring: relevance in acute and chronic graft versus host disease and its relation with albumin. CLIN TRANSPLANT , 25 (2) 222 - 227. 10.1111/j.1399-0012.2010.01226.x.

Huston, MW and van Til, NP and Visser, TP and Arshad, S and Brugman, MH and Cattoglio, C and Nowrouzi, A and Li, YD and Schambach, A and Schmidt, M and Baum, C and von Kalle, C and Mavilio, F and Zhang, F and Blundell, MP and Thrasher, AJ and Verstegen, MMA and Wagemaker, G (2011) Correction of Murine SCID-X1 by Lentiviral Gene Therapy Using a Codon-optimized IL2RG Gene and Minimal Pretransplant Conditioning. MOL THER , 19 (10) 1867 - 1877. 10.1038/mt.2011.127.

J

Jonuschies, J and Boldrin, L and Thrasher, A and Morgan, JE and Muntoni, F (2011) Assessment of potential promoters for lentiviral gene therapy in DMD. HUM GENE THER , 22 (10) A5 - A5.

Jonuschies, J and Boldrin, L and Thrasher, A and Morgan, JE and Muntoni, F (2011) Assessment of potential promoters for lentiviral gene therapy in DMD. HUM GENE THER , 22 (10) A62 - A63.

K

Kallinikou, K and Anjos-Afonso, F and Blundell, MP and Ings, SJ and Kassen, D and Thrasher, AJ and Linch, DC and Bonnet, D and Yong, K (2011) CD26 Inhibition Can Aid the Homing of Cytokine Activated Mobilized Peripheral Blood (MPB)CD34+Cells to the Bone Marrow (BM) but a Ligand Dependent Attachment Defect Prevents Their Long Term Retention and Subsequent Engraftment. In: BLOOD. (pp. 68 - 68).

Kao, VYC and Waddington, S and Equihua, CM and Thrasher, AJ and Antoniou, MN (2011) Curative F.IX production from low-dose UCOE-lentiviral vectors. HUM GENE THER , 22 (10) A110 - A110.

Kenny, GD and Kamaly, N and Kalber, TL and Brody, LP and Sahuri, M and Shamsaei, E and Miller, AD and Bell, JD (2011) Novel multifunctional nanoparticle mediates siRNA tumour delivery, visualisation and therapeutic tumour reduction in vivo. J CONTROL RELEASE , 149 (2) 111 - 116. 10.1016/j.jconrel.2010.09.020.

Kudsiova, L and Ho, J and Fridrich, B and Harvey, R and Keppler, M and Ng, T and Hart, SL and Tabor, AB and Hailes, HC and Lawrence, MJ (2011) Lipid chain geometry of C14 glycerol-based lipids: effect on lipoplex structure and transfection. MOL BIOSYST , 7 (2) 422 - 436. 10.1039/c0mb00149j.

Kwok, A and Hart, SL (2011) Comparative structural and functional studies of nanoparticle formulations for DNA and siRNA delivery. NANOMED-NANOTECHNOL , 7 (2) 210 - 219. 10.1016/j.nano.2010.07.005.

L

Langford-Smith, A and Wilkinson, F and Langford-Smith, KJ and Sergijenko, A and Bennett, W and Howe, S and Thrasher, A and Jones, S and Wraith, JE and Wynn, R and Bigger, B (2011) Lentiviral vector enhanced haematopoietic stem cell gene therapy for mucopolysaccharidosis type IIIA. In: BONE MARROW TRANSPLANTATION. (pp. S316 - S316).

M

Malik, SJ and Kenny, GD and Hajnal, JV (2011) Slice profile correction for transmit sensitivity mapping using actual flip angle imaging. Magn Reson Med , 65 (5) 1393 - 1399. 10.1002/mrm.22739.

Mandegar, MA and Moralli, D and Khoja, S and Cowley, S and Chan, DYL and Yusuf, M and Mukherjee, S and Blundell, MP and Volpi, EV and Thrasher, AJ and James, W and Monaco, ZL (2011) Functional human artificial chromosomes are generated and stably maintained in human embryonic stem cells. HUM MOL GENET , 20 (15) 2905 - 2913. 10.1093/hmg/ddr144.

Manunta, MDI and McAnulty, RJ and Tagalakis, AD and Bottoms, SE and Campbell, F and Hailes, HC and Laurent, GJ and O'Callaghan, C and Hart, SL (2011) Nebulisation of Receptor-Targeted Nanocomplexes for Gene Delivery to the Airway Epithelium. PLoS ONE , 6 (10) , Article e26768. 10.1371/journal.pone.0026768.
An open access publication. A version is also available from UCL Discovery.

Manunta, MDI and McAnulty, RJ and Tagalakis, AD and Bottoms, SE and Campbell, F and Hailes, HC and Tabor, AB and Laurent, GJ and O'Callaghan, C and Hart, SL (2011) Nebulisation of Receptor-Targeted Nanocomplexes for Gene Delivery to the Airway Epithelium. PLOS ONE , 6 (10) , Article e26768. 10.1371/journal.pone.0026768.
An open access publication. A version is also available from UCL Discovery.

McKay, TR and Rahim, AA and Buckley, SM and Ward, NJ and Chan, JK and Howe, SJ and Waddington, SN (2011) Perinatal gene transfer to the liver. Curr Pharm Des , 17 (24) 2528 - 2541.

Mendoza-Naranjo, A and Bouma, G and Pereda, C and Ramirez, M and Webb, KF and Tittarelli, A and Lopez, MN and Kalergis, AM and Thrasher, AJ and Becker, DL and Salazar-Onfray, F (2011) Functional Gap Junctions Accumulate at the Immunological Synapse and Contribute to T Cell Activation. J IMMUNOL , 187 (6) 3121 - 3132. 10.4049/jimmunol.1100378.

Metelo, J and Ward, N and Thrasher, AJ and Burns, SO (2011) Lentivectors Are Efficient Tools to Manipulate the Dendritic Cell Cytoskeleton. CYTOSKELETON , 68 (8) 434 - 445. 10.1002/cm.20521.

Montiel-Equihua, CA and Zhang, L and Knight, S and Collins, M and Saadeh, H and Schulz, R and Scholz, S and Schmidt, M and Carmo, M and Alonso-Ferrero, ME and Blundell, MP and Fairbanks, LD and Monkeviciute, A and Antoniou, M and Thrasher, AJ and Gaspar, HB (2011) Development of a gene expression system for enhanced erythroid expression. HUMAN GENE THERAPY , 22 (10) A105 - A105.

Monypenny, J and Chou, HC and Banon-Rodriguez, I and Thrasher, AJ and Anton, IM and Jones, GE and Calle, Y (2011) Role of WASP in cell polarity and podosome dynamics of myeloid cells. EUR J CELL BIOL , 90 (2-3) 198 - 204. 10.1016/j.ejcb.2010.05.009.

Moratto, D and Giliani, S and Bonfim, C and Mazzolari, E and Fischer, A and Ochs, HD and Cant, AJ and Thrasher, AJ and Cowan, MJ and Albert, MH and Small, T and Pai, SY and Haddad, E and Lisa, A and Hambleton, S and Slatter, M and Cavazzana-Calvo, M and Mahlaoui, N and Picard, C and Torgerson, TR and Burroughs, L and Koliski, A and Neto, JZ and Porta, F and Qasim, W and Veys, P and Kavanau, K and Honig, M and Schulz, A and Friedrich, W and Notarangelo, LD (2011) Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study. BLOOD , 118 (6) 1675 - 1684. 10.1182/blood-2010-11-319376.

Mukherjee, S and Santilli, G and Blundell, MP and Navarro, S and Bueren, JA and Thrasher, AJ (2011) Generation of Functional Neutrophils from a Mouse Model of X-Linked Chronic Granulomatous Disorder Using Induced Pluripotent Stem Cells. PLOS ONE , 6 (3) , Article e17565. 10.1371/journal.pone.0017565.
An open access publication. A version is also available from UCL Discovery.

Mukherjee, S and Thrasher, AJ (2011) iPSCs: Unstable Origins? MOL THER , 19 (7) 1188 - 1190. 10.1038/mt.2011.116.

MUKHERJEE, S and Thrasher, AJ (2011) iPSCs:Unstable Origins? Molecular Therapy , 19 (7) 1188 - 1190. 10.1038/mt.2011.116.

Mukherjee, S and Thrasher, AJ (2011) Progress and Prospects: Advancements in Retroviral Vector Design, Generation, and Application. HUM GENE THER , 22 (10) 1171 - 1174. 10.1089/hum.2011.2523.

P

Palendira, U and Low, C and Chan, A and Hislop, AD and Ho, E and Phan, TG and Deenick, E and Cook, MC and Riminton, DS and Choo, S and Loh, R and Alvaro, F and Booth, C and Gaspar, HB and Moretta, A and Khanna, R and Rickinson, AB and Tangye, SG (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 ARTN e1001187. 10.1371/journal.pbio.1001187.
An open access publication

Pallant, C and Cathomen, T and Dannemann, N and Quasim, W and Naldini, L and Kinnon, C and Howe, S and Thrasher, A (2011) Targeting models of severe combined immunodeficiency for correction by zinc finger nucleases. HUM GENE THER , 22 (10) A41 - A41.

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 (2011) Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer. LEUKEMIA , 25 (9) 1471 - 1483. 10.1038/leu.2011.106.

Pizzolla, A and Gelderman, KA and Hultqvist, M and Vestberg, M and Gustafsson, K and Mattsson, R and Holmdahl, R (2011) CD68-expressing cells can prime T cells and initiate autoimmune arthritis in the absence of reactive oxygen species. Eur J Immunol , 41 (2) 403 - 412. 10.1002/eji.201040598.

Q

Qasim, W and Derniame, S and Gilmour, K and Chiesa, R and Weber, M and Adams, S and Rao, K and Amrolia, P and Goulden, N and Veys, P and Gaspar, H (2011) Third-party virus-specific T cells eradicate adenoviraemia but trigger bystander graft-versus-host disease. BRIT J HAEMATOL , 154 (1) 150 - 153. 10.1111/j.1365-2141.2011.08579.x.

R

Rocca, C and Abdul-Razak, HH and Estevez, FJM and Guenechea, G and Howe, SJ and Holmes, MC and Gregory, PD and Thrasher, AJ and Bueren, JA and Yanez-Munoz, RJ (2011) ZFN-driven gene targeting to correct mouse Prkdc scid deficiency. HUM GENE THER , 22 (10) A44 - A44.

S

Samarasinghe, S and Steward, C and Hiwarkar, P and Bonney, D and Keogh, S and Rao, K and Amrolia, P and Goulden, N and Mathew, S and Hough, R and Wynn, R and Veys, P (2011) ALEMTUZUMAB-BASED CONDITIONING PROTOCOLS FOR PAEDIATRIC MATCHED UNRELATED DONOR TRANSPLANTS FOR SEVERE APLASTIC ANAEMIA: AN IMPORTANT ALTERNATIVE TO TBI CONTAINING REGIMENS. In: BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION. (pp. S186 - S187). ELSEVIER SCIENCE INC

Santilli, G and Almarza, E and Brendel, C and Choi, U and Beilin, C and Blundell, MP and Haria, S and Parsley, KL and Kinnon, C and Malech, HL and Bueren, JA and Grez, M and Thrasher, AJ (2011) Biochemical Correction of X-CGD by a Novel Chimeric Promoter Regulating High Levels of Transgene Expression in Myeloid Cells. MOL THER , 19 (1) 122 - 132. 10.1038/mt.2010.226.

Santilli, G and Kehl, E and Parsley, K and Cooray, S and Buckland, K and Blundell, M and Bouma, G and Galy, A and Charrier, S and Kinnon, C and Thrasher, AJ (2011) Development of a clinical trial of gene therapy for X-CGD using the pCCLCHIMGp91WPRE4 lentiviral vector. HUM GENE THER , 22 (10) A52 - A52.

Semenova, E and Di, WL and Larcher, F and Ghani, S and Harper, J and Thrasher, A and Qasim, W (2011) Human involucrin promoter resists spreading methylation and supports compartment specific gene expression in engineered skin grafts. HUM GENE THER , 22 (10) A52 - A52.

Shaw, SW 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 (2011) Sheep amniotic fluid derived CD34+stem cells engraft in NOD-SCID gamma mice and in lambs after prenatal autologous transplantation. Presented at: UNSPECIFIED.

Shaw, SWS and Bollini, S and Nader, KA and Gastadello, A and Mehta, V and Filppi, E and Cananzi, M and Gaspar, HB and Qasim, W and De Coppi, P and David, AL (2011) Autologous Transplantation of Amniotic Fluid-Derived Mesenchymal Stem Cells Into Sheep Fetuses. CELL TRANSPLANT , 20 (7) 1015 - 1031. 10.3727/096368910X543402.

Skucek, E and Butler, S and Gaspar, HB and Titman, P (2011) Social outcome in children treated by haematopoietic cell transplant for congenital immunodeficiency. BONE MARROW TRANSPL , 46 (10) 1314 - 1320. 10.1038/bmt.2010.318.

Slatter, MA and Rao, K and Amrolia, P and Flood, T and Abinun, M and Hambleton, S and Nademi, Z and Goulden, N and Davies, G and Qasim, W and Gaspar, HB and Cant, A and Gennery, AR and Veys, P (2011) Treosulfan-based conditioning regimens for hematopoietic stem cell transplantation in children with primary immunodeficiency: United Kingdom experience. BLOOD , 117 (16) 4367 - 4375. 10.1182/blood-2010-10-312082.

Surman, AJ and Bonnet, CS and Lowe, MP and Kenny, GD and Bell, JD and Tóth, E and Vilar, R (2011) A pyrophosphate-responsive gadolinium(III) MRI contrast agent. Chemistry , 17 (1) 223 - 230. 10.1002/chem.201001397.

Surman, AJ and Kenny, GD and Kumar, DK and Bell, JD and Casey, DR and Vilar, R (2011) Targeting of anionic membrane species by lanthanide(III) complexes: towards improved MRI contrast agents for apoptosis. Chem Commun (Camb) , 47 (37) 10245 - 10247. 10.1039/c1cc13284a.

T

Tagalakis, AD and Grosse, SM and Meng, QH and Mustapa, MFM and Kwok, A and Salehi, SE and Tabor, AB and Hailes, HC and Hart, SL (2011) Integrin-targeted nanocomplexes for tumour specific delivery and therapy by systemic administration. BIOMATERIALS , 32 (5) 1370 - 1376. 10.1016/j.biomaterials.2010.10.037.

Tagalakis, AD and He, L and Saraiva, L and Gustafsson, KT and Hart, SL (2011) Receptor-targeted liposome-peptide nanocomplexes for siRNA delivery. BIOMATERIALS , 32 (26) 6302 - 6315. 10.1016/j.biomaterials.2011.05.022.

Thrasher, A (2011) Gene therapy for immunodeficiency. HUMAN GENE THERAPY , 22 (10) A5 - A6.

Thrasher, AJ and Segal, AW (2011) A phagocyte dilemma... NAT IMMUNOL , 12 (3) 201 - 202. 10.1038/ni0311-201.

Tolar, J and Adair, JE and Antoniou, M and Bartholomae, CC and Becker, PS and Blazar, BR and Bueren, J and Carroll, T and Cavazzana-Calvo, M and Clapp, DW and Dalgleish, R and Galy, A and Gaspar, HB and Hanenberg, H and Von Kalle, C and Kiem, HP and Lindeman, D and Naldini, L and Navarro, S and Renella, R and Rio, P and Sevilla, J and Schmidt, M and Verhoeyen, E and Wagner, JE and Williams, DA and Thrasher, AJ (2011) Stem Cell Gene Therapy for Fanconi Anemia: Report from the 1st International Fanconi Anemia Gene Therapy Working Group Meeting. MOL THER , 19 (7) 1193 - 1198. 10.1038/mt.2011.78.

W

Ward, NJ and Buckley, SMK and Rahim, AA and McVey, JH and Thrasher, AJ and Waddington, SN (2011) Optimised vectors for the treatment of haemophilia A. HUM GENE THER , 22 (10) A109 - A109.

Ward, NJ and Buckley, SMK and Waddington, SN and VandenDriessche, T and Chuah, MKL and Nathwani, AC and McIntosh, J and Tuddenham, EGD and Kinnon, C and Thrasher, AJ and McVey, JH (2011) Codon optimization of human factor VIII cDNAs leads to high-level expression. BLOOD , 117 (3) 798 - 807. 10.1182/blood-2010-05-282707.

Williams, DA and Thrasher, AJ (2011) Out of harm's way. Nature Biotechnology , 29 (1) 41 - 42.

Worth, A and Conyers, R and Cohen, J and Jagani, M and Chiesa, R and Rao, K and Goulden, N and Veys, P and Amrolia, PJ (2011) Pre-emptive rituximab based on viraemia and T cell reconstitution: a highly effective strategy for the prevention of Epstein-Barr virus-associated lymphoproliferative disease following stem cell transplantation. BRIT J HAEMATOL , 155 (3) 377 - 385. 10.1111/j.1365-2141.2011.08855.x.

Y

Yan, MY and Himoudi, N and Basu, BP and Wallace, R and Poon, E and Adams, S and Hasan, F and Xue, SA and Wilson, N and Dalgleish, A and Williams, O and Anderson, J (2011) Increased PRAME antigen-specific killing of malignant cell lines by low avidity CTL clones, following treatment with 5-Aza-2'-Deoxycytidine. CANCER IMMUNOL IMMUN , 60 (9) 1243 - 1255. 10.1007/s00262-011-1024-4.

Z

Zhan, H and Farzaneh, F and Gilmour, K and Bhalla, J and Chan, L and Fehse, B and Parsley, K and Mojidra, C and Swift, S and Veys, P and Thrasher, A and Gaspar, B and Qasim, W (2011) Pathway to delivery of a gene modified T cell Investigational Medicinal Product (IMP). HUM GENE THER , 22 (10) A74 - A75.

Zhang, F and Thrasher, A and Antoniou, M (2011) The lentiviral vector containing UCOE element driving IL2 receptor gene confers a long term immuno-reconstitution in mouse model of SCID-X1. HUM GENE THER , 22 (10) A110 - A110.

Zhang, L and Montiel-Equihua, CA and Thrasher, AJ and Gaspar, HB (2011) Dr Lin Zhang. HUMAN GENE THERAPY , 22 (10) A53 - A54.

This list was generated on Fri May 24 10:57:57 2013 BST.

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