The era of gene therapy: From preclinical development to clinical application

Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors

(2020) Andrew V. Anzalone et al.   NATURE BIOTECHNOLOGY

CRISPR/Cas9: A preclinical and clinical perspective for the treatment of human diseases

(2020) Garima Sharma et al.   MOLECULAR THERAPY

Glatiramer Acetate (Copaxone) is a Promising Gene Delivery Vector

(2019) Nabil A. Alhakamy et al.   MOLECULAR PHARMACEUTICS

Chimeric antigen receptor–T cell therapy manufacturing: modelling the effect of offshore production on aggregate cost of goods

(2019) Richard P. Harrison et al.   CYTOTHERAPY

The next generation of CRISPR–Cas technologies and applications

(2019) Adrian Pickar-Oliver et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Important Considerations in Modeling the Cost-Effectiveness for the First Food and Drug Administration–Approved Gene Therapy and Implications for Future One-Time Therapies

(2019) Marric Buessing et al.   VALUE IN HEALTH

Gene Therapy Leaves a Vicious Cycle

(2019) Reena Goswami et al.   Frontiers in Oncology

CAR-T cell therapy for multiple myeloma: a consensus statement from The European Myeloma Network

(2019) Philippe Moreau et al.   HAEMATOLOGICA

Exploiting autoimmunity unleashed by low‐dose immune checkpoint blockade to treat advanced cancer

(2019) Tibor Bakacs et al.   SCANDINAVIAN JOURNAL OF IMMUNOLOGY

Gene therapy clinical trials worldwide to 2017: An update

(2018) Samantha L. Ginn et al.   JOURNAL OF GENE MEDICINE

Gene therapy comes of age

(2018) Cynthia E. Dunbar et al.   SCIENCE

CAR-T cells: the long and winding road to solid tumors

(2018) Maria Michela D’Aloia et al.   Cell Death & Disease

Gene therapy: evidence, value and affordability in the US health care system

(2018) Grace Hampson et al.   Journal of Comparative Effectiveness Research

A New Approach to Treat Childhood Leukemia: Novartis' CAR-T Therapy

(2017) Frazer A. Tessema et al.   JOURNAL OF LAW MEDICINE & ETHICS

Phase I Escalating-Dose Trial of CAR-T Therapy Targeting CEA + Metastatic Colorectal Cancers

(2017) Chengcheng Zhang et al.   MOLECULAR THERAPY

Chimeric Antigen Receptors: A Cell and Gene Therapy Perspective

(2017) Isabelle Rivière et al.   MOLECULAR THERAPY

Therapeutic T cell engineering

(2017) Michel Sadelain et al.   NATURE

The human protein atlas: A spatial map of the human proteome

(2017) Peter J. Thul et al.   PROTEIN SCIENCE

Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells

(2017) Waseem Qasim et al.   Science Translational Medicine

Nanotherapy for Duchenne muscular dystrophy

(2017) Michael E. Nance et al.   Wiley Interdisciplinary Reviews-Nanomedicine and Nanobiotechnology

Delivery of ENaC siRNA to epithelial cells mediated by a targeted nanocomplex: a therapeutic strategy for cystic fibrosis

(2017) Maria D. I. Manunta et al.   Scientific Reports

CAR-T Cell Therapy for Lymphoma

(2016) Carlos A. Ramos et al.   Annual Review of Medicine

Cancer immunotherapy: the beginning of the end of cancer?

(2016) Sofia Farkona et al.   BMC Medicine

Cell and Gene Therapy for the Beta-Thalassemias: Advances and Prospects

(2016) Jorge Mansilla-Soto et al.   HUMAN GENE THERAPY

Metrics for the Human Proteome Project 2016: Progress on Identifying and Characterizing the Human Proteome, Including Post-Translational Modifications

(2016) Gilbert S. Omenn et al.   JOURNAL OF PROTEOME RESEARCH

Charge Type, Charge Spacing, and Hydrophobicity of Arginine-Rich Cell-Penetrating Peptides Dictate Gene Transfection

(2016) Nabil A. Alhakamy et al.   MOLECULAR PHARMACEUTICS

Genome-editing Technologies for Gene and Cell Therapy

(2016) Morgan L Maeder et al.   MOLECULAR THERAPY

Editing the epigenome: technologies for programmable transcription and epigenetic modulation

(2016) Pratiksha I Thakore et al.   NATURE METHODS

Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles

(2016) Ming Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A RUNX2-Mediated Epigenetic Regulation of the Survival of p53 Defective Cancer Cells

(2016) Min Hwa Shin et al.   PLoS Genetics

Clinical development of gene therapy: results and lessons from recent successes

(2016) Sandeep RP Kumar et al.   Molecular Therapy-Methods & Clinical Development

Targeting hepatitis B virus cccDNA by CRISPR/Cas9 nuclease efficiently inhibits viral replication

(2015) Chunsheng Dong et al.   ANTIVIRAL RESEARCH

Gene therapy for cancer: regulatory considerations for approval

(2015) S R Husain et al.   CANCER GENE THERAPY

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

pCMV-vegf165 Intramuscular Gene Transfer is an Effective Method of Treatment for Patients With Chronic Lower Limb Ischemia

(2015) Roman V. Deev et al.   JOURNAL OF CARDIOVASCULAR PHARMACOLOGY AND THERAPEUTICS

CAR therapy: the CD19 paradigm

(2015) Michel Sadelain   JOURNAL OF CLINICAL INVESTIGATION

ASGCT and JSGT Joint Position Statement on Human Genomic Editing

(2015) Theodore Friedmann et al.   MOLECULAR THERAPY

Gene therapy returns to centre stage

(2015) Luigi Naldini   NATURE

Therapeutic genome editing: prospects and challenges

(2015) David Benjamin Turitz Cox et al.   NATURE MEDICINE

High-throughput functional genomics using CRISPR–Cas9

(2015) Ophir Shalem et al.   NATURE REVIEWS GENETICS

Engineering CAR-T cells: Design concepts

(2015) Shivani Srivastava et al.   TRENDS IN IMMUNOLOGY

Systematic Discovery of Human Gene Function and Principles of Modular Organization through Phylogenetic Profiling

(2015) Gautam Dey et al.   Cell Reports

Baboon envelope pseudotyped LVs outperform VSV-G-LVs for gene transfer into early-cytokine-stimulated and resting HSCs

(2014) A. Girard-Gagnepain et al.   BLOOD

The Emergence of T-Bodies/CAR T Cells

(2014) Zelig Eshhar et al.   CANCER JOURNAL

Gene disruption by cell-penetrating peptide-mediated delivery of Cas9 protein and guide RNA

(2014) S. Ramakrishna et al.   GENOME RESEARCH

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice

(2014) A. Barzel et al.   NATURE

In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9

(2014) Lukasz Swiech et al.   NATURE BIOTECHNOLOGY

Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo

(2014) John A Zuris et al.   NATURE BIOTECHNOLOGY

Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype

(2014) Hao Yin 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

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

Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells

(2013) A. T. Martino et al.   BLOOD

Transcriptional Regulation and Its Misregulation in Disease

(2013) Tong Ihn Lee et al.   CELL

Super-Enhancers in the Control of Cell Identity and Disease

(2013) Denes Hnisz et al.   CELL

Gene therapy on the move

(2013) Kerstin B. Kaufmann et al.   EMBO Molecular Medicine

Gene therapy and DNA delivery systems

(2013) D. Ibraheem et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS

Polyarginine Molecular Weight Determines Transfection Efficiency of Calcium Condensed Complexes

(2013) Nabil A. Alhakamy et al.   MOLECULAR PHARMACEUTICS

Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

(2013) Leszek Lisowski et al.   NATURE

A library of TAL effector nucleases spanning the human genome

(2013) Yongsub Kim et al.   NATURE BIOTECHNOLOGY

Chimeric Antigen Receptor–Modified T Cells for Acute Lymphoid Leukemia

(2013) Stephan A. Grupp et al.   NEW ENGLAND JOURNAL OF MEDICINE

Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

(2013) A. Aiuti 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

An ex vivo gene therapy approach to treat muscular dystrophy using inducible pluripotent stem cells

(2013) Antonio Filareto et al.   Nature Communications

The Basic Principles of Chimeric Antigen Receptor Design

(2013) Michel Sadelain et al.   Cancer Discovery

Large animal models for stem cell therapy

(2013) John Harding et al.   Stem Cell Research & Therapy

Endgame: Glybera Finally Recommended for Approval as the First Gene Therapy Drug in the European Union

(2012) Seppo Ylä-Herttuala   MOLECULAR THERAPY

TALENs: a widely applicable technology for targeted genome editing

(2012) J. Keith Joung et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

CAR–T cells and solid tumors: tuning T cells to challenge an inveterate foe

(2012) David E. Gilham et al.   TRENDS IN MOLECULAR MEDICINE

Retinitis Pigmentosa: Genes and Disease Mechanisms

(2011) Francesco Parmeggiani et al.   CURRENT GENOMICS

DNA delivery to ‘ex vivo’ human liver segments

(2011) M J Herrero et al.   GENE THERAPY

Generation of CD34+ Cells from CCR5-Disrupted Human Embryonic and Induced Pluripotent Stem Cells

(2011) Yongchao Yao et al.   HUMAN GENE THERAPY

Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

(2011) Dawn E Bowles et al.   MOLECULAR THERAPY

Evolution and classification of the CRISPR–Cas systems

(2011) Kira S. Makarova et al.   NATURE REVIEWS MICROBIOLOGY

Chimeric Antigen Receptor–Modified T Cells in Chronic Lymphoid Leukemia

(2011) David L. Porter et al.   NEW ENGLAND JOURNAL OF MEDICINE

Huntington's disease: from molecular pathogenesis to clinical treatment

(2010) Christopher A Ross et al.   LANCET NEUROLOGY

Genome editing with engineered zinc finger nucleases

(2010) Fyodor D. Urnov et al.   NATURE REVIEWS GENETICS

Gene Ontology Analysis of GWA Study Data Sets Provides Insights into the Biology of Bipolar Disorder

(2009) Peter Holmans et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Progress and prospects: immune responses to viral vectors

(2009) S Nayak et al.   GENE THERAPY

Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells

(2009) Ronald T Mitsuyasu et al.   NATURE MEDICINE

Gene Therapy: Some History, Applications, Problems, and Prospects

(2008) Ana P. Cotrim et al.   TOXICOLOGIC PATHOLOGY