标题
Gene therapy comes of age
作者
关键词
-
出版物
SCIENCE
Volume 359, Issue 6372, Pages eaan4672
出版商
American Association for the Advancement of Science (AAAS)
发表日期
2018-01-17
DOI
10.1126/science.aan4672
参考文献
相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。- The clinical landscape for SMA in a new therapeutic era
- (2017) K Talbot et al. GENE THERAPY
- Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
- (2017) Stephen Russell et al. LANCET
- Chimeric Antigen Receptors: A Cell and Gene Therapy Perspective
- (2017) Isabelle Rivière et al. MOLECULAR THERAPY
- Targeting a CAR to the TRAC locus with CRISPR/Cas9 enhances tumour rejection
- (2017) Justin Eyquem et al. NATURE
- Therapeutic T cell engineering
- (2017) Michel Sadelain et al. NATURE
- Enhanced proofreading governs CRISPR–Cas9 targeting accuracy
- (2017) Janice S. Chen et al. NATURE
- Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage
- (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
- Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
- (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
- Long-Term Effects of Retinal Gene Therapy in Childhood Blindness
- (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
- Rationally engineered Cas9 nucleases with improved specificity
- (2015) I. M. Slaymaker et al. SCIENCE
- Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy
- (2015) C. Long et al. SCIENCE
- In vivo gene editing in dystrophic mouse muscle and muscle stem cells
- (2015) M. Tabebordbar et al. SCIENCE
- In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy
- (2015) C. E. Nelson et al. SCIENCE
- CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes
- (2015) Puping Liang et al. Protein & Cell
- Genome Engineering with Targetable Nucleases
- (2014) Dana Carroll Annual Review of Biochemistry
- Current concepts in the diagnosis and management of cytokine release syndrome
- (2014) D. W. Lee et al. BLOOD
- The Emergence of T-Bodies/CAR T Cells
- (2014) Zelig Eshhar et al. CANCER JOURNAL
- Retroviral Vectors: From Cancer Viruses to Therapeutic Tools
- (2014) A. Dusty Miller HUMAN GENE THERAPY
- Role of the vector genome and underlying factor IX mutation in immune responses to AAV gene therapy for hemophilia B
- (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
- CRISPR-Cas systems for editing, regulating and targeting genomes
- (2014) Jeffry D Sander et al. NATURE BIOTECHNOLOGY
- Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV
- (2014) Pablo Tebas et al. NEW ENGLAND JOURNAL OF MEDICINE
- Chimeric Antigen Receptor T Cells for Sustained Remissions in Leukemia
- (2014) Shannon L. Maude et al. NEW ENGLAND JOURNAL OF MEDICINE
- A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency
- (2014) Salima Hacein-Bey-Abina et al. NEW ENGLAND JOURNAL OF MEDICINE
- Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
- (2014) Amit C. Nathwani et al. NEW ENGLAND JOURNAL OF MEDICINE
- The new frontier of genome engineering with CRISPR-Cas9
- (2014) J. A. Doudna et al. SCIENCE
- Gene Therapy for Wiskott-Aldrich Syndrome--Long-Term Efficacy and Genotoxicity
- (2014) C. J. Braun et al. Science Translational Medicine
- Efficacy and Toxicity Management of 19-28z CAR T Cell Therapy in B Cell Acute Lymphoblastic Leukemia
- (2014) M. L. Davila et al. Science Translational Medicine
- Immune responses to AAV vectors: overcoming barriers to successful gene therapy
- (2013) F. Mingozzi et al. BLOOD
- Repurposing CRISPR as an RNA-Guided Platform for Sequence-Specific Control of Gene Expression
- (2013) Lei S. Qi et al. CELL
- CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotes
- (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
- Genomic Editing of the HIV-1 Coreceptor CCR5 in Adult Hematopoietic Stem and Progenitor Cells Using Zinc Finger Nucleases
- (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
- Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial
- (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
Find Funding. Review Successful Grants.
Explore over 25,000 new funding opportunities and over 6,000,000 successful grants.
ExploreBecome a Peeref-certified reviewer
The Peeref Institute provides free reviewer training that teaches the core competencies of the academic peer review process.
Get Started