Context-Dependent Strategies for Enhanced Genome Editing of Genodermatoses

CRISPR-Cas9 genome editing induces megabase-scale chromosomal truncations

(2019) Grégoire Cullot et al.   Nature Communications

A New Class of Medicines through DNA Editing

(2019) Matthew H. Porteus   NEW ENGLAND JOURNAL OF MEDICINE

Clinically Relevant Correction of Recessive Dystrophic Epidermolysis Bullosa by Dual sgRNA CRISPR/Cas9-Mediated Gene Editing

(2019) Jose Bonafont et al.   MOLECULAR THERAPY

Efficient gene reframing therapy for recessive dystrophic epidermolysis bullosa using CRISPR/Cas9

(2019) Shota Takashima et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

Gene editing-mediated disruption of epidermolytic ichthyosis-associated KRT10 alleles restores filament stability in keratinocytes

(2019) Oliver Patrick March et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

Transcriptome-wide off-target RNA editing induced by CRISPR-guided DNA base editors

(2019) Julian Grünewald et al.   NATURE

Microhomologies are prevalent at Cas9-induced larger deletions

(2019) Dominic D G Owens et al.   NUCLEIC ACIDS RESEARCH

Base editor correction of COL7A1 in recessive dystrophic epidermolysis bullosa patient-derived fibroblasts and iPSCs

(2019) Mark J. Osborn et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

Predicting CRISPR/Cas9-Induced Mutations for Precise Genome Editing

(2019) Kutubuddin A. Molla et al.   TRENDS IN BIOTECHNOLOGY

Improved double-nicking strategies for COL7A1 editing by homologous recombination

(2019) Thomas Kocher et al.   Molecular Therapy-Nucleic Acids

Search-and-replace genome editing without double-strand breaks or donor DNA

(2019) Andrew V. Anzalone et al.   NATURE

CRISPR/Cas9-Based Cellular Engineering for Targeted Gene Overexpression

(2018) Mark Osborn et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

How cells ensure correct repair of DNA double-strand breaks

(2018) Joonyoung Her et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Mechanical forces in skin disorders

(2018) Chao-Kai Hsu et al.   JOURNAL OF DERMATOLOGICAL SCIENCE

Kinetics and Fidelity of the Repair of Cas9-Induced Double-Strand DNA Breaks

(2018) Eva K. Brinkman et al.   MOLECULAR CELL

Repair of double-strand breaks induced by CRISPR–Cas9 leads to large deletions and complex rearrangements

(2018) Michael Kosicki et al.   NATURE BIOTECHNOLOGY

Improving cytidine and adenine base editors by expression optimization and ancestral reconstruction

(2018) Luke W Koblan et al.   NATURE BIOTECHNOLOGY

p53 inhibits CRISPR–Cas9 engineering in human pluripotent stem cells

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

Skin Cell Heterogeneity in Development, Wound Healing, and Cancer

(2018) Emanuel Rognoni et al.   TRENDS IN CELL BIOLOGY

Deletion of a Pathogenic Mutation-Containing Exon of COL7A1 Allows Clonal Gene Editing Correction of RDEB Patient Epidermal Stem Cells

(2018) Ángeles Mencía et al.   Molecular Therapy-Nucleic Acids

CRISPR/Cas9-Mediated Treatment Ameliorates the Phenotype of the Epidermolytic Palmoplantar Keratoderma-like Mouse

(2018) Xiao-Rui Luan et al.   Molecular Therapy-Nucleic Acids

Ex vivo COL7A1 correction for Recessive Dystrophic Epidermolysis Bullosa using CRISPR/Cas9 and Homology Directed Repair

(2018) Araksya Izmiryan et al.   Molecular Therapy-Nucleic Acids

CRISPR/Cas9-Mediated In Situ Correction of LAMB3 Gene in Keratinocytes Derived from a Junctional Epidermolysis Bullosa Patient

(2018) Daniela Benati et al.   MOLECULAR THERAPY

CRISPR Correction of Duchenne Muscular Dystrophy

(2018) Yi-Li Min et al.   Annual Review of Medicine

Target-Specific Precision of CRISPR-Mediated Genome Editing

(2018) Anob M. Chakrabarti et al.   MOLECULAR CELL

Predictable and precise template-free CRISPR editing of pathogenic variants

(2018) Max W. Shen et al.   NATURE

Base editing: precision chemistry on the genome and transcriptome of living cells

(2018) Holly A. Rees et al.   NATURE REVIEWS GENETICS

Gene editing for skin diseases: designer nucleases as tools for gene therapy of skin fragility disorders

(2017) Oliver P. March et al.   EXPERIMENTAL PHYSIOLOGY

CRISPR-offinder: a CRISPR guide RNA design and off-target searching tool for user-defined protospacer adjacent motif

(2017) Changzhi Zhao et al.   International Journal of Biological Sciences

Closure of a Large Chronic Wound through Transplantation of Gene-Corrected Epidermal Stem Cells

(2017) Johann W. Bauer et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

Control of gene editing by manipulation of DNA repair mechanisms

(2017) Eric Danner et al.   MAMMALIAN GENOME

Cut and Paste: Efficient Homology-Directed Repair of a Dominant Negative KRT14 Mutation via CRISPR/Cas9 Nickases

(2017) Thomas Kocher et al.   MOLECULAR THERAPY

COL7A1 Editing via CRISPR/Cas9 in Recessive Dystrophic Epidermolysis Bullosa

(2017) Stefan Hainzl et al.   MOLECULAR THERAPY

Regeneration of the entire human epidermis using transgenic stem cells

(2017) Tobias Hirsch et al.   NATURE

Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage

(2017) Nicole M. Gaudelli et al.   NATURE

The control of DNA repair by the cell cycle

(2017) Nicole Hustedt et al.   NATURE CELL BIOLOGY

Efficient in vivo gene editing using ribonucleoproteins in skin stem cells of recessive dystrophic epidermolysis bullosa mouse model

(2017) Wenbo Wu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy

(2017) Leonela Amoasii et al.   Science Translational Medicine

In trans paired nicking triggers seamless genome editing without double-stranded DNA cutting

(2017) Xiaoyu Chen et al.   Nature Communications

Traceless Targeting and Isolation of Gene-Edited Immortalized Keratinocytes from Epidermolysis Bullosa Simplex Patients

(2017) Magomet Aushev et al.   Molecular Therapy-Methods & Clinical Development

CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice

(2017) Yu Zhang et al.   Science Advances

RNA-based therapies for genodermatoses

(2016) Olivier Bornert et al.   EXPERIMENTAL DERMATOLOGY

Targeted Exon Skipping Restores Type VII Collagen Expression and Anchoring Fibril Formation in an In Vivo RDEB Model

(2016) Sandrina Turczynski et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

Meganuclease-Mediated COL7A1 Gene Correction for Recessive Dystrophic Epidermolysis Bullosa

(2016) Araksya Izmiryan et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

DNA Repair Profiling Reveals Nonrandom Outcomes at Cas9-Mediated Breaks

(2016) Megan van Overbeek et al.   MOLECULAR CELL

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

(2016) Alexis C. Komor 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

Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems

(2016) K. Nishida et al.   SCIENCE

Repair Pathway Choices and Consequences at the Double-Strand Break

(2016) Raphael Ceccaldi et al.   TRENDS IN CELL BIOLOGY

Antisense Oligonucleotide-mediated Exon Skipping as a Systemic Therapeutic Approach for Recessive Dystrophic Epidermolysis Bullosa

(2016) Jeroen Bremer et al.   Molecular Therapy-Nucleic Acids

Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method

(2016) Jean-Paul Iyombe-Engembe et al.   Molecular Therapy-Nucleic Acids

Gene Editing for the Efficient Correction of a Recurrent COL7A1 Mutation in Recessive Dystrophic Epidermolysis Bullosa Keratinocytes

(2016) Cristina Chamorro et al.   Molecular Therapy-Nucleic Acids

Cell biology: More than skin deep

(2015) Elaine Fuchs   JOURNAL OF CELL BIOLOGY

Error-Prone Repair of DNA Double-Strand Breaks

(2015) Kasey Rodgers et al.   JOURNAL OF CELLULAR PHYSIOLOGY

Microhomology-Mediated End Joining: A Back-up Survival Mechanism or Dedicated Pathway?

(2015) Agnel Sfeir et al.   TRENDS IN BIOCHEMICAL SCIENCES

DNA-Pairing and Annealing Processes in Homologous Recombination and Homology-Directed Repair

(2015) Scott W. Morrical   Cold Spring Harbor Perspectives in Biology

An Overview of the Molecular Mechanisms of Recombinational DNA Repair

(2015) Stephen C. Kowalczykowski   Cold Spring Harbor Perspectives in Biology

Homology and enzymatic requirements of microhomology-dependent alternative end joining

(2015) S Sharma et al.   Cell Death & Disease

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

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

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

Xeroderma pigmentosum: Diagnostic procedures, interdisciplinary patient care, and novel therapeutic approaches

(2014) Janin Lehmann et al.   JOURNAL DER DEUTSCHEN DERMATOLOGISCHEN GESELLSCHAFT

Inherited epidermolysis bullosa: Updated recommendations on diagnosis and classification

(2014) Jo-David Fine et al.   JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY

Emerging interactions between skin stem cells and their niches

(2014) Ya-Chieh Hsu et al.   NATURE MEDICINE

Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA

(2014) C. Long et al.   SCIENCE

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

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

The Dissolution of Double Holliday Junctions

(2014) A. H. Bizard et al.   Cold Spring Harbor Perspectives in Biology

Holliday Junction Resolvases

(2014) H. D. M. Wyatt et al.   Cold Spring Harbor Perspectives in Biology

Mechanism of Suppression of Chromosomal Instability by DNA Polymerase POLQ

(2014) Matthew J. Yousefzadeh et al.   PLoS Genetics

Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity

(2013) F. Ann Ran et al.   CELL

Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients

(2013) David G Ousterout et al.   MOLECULAR THERAPY

TALEN-based Gene Correction for Epidermolysis Bullosa

(2013) Mark J Osborn et al.   MOLECULAR THERAPY

Targeted Gene Addition in Human Epithelial Stem Cells by Zinc-finger Nuclease-mediated Homologous Recombination

(2013) Andrea Coluccio et al.   MOLECULAR THERAPY

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

(2013) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

Targeted Gene Therapy of Xeroderma Pigmentosum Cells Using Meganuclease and TALEN™

(2013) Aurélie Dupuy et al.   PLoS One

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Quantitative Live Cell Imaging Reveals a Gradual Shift between DNA Repair Mechanisms and a Maximal Use of HR in Mid S Phase

(2012) Ketki Karanam et al.   MOLECULAR CELL

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

TheCOL7A1mutation database

(2011) Katarzyna Wertheim-Tysarowska et al.   HUMAN MUTATION

The international dystrophic epidermolysis bullosa patient registry: An online database of dystrophic epidermolysis bullosa patients and their COL7A1 mutations

(2011) Peter C. van den Akker et al.   HUMAN MUTATION

Deconstructing the skin: cytoarchitectural determinants of epidermal morphogenesis

(2011) Cory L. Simpson et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Expanding the keratin mutation database: novel and recurrent mutations and genotype-phenotype correlations in 28 patients with epidermolytic ichthyosis

(2010) M.J. Arin et al.   BRITISH JOURNAL OF DERMATOLOGY

Self-organization of keratin intermediate filaments into cross-linked networks

(2009) Chang-Hun Lee et al.   JOURNAL OF CELL BIOLOGY

Phenotypic and Genotypic Analyses of Genetic Skin Disease through the Online Mendelian Inheritance in Man (OMIM) Database

(2009) Jamison D. Feramisco et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

Recessive Epidermolytic Hyperkeratosis Caused by a Previously Unreported Termination Codon Mutation in the Keratin 10 Gene

(2009) Patrick Terheyden et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

Targeted gene knockout in mammalian cells by using engineered zinc-finger nucleases

(2008) Y. Santiago et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

MMEJ repair of double-strand breaks (director’s cut): deleted sequences and alternative endings

(2008) Mitch McVey et al.   TRENDS IN GENETICS