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

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

(2017) Johann W. Bauer 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

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

A single epidermal stem cell strategy for safe ex vivo gene therapy

(2015) S. Droz-Georget Lathion et al.   EMBO Molecular Medicine

mRNA transfection of a novel TAL effector nuclease (TALEN) facilitates efficient knockout of HIV co-receptor CCR5

(2015) U. Mock et al.   NUCLEIC ACIDS RESEARCH

CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes

(2015) Puping Liang et al.   Protein & Cell

Inherited epidermolysis bullosa: Updated recommendations on diagnosis and classification

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

Long-Term Skin Regeneration From a Gene-Targeted Human Epidermal Stem Cell Clone

(2014) Blanca Duarte et al.   MOLECULAR THERAPY

Saturation editing of genomic regions by multiplex homology-directed repair

(2014) Gregory M. Findlay et al.   NATURE

Comprehensive analysis of the specificity of transcription activator-like effector nucleases

(2014) Alexandre Juillerat et al.   NUCLEIC ACIDS RESEARCH

Structures of Cas9 Endonucleases Reveal RNA-Mediated Conformational Activation

(2014) M. Jinek et al.   SCIENCE

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

(2013) F. Ann Ran et al.   CELL

TALEN-based Gene Correction for Epidermolysis Bullosa

(2013) Mark J Osborn et al.   MOLECULAR THERAPY

Directed evolution of the TALE N-terminal domain for recognition of all 5′ bases

(2013) Brian M. Lamb et al.   NUCLEIC ACIDS RESEARCH

Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells

(2012) Maarten Holkers et al.   NUCLEIC ACIDS RESEARCH

CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III

(2011) Elitza Deltcheva et al.   NATURE

Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting

(2011) Tomas Cermak et al.   NUCLEIC ACIDS RESEARCH

A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity

(2011) Claudio Mussolino et al.   NUCLEIC ACIDS RESEARCH

Targeting DNA Double-Strand Breaks with TAL Effector Nucleases

(2010) M. Christian et al.   GENETICS

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