Adrian J Thrasher

Adrian J Thrasher

UNVERIFIED PROFILE

Are you Adrian J Thrasher?   Register this Author

Register author
Adrian J Thrasher

Adrian J Thrasher

Publications by authors named "Adrian J Thrasher"

Are you Adrian J Thrasher?   Register this Author

100Publications

3039Reads

32Profile Views

Gene therapy for primary immunodeficiency.

Hum Mol Genet 2019 Oct;28(R1):R15-R23

Molecular and Cellular Immunology Section, UCL Great Ormond Street Institute of Child Health, London, UK.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1093/hmg/ddz170DOI Listing
October 2019

Targeted Repair of p47-CGD in iPSCs by CRISPR/Cas9: Functional Correction without Cleavage in the Highly Homologous Pseudogenes.

Stem Cell Reports 2019 Oct 19;13(4):590-598. Epub 2019 Sep 19.

Institute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625 Hannover, Germany; REBIRTH Cluster of Excellence, Hannover Medical School, 30625 Hannover, Germany; Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA. Electronic address:

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.stemcr.2019.08.008DOI Listing
October 2019

Enhancing Lentiviral and Alpharetroviral Transduction of Human Hematopoietic Stem Cells for Clinical Application.

Mol Ther Methods Clin Dev 2019 Sep 7;14:134-147. Epub 2019 Jun 7.

Infection, Immunity and Inflammation Program, Molecular and Cellular Immunology Section, UCL Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.omtm.2019.05.015DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6629974PMC
September 2019

How I manage patients with Wiskott Aldrich syndrome.

Br J Haematol 2019 May 12;185(4):647-655. Epub 2019 Mar 12.

Department of Immunology, Royal Free London NHS Foundation Trust, London, UK.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1111/bjh.15831DOI Listing
May 2019

Bleeding and splenectomy in Wiskott-Aldrich syndrome: A single-centre experience.

J Allergy Clin Immunol Pract 2019 Mar 23;7(3):1042-1044.e1. Epub 2018 Jul 23.

Department of Immunology, Royal Free London NHS Foundation Trust, London, United Kingdom; University College London, Institute of Immunity and Transplantation, London, United Kingdom.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.jaip.2018.07.009DOI Listing
March 2019

Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort.

Hum Gene Ther 2019 Jan 19;30(1):79-87. Epub 2018 Oct 19.

1 Genetics and Genomic Medicine Programme, Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.

View Article

Download full-text PDF

Source
http://www.liebertpub.com/doi/10.1089/hum.2018.098
Publisher Site
http://dx.doi.org/10.1089/hum.2018.098DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6343184PMC
January 2019

One hundred percent survival after transplantation of 34 patients with Wiskott-Aldrich syndrome over 20 years.

J Allergy Clin Immunol 2018 11 25;142(5):1654-1656.e7. Epub 2018 Jul 25.

Blood and Marrow Transplant Unit, Great Ormond Street Hospital NHS Trust, University College London, London, United Kingdom.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.jaci.2018.06.042DOI Listing
November 2018

Autonomous role of Wiskott-Aldrich syndrome platelet deficiency in inducing autoimmunity and inflammation.

J Allergy Clin Immunol 2018 10 6;142(4):1272-1284. Epub 2018 Feb 6.

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Division of Regenerative Medicine, Stem Cells and Gene Therapy, San Raffaele Scientific Institute, Milan, Italy; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy. Electronic address:

View Article

Download full-text PDF

Source
https://linkinghub.elsevier.com/retrieve/pii/S00916749183020
Publisher Site
http://dx.doi.org/10.1016/j.jaci.2017.12.1000DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6078823PMC
October 2018

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

J Allergy Clin Immunol 2018 09 31;142(3):904-913.e3. Epub 2018 Jan 31.

Infection, Immunity, Inflammation, Molecular and Cellular Immunology Section, UCL Great Ormond Street Institute of Child Health, London, United Kingdom. Electronic address:

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.jaci.2017.11.050DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6127027PMC
September 2018

Transfer of gene-corrected T cells corrects humoral and cytotoxic defects in patients with X-linked lymphoproliferative disease.

J Allergy Clin Immunol 2018 07 27;142(1):235-245.e6. Epub 2018 Apr 27.

Molecular and Cellular Immunology Section, UCL GOS Institute of Child Health, London, United Kingdom; Department of Paediatric Immunology, Great Ormond Street Hospital NHS Trust, London, United Kingdom. Electronic address:

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.jaci.2018.02.053DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6034012PMC
July 2018

Leukocyte adhesion deficiency-I: A comprehensive review of all published cases.

J Allergy Clin Immunol Pract 2018 Jul - Aug;6(4):1418-1420.e10. Epub 2018 Jan 20.

Division of Hematopoietic Innovative Therapies, Centro de Investigaciones Energéticas Medioambientales y Tecnológicas (CIEMAT) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER-ISCIII), Madrid, Spain; Advanced Therapies Unit, Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD/UAM), Madrid, Spain.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.jaip.2017.12.008DOI Listing
January 2018

Wiskott-Aldrich syndrome protein: Emerging mechanisms in immunity.

Eur J Immunol 2017 11 12;47(11):1857-1866. Epub 2017 Sep 12.

UCL Great Ormond Street Institute of Child Health, London.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1002/eji.201646715DOI Listing
November 2017

A Personal Reflection from London.

Hum Gene Ther 2017 11;28(11):959

1 UCL Great Ormond Street Institute of Child Health, Molecular and Cellular Immunology, London, United Kingdom.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1089/hum.2017.29052.atrDOI Listing
November 2017

Absence of γ-Chain in Keratinocytes Alters Chemokine Secretion, Resulting in Reduced Immune Cell Recruitment.

J Invest Dermatol 2017 10 17;137(10):2120-2130. Epub 2017 Jun 17.

Institute of Immunity and Transplantation, University College London, London, UK; Department of Immunology, Royal Free Hospital Foundation Trust, London, UK. Electronic address:

View Article

Download full-text PDF

Source
https://linkinghub.elsevier.com/retrieve/pii/S0022202X173164
Publisher Site
http://dx.doi.org/10.1016/j.jid.2017.05.024DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5896775PMC
October 2017

Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.

N Engl J Med 2017 10 4;377(17):1630-1638. Epub 2017 Oct 4.

From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.), Dana-Farber and Boston Children's Cancer and Blood Disorders Center (C. Duncan, M.A., C. Dansereau, D.A.W.), and Boston Children's Hospital, Harvard Medical School, and Harvard Stem-Cell Institute (D.A.W.), Boston, and Bluebird Bio, Cambridge (A.M.P., E.S., T.O., D.D.) - all in Massachusetts; University of Minnesota Children's Hospital, Minneapolis (P.J.O., T.C.L., W.P.M., G.V.R.); University of California, Los Angeles, Los Angeles (S.D.O., R.S., A.J.S.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., H.B.G., P.G.); Pediatric Neurology Department, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Le Kremlin Bicêtre, France (C.S., P.A.); Fundacion Investigar, Buenos Aires (H.A.); and Women's and Children's Hospital, North Adelaide, SA, Australia (D.B., N.J.C.S.).

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1056/NEJMoa1700554DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5708849PMC
October 2017

Targeted genome editing restores T cell differentiation in a humanized X-SCID pluripotent stem cell disease model.

Sci Rep 2017 09 29;7(1):12475. Epub 2017 Sep 29.

Institute for Transfusion Medicine and Gene Therapy, Medical Center - University of Freiburg, Freiburg, Germany.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1038/s41598-017-12750-4DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5622068PMC
September 2017

Characterization of a core region in the A2UCOE that confers effective anti-silencing activity.

Sci Rep 2017 08 31;7(1):10213. Epub 2017 Aug 31.

Molecular and Cellular Immunology, UCL GOS Institute of Child Health, London, WC1N 1EH, UK.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1038/s41598-017-10222-3DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5578987PMC
August 2017

Evolving Gene Therapy in Primary Immunodeficiency.

Mol Ther 2017 05 31;25(5):1132-1141. Epub 2017 Mar 31.

Boston Children's Hospital and Dana-Farber Cancer Institute, Harvard Medical School and Harvard Stem Cell Institute, 300 Longwood Avenue, Boston, MA 02115, USA. Electronic address:

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.ymthe.2017.03.018DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5417846PMC
May 2017

Lentiviral vectors can be used for full-length dystrophin gene therapy.

Sci Rep 2017 03 6;7(1):79. Epub 2017 Mar 6.

UCL Cancer Institute, Paul O 'Gorman Building, University College London, 72 Huntley Street, London, WC1E 6BT, UK.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1038/s41598-017-00152-5DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5427806PMC
March 2017

Lentiviral vectors can be used for full-length dystrophin gene therapy.

Sci Rep 2017 03 17;7:44775. Epub 2017 Mar 17.

UCL Cancer Institute, Paul O 'Gorman Building, University College London, 72 Huntley Street, London, WC1E 6BT, UK.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1038/srep44775DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5356018PMC
March 2017

Limiting Thymic Precursor Supply Increases the Risk of Lymphoid Malignancy in Murine X-Linked Severe Combined Immunodeficiency.

Mol Ther Nucleic Acids 2017 Mar 10;6:1-14. Epub 2016 Dec 10.

Gene Therapy Research Unit, Children's Medical Research Institute, The University of Sydney and The Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia; Discipline of Child and Adolescent Health, The University of Sydney, Westmead, NSW 2145, Australia. Electronic address:

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.omtn.2016.11.011DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5363493PMC
March 2017

A New Chapter on Targeted Gene Insertion for X-CGD: Do Not Skip the Intro(n).

Mol Ther 2017 02 18;25(2):307-309. Epub 2017 Jan 18.

Molecular Immunology Unit, Centre for Immunodeficiency, Institute of Child Health, University College London, 30 Guilford Street, London, WC1N 1EH, UK. Electronic address:

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.ymthe.2017.01.002DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5368843PMC
February 2017

Primary immunodeficiencies due to abnormalities of the actin cytoskeleton.

Curr Opin Hematol 2017 01;24(1):16-22

aUniversity College London Institute of Immunity and Transplantation bDepartment of Immunology, Royal Free London NHS Foundation Trust cUniversity College London Institute of Child Health dGreat Ormond Street Hospital for Children NHS Foundation Trust, London, UK.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1097/MOH.0000000000000296DOI Listing
January 2017

Lentiviral Vector-Mediated Correction of a Mouse Model of Leukocyte Adhesion Deficiency Type I.

Hum Gene Ther 2016 09 5;27(9):668-78. Epub 2016 May 5.

1 Division of Hematopoietic Innovative Therapies, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) , and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid, Spain .

View Article

Download full-text PDF

Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5035374PMC
http://dx.doi.org/10.1089/hum.2016.016DOI Listing
September 2016

Impact of BREXIT on UK Gene and Cell Therapy: The Need for Continued Pan-European Collaboration.

Hum Gene Ther 2016 09;27(9):653-5

3 Institute of Child Health, University College London , London, United Kingdom .

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1089/hum.2016.29033.ahbDOI Listing
September 2016

Current and emerging treatment options for Wiskott-Aldrich syndrome.

Expert Rev Clin Immunol 2015 9;11(9):1015-32. Epub 2015 Jul 9.

Department of Clinical Immunology and Bone Marrow Transplantation, Great Ormond Street Hospital National Health Service Trust, London WC1N 1JH, UK.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1586/1744666X.2015.1062366DOI Listing
May 2016

WASp-dependent actin cytoskeleton stability at the dendritic cell immunological synapse is required for extensive, functional T cell contacts.

J Leukoc Biol 2016 05 20;99(5):699-710. Epub 2015 Nov 20.

Molecular Immunology Unit, University College London Institute of Child Health, London, United Kingdom; Great Ormond Street Hospital for Children, National Health Service Foundation Trust, London, United Kingdom

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1189/jlb.2A0215-050RRDOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5404712PMC
May 2016

Treating Immunodeficiency through HSC Gene Therapy.

Trends Mol Med 2016 Apr 15;22(4):317-327. Epub 2016 Mar 15.

Molecular and Cellular Immunology Section, UCL Institute of Child Health, London, UK; Department of Paediatric Immunology, Great Ormond Street Hospital, London, UK. Electronic address:

View Article

Download full-text PDF

Source
http://www.cell.com/trends/molecular-medicine/pdf/S1471-4914
Web Search
http://linkinghub.elsevier.com/retrieve/pii/S147149141600032
Publisher Site
http://dx.doi.org/10.1016/j.molmed.2016.02.002DOI Listing
April 2016

Common variable immunodeficiency and natural killer cell lymphopenia caused by Ets-binding site mutation in the IL-2 receptor γ (IL2RG) gene promoter.

J Allergy Clin Immunol 2016 Mar 31;137(3):940-2.e4. Epub 2015 Oct 31.

Molecular and Cellular Immunology, Institute of Child Health, University College London, London, United Kingdom; Great Ormond Street Hospital NHS Foundation Trust, London, United Kingdom.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.jaci.2015.08.049DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4774944PMC
March 2016

Lentiviral Engineered Fibroblasts Expressing Codon-Optimized COL7A1 Restore Anchoring Fibrils in RDEB.

J Invest Dermatol 2016 Jan;136(1):284-92

UCL Institute of Child Health, Molecular and Cellular Immunology Section & Great Ormond Street Hospital NHS Foundation Trust, London, United Kingdom. Electronic address:

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1038/JID.2015.364DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4759620PMC
January 2016

Patching up hematopoietic stem cells.

Nat Biotechnol 2015 Dec;33(12):1236-1238

Department of Molecular and Cellular Immunology, Institute of Child Health, University College London, London, UK.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1038/nbt.3425DOI Listing
December 2015

Gene therapy for monogenic disorders of the bone marrow.

Br J Haematol 2015 Oct 5;171(2):155-170. Epub 2015 Jun 5.

Infection, Immunity, Inflammation and Physiological Medicine, Molecular and Cellular Immunology Section, University College London - Institute of Child Health, London, UK.

View Article

Download full-text PDF

Source
http://doi.wiley.com/10.1111/bjh.13520
Publisher Site
http://dx.doi.org/10.1111/bjh.13520DOI Listing
October 2015

Lentivirus technologies for modulation of the immune system.

Curr Opin Pharmacol 2015 Oct 10;24:119-27. Epub 2015 Sep 10.

Molecular and Cellular Immunology, Institute of Child Health, University College London, London WC1N 1EH, UK; Department of Paediatric Immunology, Great Ormond Street Hospital NHS Foundation Trust, London WC1N 3JH, UK.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.coph.2015.08.007DOI Listing
October 2015

Editorial overview: New technologies.

Curr Opin Pharmacol 2015 Oct 28;24:vii-viii. Epub 2015 Sep 28.

Molecular and Cellular Immunology, Institute of Child Health, University College London, London WC1N 1EH, UK; Department of Paediatric Immunology, Great Ormond Street Hospital, NHS Foundation Trust, London WC1N 3JH, UK.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.coph.2015.09.005DOI Listing
October 2015

Immunodeficiency and severe susceptibility to bacterial infection associated with a loss-of-function homozygous mutation of MKL1.

Blood 2015 Sep 29;126(13):1527-35. Epub 2015 Jul 29.

Department of Immunology, Royal Free London NHS Foundation Trust, London, United Kingdom; and University College London Institute of Immunity and Transplantation, London, United Kingdom.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1182/blood-2014-12-611012DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4671330PMC
September 2015

Altered BCR and TLR signals promote enhanced positive selection of autoreactive transitional B cells in Wiskott-Aldrich syndrome.

J Exp Med 2015 Sep 14;212(10):1663-77. Epub 2015 Sep 14.

Department of Immunology and Department of Pediatrics, University of Washington School of Medicine, Seattle, WA 98195 Department of Immunology and Department of Pediatrics, University of Washington School of Medicine, Seattle, WA 98195 Center for Immunity and Immunotherapies, Seattle Children's Research Institute, Seattle, WA 98101

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1084/jem.20150585DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4577851PMC
September 2015

Coherence analysis discriminates between retroviral integration patterns in CD34(+) cells transduced under differing clinical trial conditions.

Mol Ther Methods Clin Dev 2015 29;2:15015. Epub 2015 Apr 29.

Gene Therapy Research Unit, Children's Medical Research Institute and The Children's Hospital at Westmead , Westmead, Australia ; The University of Sydney, Discipline of Paediatrics and Child Health , Westmead, Australia.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1038/mtm.2015.15DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4445430PMC
June 2015

Sheep CD34+ amniotic fluid cells have hematopoietic potential and engraft after autologous in utero transplantation.

Stem Cells 2015 Jan;33(1):122-32

Stem Cells and Regenerative Medicine Section, Institute of Child Health, University College London, London, United Kingdom; Prenatal Cell and Gene Therapy Group, Institute for Women's Health, University College London, London, United Kingdom; Department of Obstetrics and Gynaecology, Chang Gung Memorial Hospital at Linkou and Chang Gung University, College of Medicine, Taoyuan, Taiwan.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1002/stem.1839DOI Listing
January 2015

Tyrosine phosphorylation of WIP releases bound WASP and impairs podosome assembly in macrophages.

J Cell Sci 2015 Jan 20;128(2):251-65. Epub 2014 Nov 20.

Randall Division of Cell and Molecular Biophysics, King's College London, London SE1 1UL, UK

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1242/jcs.154880DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4294773PMC
January 2015

Gene correction of induced pluripotent stem cells derived from a murine model of X-linked chronic granulomatous disorder.

Methods Mol Biol 2014 ;1114:427-40

Molecular Immunology Unit, Centre for Immunodeficiency, UCL Institute of Child Health, London, UK.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1007/978-1-62703-761-7_28DOI Listing
October 2014

Patients with T⁺/low NK⁺ IL-2 receptor γ chain deficiency have differentially-impaired cytokine signaling resulting in severe combined immunodeficiency.

Eur J Immunol 2014 Oct 28;44(10):3129-40. Epub 2014 Aug 28.

Center for Chronic Immunodeficiency (CCI), University Medical Center and University of Freiburg, Freiburg, Germany; Faculty of Biology, University of Freiburg, Freiburg, Germany.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1002/eji.201444689DOI Listing
October 2014

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

N Engl J Med 2014 Oct;371(15):1407-17

From the Departments of Biotherapy (S.H.-B.-A., J. Blondeau, L.C., F.T., M.C.) and Immunology and Pediatric Hematology (S.B., G.C., D.M., B.N., C.P., F.T., A.F.) and the Centre d'Étude des Déficits Immunitaires (C.P.), Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (AP-HP), the Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM (S.H.-B.-A., J. Blondeau, L.C., F.T., M.C.), Unité de Technologies Chimiques et Biologiques pour la Santé, Centre National de la Recherche Scientifique, 8258-INSERM Unité 1022, Faculté des Sciences Pharmaceutiques et Biologiques, Université Paris Descartes (S.H.-B.-A.), Immunology Laboratory, Groupe Hospitalier Universitaire Paris-Sud, AP-HP, Le Kremlin-Bicêtre (S.H.-B.-A.), Imagine Institute, Paris Descartes-Sorbonne Paris Cité University (S.B., J. Blondeau, L.C., D.M., B.N., C.P., E.S., A.F., M.C.), INSERM Unités Mixtes de Recherche 1163, Laboratory of Human Lymphohematopoiesis (J. Blondeau, L.C., E.S., F.T., A.F., M.C.), Groupe Immunoscope, Immunology Department, Institut Pasteur (A.L.), and Collège de France (A.F.) - all in Paris; Division of Hematology-Oncology (S.-Y.P., H.B., D.G., C.E.H., G.H., L.E.L., W.B.L., D.A.W.) and Division of Immunology (L.D.N.), Boston Children's Hospital, Department of Pediatric Oncology, Dana-Farber Cancer Institute (S.-Y.P., D.G., L.E.L., W.B.L., D.A.W.), Harvard Medical School (S.-Y.P., M.A., L.E.L., W.B.L., J.R., L.E.S., A.T., L.D.N., D.A.W.), Center for Human Cell Therapy, Program in Cellular and Molecular Medicine, Boston Children's Hospital (M.A., J.R., L.E.S., A.T.), Division of Hematologic Malignancies, Dana-Farber Cancer Institute (J.R.), and the Manton Center for Orphan Disease Research (L.D.N.) - all in Boston; Great Ormond Street Hospital for Children NHS Foundation Trust (H.B.G., J.X.-B., A.J.T.) and Section of Molecular and Cellular Immunology, University College London Institute of Child Health (H.B.G., K.F.B., A.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1056/NEJMoa1404588DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4274995PMC
October 2014

Progress and prospects for engineered T cell therapies.

Br J Haematol 2014 Sep 17;166(6):818-29. Epub 2014 Jun 17.

Molecular & Cellular Immunology, Institute of Child Health, University College London, London, UK; Great Ormond Street Hospital Trust, London, UK.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1111/bjh.12981DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4282038PMC
September 2014

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

Mol Ther 2014 Sep;22(9):1562-3

Molecular and Cellular Immunology Unit, Wolfson Centre for Gene Therapy of Childhood Disease, UCL Institute of Child Health, London, UK.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1038/mt.2014.147DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4435491PMC
September 2014

Emerging neuronal precursors from amniotic fluid-derived down syndrome induced pluripotent stem cells.

Hum Gene Ther 2014 Aug;25(8):682-3

1 Department of Biological Sciences, School of Natural Sciences, Bangalore University , Bangalore 560056, Karnataka, India .

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1089/hum.2014.074DOI Listing
August 2014

Cellular control of cortical actin nucleation.

Curr Biol 2014 Jul 10;24(14):1628-1635. Epub 2014 Jul 10.

London Centre for Nanotechnology, University College London, London WC1H 0AH, UK; Department of Cell and Developmental Biology, University College London, London WC1E 6BT, UK. Electronic address:

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.cub.2014.05.069DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4110400PMC
July 2014

Lentiviral vector transduction of spermatozoa as a tool for the study of early development.

FEBS Open Bio 2014 3;4:266-75. Epub 2014 Mar 3.

Department of Surgery and Cancer, Division of Cancer, Imperial College London, Hammersmith Campus, Institute of Reproductive and Developmental Biology (IRDB), Du Cane Road, London W12 0NN, UK.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.fob.2014.02.008DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4048842PMC
June 2014

Concise review: MicroRNAs as modulators of stem cells and angiogenesis.

Stem Cells 2014 May;32(5):1059-66

Molecular Immunology Unit, Institute of Child Health, University College of London, London, United Kingdom.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1002/stem.1629DOI Listing
May 2014

Concise review: lessons learned from clinical trials of gene therapy in monogenic immunodeficiency diseases.

Stem Cells Transl Med 2014 May 28;3(5):636-42. Epub 2014 Mar 28.

Division of Hematology/Oncology, Boston Children's Hospital, and Department of Pediatric Oncology, Dana Farber Cancer Institute, Harvard Medical School, Harvard Stem Cell Institute, Boston, Massachusetts, USA; Centre for Immunodeficiency, Molecular Immunology Unit, Institute of Child Health, London, United Kingdom.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.5966/sctm.2013-0206DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4006494PMC
May 2014

Actin cytoskeletal defects in immunodeficiency.

Immunol Rev 2013 Nov;256(1):282-99

Molecular Immunology Unit, Center for Immunodeficiency, Institute of Child Health, University College London, London, UK.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1111/imr.12114DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3884764PMC
November 2013

Modelling of human Wiskott-Aldrich syndrome protein mutants in zebrafish larvae using in vivo live imaging.

J Cell Sci 2013 Sep 18;126(Pt 18):4077-84. Epub 2013 Jul 18.

School of Biochemistry, Faculty of Medical and Veterinary Sciences, University of Bristol, University Walk, Bristol, BS8 1TD, UK.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1242/jcs.128728DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3772384PMC
September 2013

Gene therapy for PIDs: progress, pitfalls and prospects.

Gene 2013 Aug 6;525(2):174-81. Epub 2013 Apr 6.

Centre for Immunodeficiency, Molecular Immunology Unit, University College London Institute of Child Health, 30 Guilford Street, London WC1N1EH, UK.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.gene.2013.03.098DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3725417PMC
August 2013

Catechins and antitumor immunity: Not MDSC's cup of tea.

Oncoimmunology 2013 Jun 8;2(6):e24443. Epub 2013 May 8.

Molecular Immunology Unit; UCL Institute of Child Health; London, UK.

View Article

Download full-text PDF

Source
http://dx.doi.org/10.4161/onci.24443DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3716739PMC
June 2013

Progress in lentiviral vector technologies.

Hum Gene Ther 2013 Feb;24(2):117-8

View Article

Download full-text PDF

Source
http://dx.doi.org/10.1089/hum.2013.2500DOI Listing
February 2013