CRISPR/Cas9-based targeted genome editing for correction of recessive dystrophic epidermolysis bullosa using iPS cells

Establishment of integration-free induced pluripotent stem cells from human recessive dystrophic epidermolysis bullosa keratinocytes

(2018) Wakana Matsumura et al.   JOURNAL OF DERMATOLOGICAL SCIENCE

High-efficiency RNA-based reprogramming of human primary fibroblasts

(2018) Igor Kogut et al.   Nature Communications

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

(2018) Christopher A. Vakulskas et al.   NATURE MEDICINE

COL7A1 Editing via CRISPR/Cas9 in Recessive Dystrophic Epidermolysis Bullosa

(2017) Stefan Hainzl et al.   MOLECULAR THERAPY

Enhanced proofreading governs CRISPR–Cas9 targeting accuracy

(2017) Janice S. Chen et al.   NATURE

Therapeutic genome engineering via CRISPR-Cas systems

(2017) Ana M. Moreno et al.   Wiley Interdisciplinary Reviews-Systems Biology and Medicine

CRISPR-Cas9: a promising tool for gene editing on induced pluripotent stem cells

(2017) Eun Ji Kim et al.   KOREAN JOURNAL OF INTERNAL MEDICINE

One-Step Biallelic and Scarless Correction of a β-Thalassemia Mutation in Patient-Specific iPSCs without Drug Selection

(2017) Yali Liu et al.   Molecular Therapy-Nucleic Acids

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

High-fidelity CRISPR–Cas9 nucleases with no detectable genome-wide off-target effects

(2016) Benjamin P. Kleinstiver et al.   NATURE

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

Integration-free T cell-derived human induced pluripotent stem cells (iPSCs) from a patient with recessive dystrophic epidermolysis bullosa (RDEB) carrying two compound heterozygous mutations in the COL7A1 gene

(2016) Munenari Itoh et al.   Stem Cell Research

Human Skin Constructs with Spatially Controlled Vasculature Using Primary and iPSC-Derived Endothelial Cells

(2016) Hasan E. Abaci et al.   Advanced Healthcare Materials

Therapeutic genome editing: prospects and challenges

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

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

Rationally engineered Cas9 nucleases with improved specificity

(2015) I. M. Slaymaker et al.   SCIENCE

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

(2014) S. Kim et al.   GENOME RESEARCH

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

An ultrasensitive method for quantitating circulating tumor DNA with broad patient coverage

(2014) Aaron M Newman et al.   NATURE MEDICINE

Genetically corrected iPSCs as cell therapy for recessive dystrophic epidermolysis bullosa

(2014) D. Wenzel et al.   Science Translational Medicine

Human COL7A1-corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa

(2014) V. Sebastiano et al.   Science Translational Medicine

Induced pluripotent stem cells from human revertant keratinocytes for the treatment of epidermolysis bullosa

(2014) N. Umegaki-Arao et al.   Science Translational Medicine

Patient-Specific Naturally Gene-Reverted Induced Pluripotent Stem Cells in Recessive Dystrophic Epidermolysis Bullosa

(2013) Jakub Tolar et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

Genome engineering using the CRISPR-Cas9 system

(2013) F Ann Ran et al.   Nature Protocols

Generation of 3D Skin Equivalents Fully Reconstituted from Human Induced Pluripotent Stem Cells (iPSCs)

(2013) Munenari Itoh et al.   PLoS One

Building a microphysiological skin model from induced pluripotent stem cells

(2013) Zongyou Guo et al.   Stem Cell Research & Therapy

Dynamic Changes in the Copy Number of Pluripotency and Cell Proliferation Genes in Human ESCs and iPSCs during Reprogramming and Time in Culture

(2011) Louise C. Laurent et al.   Cell Stem Cell

Generation of keratinocytes from normal and recessive dystrophic epidermolysis bullosa-induced pluripotent stem cells

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

Improved vascular organization enhances functional integration of engineered skeletal muscle grafts

(2011) J. Koffler et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Induced Pluripotent Stem Cells from Individuals with Recessive Dystrophic Epidermolysis Bullosa

(2010) Jakub Tolar et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY