Citation Impact
Citing Papers
Generation of knock-in primary human T cells using Cas9 ribonucleoproteins
2015 StandoutNobel
Tumor-reactive CD4+ T cells develop cytotoxic activity and eradicate large established melanoma after transfer into lymphopenic hosts
2010 StandoutNobel
Dual Inhibition of Class IA Phosphatidylinositol 3-Kinase and Mammalian Target of Rapamycin as a New Therapeutic Option for T-Cell Acute Lymphoblastic Leukemia
2009
RNA-Guided Human Genome Engineering via Cas9
2013 StandoutScience
High burden and pervasive positive selection of somatic mutations in normal human skin
2015 StandoutScience
A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice
2007
Notch Signaling in the Regulation of Stem Cell Self-Renewal and Differentiation
2010
Improved gRNA secondary structures allow editing of target sites resistant to CRISPR-Cas9 cleavage
2022 StandoutNobel
Site-specific integration in CHO cells mediated by CRISPR/Cas9 and homology-directed DNA repair pathway
2015
The promise and challenge of therapeutic genome editing
2020 StandoutNatureNobel
Digital PCR to assess gene-editing frequencies (GEF-dPCR) mediated by designer nucleases
2016
Enhancing extracellular vesicle cargo loading and functional delivery by engineering protein-lipid interactions
2024 StandoutNobel
Ultrastructural Characterization of the Lower Motor System in a Mouse Model of Krabbe Disease
2016 Standout
Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage
2016 StandoutNature
A guide to genome engineering with programmable nucleases
2014 Standout
Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response
2019
Cancer nanomedicine: progress, challenges and opportunities
2016 Standout
Adoptive T cell therapy for cancer in the clinic
2007
Efficient and Allele-Specific Genome Editing of Disease Loci in Human iPSCs
2014
Expanding the Biologist’s Toolkit with CRISPR-Cas9
2015 StandoutNobel
Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells
2015
Hematopoietic stem cell gene transfer for the treatment of hemoglobin disorders
2009
Viral vectors: from virology to transgene expression
2008
Generation of Potent and Stable Human CD4+ T Regulatory Cells by Activation-independent Expression of FOXP3
2007
Ex-vivo Gene Therapy Restores LEKTI Activity and Corrects the Architecture of Netherton Syndrome-derived Skin Grafts
2010
ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering
2013 Standout
Genome-Scale CRISPR-Mediated Control of Gene Repression and Activation
2014 Standout
A ceRNA Hypothesis: The Rosetta Stone of a Hidden RNA Language?
2011 Standout
Safe harbours for the integration of new DNA in the human genome
2011
Highly efficient homology-driven genome editing in human T cells by combining zinc-finger nuclease mRNA and AAV6 donor delivery
2015
The multilayered complexity of ceRNA crosstalk and competition
2014 StandoutNature
Engineering lymphocyte subsets: tools, trials and tribulations
2009
Virus-mediated gene delivery for human gene therapy
2012
Easy quantitative assessment of genome editing by sequence trace decomposition
2014 Standout
Chimeric Antigen Receptor–Modified T Cells in Chronic Lymphoid Leukemia
2011 Standout
Therapeutic genome editing: prospects and challenges
2015
Engineered T cells: the promise and challenges of cancer immunotherapy
2016
Tissue Kallikrein Inhibitors Based on the Sunflower Trypsin Inhibitor Scaffold – A Potential Therapeutic Intervention for Skin Diseases
2016 StandoutNobel
Targeting a CAR to the TRAC locus with CRISPR/Cas9 enhances tumour rejection
2017 Nature
Parkinson's disease
2015 Standout
Parkinson's Disease: Gene Therapies
2012
mTOR: from growth signal integration to cancer, diabetes and ageing
2010 Standout
Advances in targeted genome editing
2012
Search-and-replace genome editing without double-strand breaks or donor DNA
2019 StandoutNature
Sickle-cell disease
2010 Standout
Development and Applications of CRISPR-Cas9 for Genome Engineering
2014 Standout
Primary, Adaptive, and Acquired Resistance to Cancer Immunotherapy
2017 Standout
Targeted genome editing in human repopulating haematopoietic stem cells
2014 Nature
Improved Modeling of Side-Chain–Base Interactions and Plasticity in Protein–DNA Interface Design
2012 StandoutNobel
Applications of CRISPR technologies in research and beyond
2016 StandoutNobel
A platform for rapid prototyping of synthetic gene networks in mammalian cells
2014
CRISPR/Cas9 cleavage of viral DNA efficiently suppresses hepatitis B virus
2015 StandoutNobel
A thermostable Cas9 with increased lifetime in human plasma
2017 StandoutNobel
FOXP3+ regulatory T cells in the human immune system
2010 StandoutNobel
Stable knockdown of microRNA in vivo by lentiviral vectors
2008
Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery
2007
Directed evolution of CRISPR-Cas9 to increase its specificity
2018
CRISPR-Based Technologies for the Manipulation of Eukaryotic Genomes
2016 Standout
Cornerstones of CRISPR–Cas in drug discovery and therapy
2016 StandoutNobel
Meganucleases and Other Tools for Targeted Genome Engineering: Perspectives and Challenges for Gene Therapy
2011
Alpharetroviral self-inactivating vectors produced by a superinfection-resistant stable packaging cell line allow genetic modification of primary human T lymphocytes
2016 StandoutNobel
The Human Condition—A Molecular Approach
2014 StandoutNobel
Integration-Free iPS Cells Engineered Using Human Artificial Chromosome Vectors
2011 StandoutNobel
Gene therapy returns to centre stage
2015 Nature
Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors
2015
Massively parallel kinetic profiling of natural and engineered CRISPR nucleases
2020 StandoutNobel
Genome-editing Technologies for Gene and Cell Therapy
2016
Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe‐affected non‐human primates by intracerebral lentiviral gene therapy
2016
Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?
2016
Fanconi Anemia Gene Editing by the CRISPR/Cas9 System
2014
Adoptive Immunotherapy for Cancer or Viruses
2014
The Unexpected Importance of the Primary Structure of the Hydrophobic Part of One-Component Ionizable Amphiphilic Janus Dendrimers in Targeted mRNA Delivery Activity
2022 StandoutNobel
Genome Engineering with Targetable Nucleases
2014
Ionizable Lipid Nanoparticles for In Vivo mRNA Delivery to the Placenta during Pregnancy
2023 StandoutNobel
Long-term T-cell reconstitution after hematopoietic stem-cell transplantation in primary T-cell–immunodeficient patients is associated with myeloid chimerism and possibly the primary disease phenotype
2007
Pivoting the Plant Immune System from Dissection to Deployment
2013 StandoutScience
Targeted and Equally Distributed Delivery of mRNA to Organs with Pentaerythritol-Based One-Component Ionizable Amphiphilic Janus Dendrimers
2023 StandoutNobel
In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of β-thalassemia
2008
One-Component Multifunctional Sequence-Defined Ionizable Amphiphilic Janus Dendrimer Delivery Systems for mRNA
2021 StandoutNobel
Limited tumor infiltration by activated T effector cells restricts the therapeutic activity of regulatory T cell depletion against established melanoma
2008 StandoutNobel
Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems
2016 StandoutScience
Reprogramming homing endonuclease specificity through computational design and directed evolution
2013 StandoutNobel
A New Class of Medicines through DNA Editing
2019
Antibody-modified T cells: CARs take the front seat for hematologic malignancies
2014
Chimeric Antigen Receptor Therapy
2018 Standout
State-of-the-Art Lentiviral Vectors for Research Use: Risk Assessment and Biosafety Recommendations
2009
Viral Vectors for Gene Therapy: Translational and Clinical Outlook
2015
Targeted Delivery of mRNA with One-Component Ionizable Amphiphilic Janus Dendrimers
2021 StandoutNobel
In vivo hematopoietic stem cell modification by mRNA delivery
2023 StandoutScienceNobel
Phase I Study Protocol for Ex Vivo Lentiviral Gene Therapy for the Inherited Skin Disease, Netherton Syndrome
2013
Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease
2016
Genome Editing: A New Approach to Human Therapeutics
2015
Targeted gene knock-in by homology-directed genome editing using Cas9 ribonucleoprotein and AAV donor delivery
2017 StandoutNobel
CRISPR-Cas guides the future of genetic engineering
2018 StandoutScienceNobel
CAR T cell immunotherapy for human cancer
2018 StandoutScience
Gene Therapy
2019
Works of Daniela Cesana being referenced
Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration
2006
Lentiviral Vector Integration Profiles Differ in Rodent Postmitotic Tissues
2011
Whole transcriptome characterization of aberrant splicing events induced by lentiviral vector integrations
2012
Site-specific integration and tailoring of cassette design for sustainable gene transfer
2011
VISPA: a computational pipeline for the identification and analysis of genomic vector integration sites
2014
Reciprocal regulation of Notch and PI3K/Akt signalling in T‐ALL cells In Vitro
2007
Uncovering and Dissecting the Genotoxicity of Self-inactivating Lentiviral Vectors In Vivo
2014
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
Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection
2011