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Title
Gene therapy comes of age
Authors
Keywords
-
Journal
SCIENCE
Volume 359, Issue 6372, Pages eaan4672
Publisher
American Association for the Advancement of Science (AAAS)
Online
2018-01-17
DOI
10.1126/science.aan4672
References
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- (2017) K Talbot et al. GENE THERAPY
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- (2017) Michel Sadelain et al. NATURE
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- (2017) Janice S. Chen et al. NATURE
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- (2017) Nicole M. Gaudelli et al. NATURE
- Gene Therapy in a Patient with Sickle Cell Disease
- (2017) Jean-Antoine Ribeil et al. NEW ENGLAND JOURNAL OF MEDICINE
- Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy
- (2017) Florian Eichler et al. NEW ENGLAND JOURNAL OF MEDICINE
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- (2017) Jerry R. Mendell et al. NEW ENGLAND JOURNAL OF MEDICINE
- Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant
- (2017) Lindsey A. George et al. NEW ENGLAND JOURNAL OF MEDICINE
- CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease
- (2017) Suk See De Ravin et al. Science Translational Medicine
- Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells
- (2017) Waseem Qasim et al. Science Translational Medicine
- Chimeric antigen receptor T cell therapy in AML: How close are we?
- (2016) Saar Gill BEST PRACTICE & RESEARCH CLINICAL HAEMATOLOGY
- T cells expressing an anti-B-cell maturation antigen chimeric antigen receptor cause remissions of multiple myeloma
- (2016) S. A. Ali et al. BLOOD
- Cell and Gene Therapy for the Beta-Thalassemias: Advances and Prospects
- (2016) Jorge Mansilla-Soto et al. HUMAN GENE THERAPY
- Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial
- (2016) Jean Bennett et al. LANCET
- Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial
- (2016) Maria Sessa et al. LANCET
- Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage
- (2016) Alexis C. Komor et al. NATURE
- High-fidelity CRISPR–Cas9 nucleases with no detectable genome-wide off-target effects
- (2016) Benjamin P. Kleinstiver et al. NATURE
- Prospects for gene-engineered T cell immunotherapy for solid cancers
- (2016) Christopher A Klebanoff et al. NATURE MEDICINE
- A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition
- (2016) Elizabeth A Traxler et al. NATURE MEDICINE
- A multifunctional AAV–CRISPR–Cas9 and its host response
- (2016) Wei Leong Chew et al. NATURE METHODS
- Defining and improving the genome-wide specificities of CRISPR–Cas9 nucleases
- (2016) Shengdar Q. Tsai et al. NATURE REVIEWS GENETICS
- Paying for future success in gene therapy
- (2016) S. H. Orkin et al. SCIENCE
- Lentiviral vectors, two decades later
- (2016) L. Naldini et al. SCIENCE
- Reengineering chimeric antigen receptor T cells for targeted therapy of autoimmune disease
- (2016) C. T. Ellebrecht et al. SCIENCE
- Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency
- (2016) Suk See De Ravin et al. Science Translational Medicine
- Immunotherapy of non-Hodgkin’s lymphoma with a defined ratio of CD8 + and CD4 + CD19-specific chimeric antigen receptor–modified T cells
- (2016) Cameron J. Turtle et al. Science Translational Medicine
- Role of memory T cell subsets for adoptive immunotherapy
- (2016) Dirk H. Busch et al. SEMINARS IN IMMUNOLOGY
- Viral Vectors for Gene Therapy: Translational and Clinical Outlook
- (2015) Melissa A. Kotterman et al. Annual Review of Biomedical Engineering
- Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells
- (2015) M. D. Hoban et al. BLOOD
- In vivo genome editing of the albumin locus as a platform for protein replacement therapy
- (2015) R. Sharma et al. BLOOD
- Designing chimeric antigen receptors to effectively and safely target tumors
- (2015) Michael C Jensen et al. CURRENT OPINION IN IMMUNOLOGY
- Adeno-associated viral vectors for the treatment of hemophilia
- (2015) Katherine A. High et al. HUMAN MOLECULAR GENETICS
- Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome
- (2015) Salima Hacein-Bey Abina et al. JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
- Chemotherapy-Refractory Diffuse Large B-Cell Lymphoma and Indolent B-Cell Malignancies Can Be Effectively Treated With Autologous T Cells Expressing an Anti-CD19 Chimeric Antigen Receptor
- (2015) James N. Kochenderfer et al. JOURNAL OF CLINICAL ONCOLOGY
- T cells expressing CD19 chimeric antigen receptors for acute lymphoblastic leukaemia in children and young adults: a phase 1 dose-escalation trial
- (2015) Daniel W Lee et al. LANCET
- ASGCT and JSGT Joint Position Statement on Human Genomic Editing
- (2015) Theodore Friedmann et al. MOLECULAR THERAPY
- BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis
- (2015) Matthew C. Canver et al. NATURE
- Gene therapy returns to centre stage
- (2015) Luigi Naldini NATURE
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- (2015) Alan F. Wright NEW ENGLAND JOURNAL OF MEDICINE
- Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis
- (2015) James W.B. Bainbridge et al. NEW ENGLAND JOURNAL OF MEDICINE
- Improvement and Decline in Vision with Gene Therapy in Childhood Blindness
- (2015) Samuel G. Jacobson et al. NEW ENGLAND JOURNAL OF MEDICINE
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- (2015) I. M. Slaymaker et al. SCIENCE
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- (2015) C. Long et al. SCIENCE
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- (2015) M. Tabebordbar et al. SCIENCE
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- (2015) C. E. Nelson et al. SCIENCE
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- (2014) Dana Carroll Annual Review of Biochemistry
- Current concepts in the diagnosis and management of cytokine release syndrome
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- (2014) Geoffrey L Rogers et al. Journal of Translational Medicine
- Targeted genome editing in human repopulating haematopoietic stem cells
- (2014) Pietro Genovese et al. NATURE
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- The new frontier of genome engineering with CRISPR-Cas9
- (2014) J. A. Doudna et al. SCIENCE
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- (2014) C. J. Braun et al. Science Translational Medicine
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- (2014) M. L. Davila et al. Science Translational Medicine
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- (2013) F. Mingozzi et al. BLOOD
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- (2013) Lei S. Qi et al. CELL
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- (2013) Luke A. Gilbert et al. CELL
- The prevalence of neutralizing antibodies against adeno-associated virus capsids is reduced in young Japanese individuals
- (2013) Jun Mimuro et al. JOURNAL OF MEDICAL VIROLOGY
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- (2013) Lijing Li et al. MOLECULAR THERAPY
- Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome
- (2013) A. Aiuti et al. SCIENCE
- Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy
- (2013) A. Biffi et al. SCIENCE
- Cancer Immunotherapy
- (2013) J. Couzin-Frankel SCIENCE
- RNA-Guided Human Genome Engineering via Cas9
- (2013) P. Mali et al. SCIENCE
- Multiplex Genome Engineering Using CRISPR/Cas Systems
- (2013) L. Cong et al. SCIENCE
- CD19-Targeted T Cells Rapidly Induce Molecular Remissions in Adults with Chemotherapy-Refractory Acute Lymphoblastic Leukemia
- (2013) R. J. Brentjens et al. Science Translational Medicine
- The Basic Principles of Chimeric Antigen Receptor Design
- (2013) Michel Sadelain et al. Cancer Discovery
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- (2012) D Gaudet et al. GENE THERAPY
- The AAV Vector Toolkit: Poised at the Clinical Crossroads
- (2012) Aravind Asokan et al. MOLECULAR THERAPY
- FLASH assembly of TALENs for high-throughput genome editing
- (2012) Deepak Reyon et al. NATURE BIOTECHNOLOGY
- TALENs: a widely applicable technology for targeted genome editing
- (2012) J. Keith Joung et al. NATURE REVIEWS MOLECULAR CELL BIOLOGY
- A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity
- (2012) M. Jinek et al. SCIENCE
- Gene Therapy for Aromatic L-Amino Acid Decarboxylase Deficiency
- (2012) W.-L. Hwu et al. Science Translational Medicine
- Safety and persistence of adoptively transferred autologous CD19-targeted T cells in patients with relapsed or chemotherapy refractory B-cell leukemias
- (2011) R. J. Brentjens et al. BLOOD
- Meganucleases and Other Tools for Targeted Genome Engineering: Perspectives and Challenges for Gene Therapy
- (2011) George Silva et al. CURRENT GENE THERAPY
- Immune Responses to AAV in Clinical Trials
- (2011) Federico Mingozzi et al. CURRENT GENE THERAPY
- Adeno-Associated Viral Vector Manufacturing: Keeping Pace with Accelerating Clinical Development
- (2011) J. Fraser Wright HUMAN GENE THERAPY
- Tumor Regression in Patients With Metastatic Synovial Cell Sarcoma and Melanoma Using Genetically Engineered Lymphocytes Reactive With NY-ESO-1
- (2011) Paul F. Robbins et al. JOURNAL OF CLINICAL ONCOLOGY
- AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial
- (2011) Peter A LeWitt et al. LANCET NEUROLOGY
- Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
- (2011) Amit C. Nathwani et al. NEW ENGLAND JOURNAL OF MEDICINE
- Chimeric Antigen Receptor–Modified T Cells in Chronic Lymphoid Leukemia
- (2011) David L. Porter et al. NEW ENGLAND JOURNAL OF MEDICINE
- Concise Review: Managing Genotoxicity in the Therapeutic Modification of Stem Cells
- (2011) Christopher Baum et al. STEM CELLS
- Stem cell gene therapy: the risks of insertional mutagenesis and approaches to minimize genotoxicity
- (2011) Chuanfeng Wu et al. Frontiers of Medicine
- Eradication of B-lineage cells and regression of lymphoma in a patient treated with autologous T cells genetically engineered to recognize CD19
- (2010) J. N. Kochenderfer et al. BLOOD
- Targeting DNA Double-Strand Breaks with TAL Effector Nucleases
- (2010) M. Christian et al. GENETICS
- Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial
- (2010) William J Marks et al. LANCET NEUROLOGY
- A Phase I Study of Aromatic L-Amino Acid Decarboxylase Gene Therapy for Parkinson's Disease
- (2010) Shin-ichi Muramatsu et al. MOLECULAR THERAPY
- Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
- (2010) Marina Cavazzana-Calvo et al. NATURE
- Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease
- (2010) Stefan Stein et al. NATURE MEDICINE
- Genome editing with engineered zinc finger nucleases
- (2010) Fyodor D. Urnov et al. NATURE REVIEWS GENETICS
- TAL nucleases (TALNs): hybrid proteins composed of TAL effectors and FokI DNA-cleavage domain
- (2010) Ting Li et al. NUCLEIC ACIDS RESEARCH
- The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy
- (2009) Eugenio Montini et al. JOURNAL OF CLINICAL INVESTIGATION
- Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
- (2009) Albert M Maguire et al. LANCET
- Safety and tolerability of putaminal AADC gene therapy for Parkinson disease
- (2009) C. W. Christine et al. NEUROLOGY
- Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency
- (2009) Alessandro Aiuti et al. NEW ENGLAND JOURNAL OF MEDICINE
- Breaking the Code of DNA Binding Specificity of TAL-Type III Effectors
- (2009) J. Boch et al. SCIENCE
- A Simple Cipher Governs DNA Recognition by TAL Effectors
- (2009) M. J. Moscou et al. SCIENCE
- Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
- (2009) N. Cartier et al. SCIENCE
- Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy
- (2008) G. P. Niemeyer et al. BLOOD
- Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial
- (2008) William W. Hauswirth et al. HUMAN GENE THERAPY
- Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases
- (2008) Elena E Perez et al. NATURE BIOTECHNOLOGY
- Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis
- (2008) James W.B. Bainbridge et al. NEW ENGLAND JOURNAL OF MEDICINE
- Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses
- (2008) L. Zhong et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
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