Improved alpharetrovirus-based Gag.MS2 particles for efficient and transient delivery of CRISPR-Cas9 into target cells

Pharmacologic Inhibitor of DNA-PK, M3814, Potentiates Radiotherapy and Regresses Human Tumors in Mouse Models

(2020) Frank T. Zenke et al.   MOLECULAR CANCER THERAPEUTICS

CRISPR/Cas9 treatment causes extended TP53-dependent cell cycle arrest in human cells

(2020) Jonathan M Geisinger et al.   NUCLEIC ACIDS RESEARCH

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

(2020) Haydar Frangoul et al.   NEW ENGLAND JOURNAL OF MEDICINE

Virus-Like Particle Mediated CRISPR/Cas9 Delivery for Efficient and Safe Genome Editing

(2020) Pin Lyu et al.   Life-Basel

CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells

(2019) Yi-Li Min et al.   Science Advances

Delivering SaCas9 mRNA by lentivirus-like bionanoparticles for transient expression and efficient genome editing

(2019) Baisong Lu et al.   NUCLEIC ACIDS RESEARCH

The multiple mechanisms that regulate p53 activity and cell fate

(2019) Antonina Hafner et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Stem Cells in the Treatment of Disease

(2019) Helen M. Blau et al.   NEW ENGLAND JOURNAL OF MEDICINE

Enhancing Lentiviral and Alpharetroviral Transduction of Human Hematopoietic Stem Cells for Clinical Application

(2019) Juliane W. Schott et al.   Molecular Therapy-Methods & Clinical Development

Optimized RNP transfection for highly efficient CRISPR/Cas9-mediated gene knockout in primary T cells

(2018) Akiko Seki et al.   JOURNAL OF EXPERIMENTAL MEDICINE

p53 inhibits CRISPR–Cas9 engineering in human pluripotent stem cells

(2018) Robert J. Ihry et al.   NATURE MEDICINE

The CRISPR tool kit for genome editing and beyond

(2018) Mazhar Adli   Nature Communications

A simple strategy for retargeting lentiviral vectors to desired cell types via a disulfide-bond-forming protein-peptide pair

(2018) Nagarjun Kasaraneni et al.   Scientific Reports

Transient retrovirus-based CRISPR/Cas9 all-in-one particles for efficient, targeted gene knockout

(2018) Yvonne Knopp et al.   Molecular Therapy-Nucleic Acids

Mutant p53 as a guardian of the cancer cell

(2018) Fiamma Mantovani et al.   CELL DEATH AND DIFFERENTIATION

Cyclosporine H Overcomes Innate Immune Restrictions to Improve Lentiviral Transduction and Gene Editing In Human Hematopoietic Stem Cells

(2018) Carolina Petrillo et al.   Cell Stem Cell

Chimeric antigen receptor (CAR) T therapies for the treatment of hematologic malignancies: clinical perspective and significance

(2018) Michael M. Boyiadzis et al.   Journal for ImmunoTherapy of Cancer

Detailed comparison of retroviral vectors and promoter configurations for stable and high transgene expression in human induced pluripotent stem cells

(2017) D Hoffmann et al.   GENE THERAPY

Transposons: Moving Forward from Preclinical Studies to Clinical Trials

(2017) Jaitip Tipanee et al.   HUMAN GENE THERAPY

Potent and reversible lentiviral vector restriction in murine induced pluripotent stem cells

(2017) Franziska K. Geis et al.   Retrovirology

Marine origin of retroviruses in the early Palaeozoic Era

(2017) Pakorn Aiewsakun et al.   Nature Communications

Origin of the retroviruses: when, where, and how?

(2017) Alexander Hayward   Current Opinion in Virology

CRISPRs for Optimal Targeting: Delivery of CRISPR Components as DNA, RNA, and Protein into Cultured Cells and Single-Cell Embryos

(2016) Evguenia Kouranova et al.   HUMAN GENE THERAPY

Versatility of chemically synthesized guide RNAs for CRISPR-Cas9 genome editing

(2016) Melissa L. Kelley et al.   JOURNAL OF BIOTECHNOLOGY

Genome-editing Technologies for Gene and Cell Therapy

(2016) Morgan L Maeder et al.   MOLECULAR THERAPY

The Host RNAs in Retroviral Particles

(2016) Alice Telesnitsky et al.   Viruses-Basel

Receptor-Targeted Nipah Virus Glycoproteins Improve Cell-Type Selective Gene Delivery and Reveal a Preference for Membrane-Proximal Cell Attachment

(2016) Ruben R. Bender et al.   PLoS Pathogens

CRISPR-mediated genotypic and phenotypic correction of a chronic granulomatous disease mutation in human iPS cells

(2015) Rowan Flynn et al.   EXPERIMENTAL HEMATOLOGY

Rapid and highly efficient mammalian cell engineering via Cas9 protein transfection

(2015) Xiquan Liang et al.   JOURNAL OF BIOTECHNOLOGY

YU238259 Is a Novel Inhibitor of Homology-Dependent DNA Repair That Exhibits Synthetic Lethality and Radiosensitization in Repair-Deficient Tumors

(2015) G. C. Stachelek et al.   MOLECULAR CANCER RESEARCH

Cyclosporin A and Rapamycin Relieve Distinct Lentiviral Restriction Blocks in Hematopoietic Stem and Progenitor Cells

(2015) Carolina Petrillo et al.   MOLECULAR THERAPY

Genome-scale transcriptional activation by an engineered CRISPR-Cas9 complex

(2015) Silvana Konermann et al.   NATURE

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

(2015) Ayal Hendel et al.   NATURE BIOTECHNOLOGY

Surface-Engineered Viral Vectors for Selective and Cell Type-Specific Gene Delivery

(2015) Christian J. Buchholz et al.   TRENDS IN BIOTECHNOLOGY

Highly efficient in vitro and in vivo delivery of functional RNAs using new versatile MS2-chimeric retrovirus-like particles

(2015) Anne Prel et al.   Molecular Therapy-Methods & Clinical Development

Global Analysis of mRNA Isoform Half-Lives Reveals Stabilizing and Destabilizing Elements in Yeast

(2014) Joseph V. Geisberg et al.   CELL

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

(2014) S. Kim et al.   GENOME RESEARCH

Generation of mouse models of myeloid malignancy with combinatorial genetic lesions using CRISPR-Cas9 genome editing

(2014) Dirk Heckl et al.   NATURE BIOTECHNOLOGY

Easy quantitative assessment of genome editing by sequence trace decomposition

(2014) Eva K. Brinkman et al.   NUCLEIC ACIDS RESEARCH

Explanted Diseased Livers – A Possible Source of Metabolic Competent Primary Human Hepatocytes

(2014) Moritz Kleine et al.   PLoS One

Gene Therapy for Wiskott-Aldrich Syndrome--Long-Term Efficacy and Genotoxicity

(2014) C. J. Braun et al.   Science Translational Medicine

Non-integrating gamma-retroviral vectors as a versatile tool for transient zinc-finger nuclease delivery

(2014) Sylwia Bobis-Wozowicz et al.   Scientific Reports

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

(2014) Ulrike Mock et al.   Scientific Reports

Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery

(2014) Steven Lin et al.   eLife

Promoter and lineage independent anti-silencing activity of the A2 ubiquitous chromatin opening element for optimized human pluripotent stem cell-based gene therapy

(2013) Mania Ackermann et al.   BIOMATERIALS

Influence of untranslated regions on retroviral mRNA transfer and expression

(2013) Anne Prel et al.   BMC BIOTECHNOLOGY

Gene therapy for PIDs: Progress, pitfalls and prospects

(2013) Sayandip Mukherjee et al.   GENE

Overcoming Nonviral Gene Delivery Barriers: Perspective and Future

(2013) Charles H. Jones et al.   MOLECULAR PHARMACEUTICS

Image-based analysis of lipid nanoparticle–mediated siRNA delivery, intracellular trafficking and endosomal escape

(2013) Jerome Gilleron et al.   NATURE BIOTECHNOLOGY

High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity

(2013) Vikram Pattanayak et al.   NATURE BIOTECHNOLOGY

High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells

(2013) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering

(2013) Thomas Gaj et al.   TRENDS IN BIOTECHNOLOGY

Host restriction factors in retroviral infection: promises in virus-host interaction

(2012) Yong-Hui Zheng et al.   Retrovirology

Nucleofection induces non-specific changes in the metabolic activity of transfected cells

(2011) Fernanda Mello de Queiroz et al.   MOLECULAR BIOLOGY REPORTS

DARPins: An Efficient Targeting Domain for Lentiviral Vectors

(2011) Robert C Münch et al.   MOLECULAR THERAPY

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

(2011) Richard Gabriel et al.   NATURE BIOTECHNOLOGY

Avoiding cytotoxicity of transposases by dose-controlled mRNA delivery

(2011) Melanie Galla et al.   NUCLEIC ACIDS RESEARCH

Antiretroviral restriction factors

(2011) Theodora Hatziioannou et al.   Current Opinion in Virology

Self-Inactivating Alpharetroviral Vectors with a Split-Packaging Design

(2010) J. D. Suerth et al.   JOURNAL OF VIROLOGY

Small-Molecule Inhibition of Human Immunodeficiency Virus Type 1 Infection by Virus Capsid Destabilization

(2010) J. Shi et al.   JOURNAL OF VIROLOGY

CRISPR/Cas, the Immune System of Bacteria and Archaea

(2010) P. Horvath et al.   SCIENCE

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

(2009) N. Cartier et al.   SCIENCE

Tethering of proteins to RNAs by bacteriophage proteins

(2008) Cecile Keryer-Bibens et al.   BIOLOGY OF THE CELL

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION

Cellular Restriction of Retrovirus Particle-Mediated mRNA Transfer

(2008) M. Galla et al.   JOURNAL OF VIROLOGY

DNA-binding Specificity Is a Major Determinant of the Activity and Toxicity of Zinc-finger Nucleases

(2007) Tatjana I Cornu et al.   MOLECULAR THERAPY