CRISPR/Cas9-Based Cellular Engineering for Targeted Gene Overexpression

Correction of Recessive Dystrophic Epidermolysis Bullosa by Transposon-Mediated Integration of COL7A1 in Transplantable Patient-Derived Primary Keratinocytes

(2017) Maria Carmela Latella et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

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

(2017) Waseem Qasim et al.   Science Translational Medicine

Detection of Sleeping Beauty transposition in the genome of host cells by non-radioactive Southern blot analysis

(2016) Rajagopal N. Aravalli et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

Reduced intensity haplo plus single cord transplant compared to double cord transplant: improved engraftment and graft-versus-host disease-free, relapse-free survival

(2016) K. van Besien et al.   HAEMATOLOGICA

Gene-Corrected Fibroblast Therapy for Recessive Dystrophic Epidermolysis Bullosa using a Self-Inactivating COL7A1 Retroviral Vector

(2016) Joanna Jacków et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

Lentiviral Engineered Fibroblasts Expressing Codon-Optimized COL7A1 Restore Anchoring Fibrils in RDEB

(2016) Christos Georgiadis et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

Haploidentical cord transplantation—The best of both worlds

(2016) Koen van Besien et al.   SEMINARS IN HEMATOLOGY

Reduced intensity haplo plus single cord transplant compared to double cord transplant: improved engraftment and graft-versus-host disease-free, relapse-free survival

(2016) K. van Besien et al.   HAEMATOLOGICA

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

(2015) Silvana Konermann et al.   NATURE

Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors

(2015) Jianbin Wang et al.   NATURE BIOTECHNOLOGY

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

(2015) Ayal Hendel et al.   NATURE BIOTECHNOLOGY

A minimal ubiquitous chromatin opening element (UCOE) effectively prevents silencing of juxtaposed heterologous promoters by epigenetic remodeling in multipotent and pluripotent stem cells

(2015) Uta Müller-Kuller et al.   NUCLEIC ACIDS RESEARCH

Utilization of the AAVS1 safe harbor locus for hematopoietic specific transgene expression and gene knockdown in human ES cells

(2014) Amita Tiyaboonchai et al.   Stem Cell Research

Fibroblast cell therapy enhances initial healing in recessive dystrophic epidermolysis bullosa wounds: results of a randomized, vehicle-controlled trial

(2013) G. Petrof et al.   BRITISH JOURNAL OF DERMATOLOGY

TALEN-based Gene Correction for Epidermolysis Bullosa

(2013) Mark J Osborn et al.   MOLECULAR THERAPY

megaTALs: a rare-cleaving nuclease architecture for therapeutic genome engineering

(2013) Sandrine Boissel et al.   NUCLEIC ACIDS RESEARCH

Ubiquitous high-level gene expression in hematopoietic lineages provides effective lentiviral gene therapy of murine Wiskott-Aldrich syndrome

(2012) A. Astrakhan et al.   BLOOD

SIN Retroviral Vectors Expressing COL7A1 Under Human Promoters for Ex Vivo Gene Therapy of Recessive Dystrophic Epidermolysis Bullosa

(2010) Matthias Titeux et al.   MOLECULAR THERAPY

A Ubiquitous Chromatin Opening Element (UCOE) Confers Resistance to DNA Methylation–mediated Silencing of Lentiviral Vectors

(2010) Fang Zhang et al.   MOLECULAR THERAPY

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

Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases

(2009) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

Zinc-finger Nucleases: The Next Generation Emerges

(2008) Toni Cathomen et al.   MOLECULAR THERAPY