Ex vivo and in vivo genome editing: a regulatory scientific framework from early development to clinical implementation

Integration of a CD19 CAR into the TCR Alpha Chain Locus Streamlines Production of Allogeneic Gene-Edited CAR T Cells

(2017) Daniel T. MacLeod et al.   MOLECULAR THERAPY

Targeted gene knock-in by homology-directed genome editing using Cas9 ribonucleoprotein and AAV donor delivery

(2017) Thomas Gaj et al.   NUCLEIC ACIDS RESEARCH

CRISPR-targeted genome editing of mesenchymal stem cell-derived therapies for type 1 diabetes: a path to clinical success?

(2017) Dario Gerace et al.   Stem Cell Research & Therapy

Antiviral treatment strategies based on gene silencing and genome editing

(2017) Roger Badia et al.   Current Opinion in Virology

CRISPR/Cas9-Mediated Genome Editing Corrects Dystrophin Mutation in Skeletal Muscle Stem Cells in a Mouse Model of Muscle Dystrophy

(2017) Pei Zhu et al.   Molecular Therapy-Nucleic Acids

CRISPR-Cas9 mediated LAG-3 disruption in CAR-T cells

(2017) Yongping Zhang et al.   Frontiers of Medicine

Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34 + Hematopoietic Stem and Progenitor Cells

(2017) Kai-Hsin Chang et al.   Molecular Therapy-Methods & Clinical Development

Programmable Genome Editing Tools and their Regulation for Efficient Genome Engineering

(2017) Tuhin Kumar Guha et al.   Computational and Structural Biotechnology Journal

AAV Vectorization of DSB-mediated Gene Editing Technologies

(2016) Rachel J. Moser et al.   CURRENT GENE THERAPY

Genome-editing technologies for gene correction of hemophilia

(2016) Chul-Yong Park et al.   HUMAN GENETICS

Gene editing advance re-ignites debate on the merits and risks of animal to human transplantation

(2016) R. K. F. Fung et al.   INTERNAL MEDICINE JOURNAL

Site-specific genome editing for correction of induced pluripotent stem cells derived from dominant dystrophic epidermolysis bullosa

(2016) Satoru Shinkuma et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

Clinical Scale Zinc Finger Nuclease-mediated Gene Editing of PD-1 in Tumor Infiltrating Lymphocytes for the Treatment of Metastatic Melanoma

(2015) Joal D Beane et al.   MOLECULAR THERAPY

Genome Editing Technologies: Defining a Path to Clinic

(2015) Jacqueline Corrigan-Curay et al.   MOLECULAR THERAPY

Correction of Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients Through Genomic Excision of Exon 51 by Zinc Finger Nucleases

(2015) David G Ousterout et al.   MOLECULAR THERAPY

Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

(2015) Ayal Hendel et al.   NATURE BIOTECHNOLOGY

Generation of knock-in primary human T cells using Cas9 ribonucleoproteins

(2015) Kathrin Schumann et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy

(2015) David G. Ousterout et al.   Nature Communications

Functional Gene Correction for Cystic Fibrosis in Lung Epithelial Cells Generated from Patient iPSCs

(2015) Amy L. Firth et al.   Cell Reports

Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9

(2015) Hongmei Lisa Li et al.   Stem Cell Reports

Codon Swapping of Zinc Finger Nucleases Confers Expression in Primary Cells and In Vivo from a Single Lentiviral Vector

(2014) Abarrategui-Pontes Cecilia et al.   CURRENT GENE THERAPY

Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins

(2014) S. Kim et al.   GENOME RESEARCH

Inactivation of the Human Papillomavirus E6 or E7 Gene in Cervical Carcinoma Cells by Using a Bacterial CRISPR/Cas RNA-Guided Endonuclease

(2014) E. M. Kennedy et al.   JOURNAL OF VIROLOGY

Somatic Correction of Junctional Epidermolysis Bullosa by a Highly Recombinogenic AAV Variant

(2014) Sandra P Melo et al.   MOLECULAR THERAPY

Targeted genome editing in human repopulating haematopoietic stem cells

(2014) Pietro Genovese et al.   NATURE

mRNA-based therapeutics — developing a new class of drugs

(2014) Ugur Sahin et al.   NATURE REVIEWS DRUG DISCOVERY

Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV

(2014) Pablo Tebas et al.   NEW ENGLAND JOURNAL OF MEDICINE

AAV-Mediated Delivery of Zinc Finger Nucleases Targeting Hepatitis B Virus Inhibits Active Replication

(2014) Nicholas D. Weber et al.   PLoS One

Novel lentiviral vectors with mutated reverse transcriptase for mRNA delivery of TALE nucleases

(2014) Ulrike Mock et al.   Scientific Reports

Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients

(2013) Gerald Schwank et al.   Cell Stem Cell

Inactivation of Hepatitis B Virus Replication in Cultured Cells and In Vivo with Engineered Transcription Activator-Like Effector Nucleases

(2013) Kristie Bloom et al.   MOLECULAR THERAPY

TALEN-based Gene Correction for Epidermolysis Bullosa

(2013) Mark J Osborn et al.   MOLECULAR THERAPY

Nuclease-mediated gene editing by homologous recombination of the human globin locus

(2013) Richard A. Voit et al.   NUCLEIC ACIDS RESEARCH

Optimization of scarless human stem cell genome editing

(2013) Luhan Yang et al.   NUCLEIC ACIDS RESEARCH

A foundation for universal T-cell based immunotherapy: T cells engineered to express a CD19-specific chimeric-antigen-receptor and eliminate expression of endogenous TCR

(2012) H. Torikai et al.   BLOOD

mRNA as a Versatile Tool for Exogenous Protein Expression

(2012) Andreas N. Kuhn et al.   CURRENT GENE THERAPY

Challenges in Vector and Trial Design Using Retroviral Vectors for Long-Term Gene Correction in Hematopoietic Stem Cell Gene Therapy

(2012) Jacqueline Corrigan-Curay et al.   MOLECULAR THERAPY

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

(2012) Seppo Ylä-Herttuala   MOLECULAR THERAPY

Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer

(2012) Elena Provasi et al.   NATURE MEDICINE

Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells

(2012) Maarten Holkers et al.   NUCLEIC ACIDS RESEARCH

Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease

(2011) J. Zou et al.   BLOOD

In vivo genome editing restores haemostasis in a mouse model of haemophilia

(2011) Hojun Li et al.   NATURE

An unbiased genome-wide analysis of zinc-finger nuclease specificity

(2011) Richard Gabriel et al.   NATURE BIOTECHNOLOGY

De novo-engineered transcription activator-like effector (TALE) hybrid nuclease with novel DNA binding specificity creates double-strand breaks

(2011) M. M. Mahfouz et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

An Analysis of Pharmaceutical Experience with Decades of Rat Carcinogenicity Testing

(2011) Frank D. Sistare et al.   TOXICOLOGIC PATHOLOGY

Recent Advances in Lentiviral Vector Development and Applications

(2010) Janka Mátrai et al.   MOLECULAR THERAPY

Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo

(2010) Nathalia Holt et al.   NATURE BIOTECHNOLOGY

Quantification of transforming capacity and cooperation of defined genetic alterations in myeloid malignancies

(2009) Yildirim Dogan et al.   EXPERIMENTAL HEMATOLOGY

Breaking the Code of DNA Binding Specificity of TAL-Type III Effectors

(2009) J. Boch et al.   SCIENCE

Deconstructing Stem Cell Tumorigenicity: A Roadmap to Safe Regenerative Medicine

(2009) Paul S. Knoepfler   STEM CELLS

Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases

(2008) Elena E Perez et al.   NATURE BIOTECHNOLOGY

Replicative Senescence of Mesenchymal Stem Cells: A Continuous and Organized Process

(2008) Wolfgang Wagner et al.   PLoS One