Related references
Note: Only part of the references are listed.Closure of a Large Chronic Wound through Transplantation of Gene-Corrected Epidermal Stem Cells
Johann W. Bauer et al.
JOURNAL OF INVESTIGATIVE DERMATOLOGY (2017)
Safety and Wound Outcomes Following Genetically Corrected Autologous Epidermal Grafts in Patients With Recessive Dystrophic Epidermolysis Bullosa
Zurab Siprashvili et al.
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION (2016)
Lentiviral Engineered Fibroblasts Expressing Codon-Optimized COL7A1 Restore Anchoring Fibrils in RDEB
Christos Georgiadis et al.
JOURNAL OF INVESTIGATIVE DERMATOLOGY (2016)
A Gene Gun-mediated Nonviral RNA trans-splicing Strategy for Col7a1 Repair
Patricia Peking et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2016)
A single epidermal stem cell strategy for safe ex vivo gene therapy
Stephanie Droz-Georget Lathion et al.
EMBO MOLECULAR MEDICINE (2015)
Trans-Splicing Improvement by the Combined Application of Antisense Strategies
Ulrich Koller et al.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2015)
Considerations for a Successful RNA Trans-splicing Repair of Genetic Disorders
Ulrich Koller et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2014)
Long-Term Stability and Safety of Transgenic Cultured Epidermal Stem Cells in Gene Therapy of Junctional Epidermolysis Bullosa
Laura De Rosa et al.
STEM CELL REPORTS (2014)
Keratinocyte cell lines derived from severe generalized recessive Epidermolysis Bullosa patients carrying a highly recurrent COL7A1 homozygous mutation: models to assess cell and gene therapies in vitro and in vivo
Cristina Chamorro et al.
EXPERIMENTAL DERMATOLOGY (2013)
Self-inactivating MLV vectors have a reduced genotoxic profile in human epidermal keratinocytes
A. Cavazza et al.
GENE THERAPY (2013)
Trans-splicing correction of tau isoform imbalance in a mouse model of tau mis-splicing
Maria Elena Avale et al.
HUMAN MOLECULAR GENETICS (2013)
TALEN-based Gene Correction for Epidermolysis Bullosa
Mark J. Osborn et al.
MOLECULAR THERAPY (2013)
Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy
Alessandra Biffi et al.
SCIENCE (2013)
A Reporter-Based Screen to Identify Potent 3' Trans-Splicing Molecules for Endogenous RNA Repair
Eva M. Murauer et al.
HUMAN GENE THERAPY METHODS (2013)
From Bench to Bedside: Preclinical Evaluation of a Self-Inactivating ammaretroviral Vector for the Gene Therapy of X-linked Chronic Granulomatous Disease
Stefan Stein et al.
HUMAN GENE THERAPY CLINICAL DEVELOPMENT (2013)
Replacement of huntingtin exon 1 by trans-splicing
Hansjoerg Rindt et al.
CELLULAR AND MOLECULAR LIFE SCIENCES (2012)
Gene therapy for primary immunodeficiencies: part 1
Marina Cavazzana-Calvo et al.
CURRENT OPINION IN IMMUNOLOGY (2012)
The COL7A1 Mutation Database
Katarzyna Wertheim-Tysarowska et al.
HUMAN MUTATION (2012)
Whole transcriptome characterization of aberrant splicing events induced by lentiviral vector integrations
Daniela Cesana et al.
JOURNAL OF CLINICAL INVESTIGATION (2012)
Spliceosome-Mediated Trans-Splicing: The Therapeutic Cut and Paste
Verena Wally et al.
JOURNAL OF INVESTIGATIVE DERMATOLOGY (2012)
The International Dystrophic Epidermolysis Bullosa Patient Registry: An Online Database of Dystrophic Epidermolysis Bullosa Patients and Their COL7A1 Mutations
Peter C. van den Akker et al.
HUMAN MUTATION (2011)
Development of Skin-Humanized Mouse Models of Pachyonychia Congenita
Marta Garcia et al.
JOURNAL OF INVESTIGATIVE DERMATOLOGY (2011)
Functional Correction of Type VII Collagen Expression in Dystrophic Epidermolysis Bullosa
Eva M. Murauer et al.
JOURNAL OF INVESTIGATIVE DERMATOLOGY (2011)
In Vivo Assessment of Acute UVB Responses in Normal and Xeroderma Pigmentosum (XP-C) Skin-Humanized Mouse Models
Marta Garcia et al.
AMERICAN JOURNAL OF PATHOLOGY (2010)
Mechanisms controlling titer and expression of bidirectional lentiviral and gammaretroviral vectors
T. Maetzig et al.
GENE THERAPY (2010)
K14 mRNA reprogramming for dominant epidermolysis bullosa simplex
Verena Wally et al.
HUMAN MOLECULAR GENETICS (2010)
Trans-Splicing-Mediated Improvement in a Severe Mouse Model of Spinal Muscular Atrophy
Tristan H. Coady et al.
JOURNAL OF NEUROSCIENCE (2010)
SIN Retroviral Vectors Expressing COL7A1 Under Human Promoters for Ex Vivo Gene Therapy of Recessive Dystrophic Epidermolysis Bullosa
Matthias Titeux et al.
MOLECULAR THERAPY (2010)
Inherited epidermolysis bullosa
Jo-David Fine
ORPHANET JOURNAL OF RARE DISEASES (2010)
How do microRNAs regulate gene expression?
Ian G. Cannell et al.
BIOCHEMICAL SOCIETY TRANSACTIONS (2008)
A hypomorphic mouse model of dystrophic epidermolysis bullosa reveals mechanisms of disease and response to fibroblast therapy
Anja Fritsch et al.
JOURNAL OF CLINICAL INVESTIGATION (2008)
Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
Salima Hacein-Bey-Abina et al.
JOURNAL OF CLINICAL INVESTIGATION (2008)
Long-term engraftment of single genetically modified human epidermal holoclones enables safety pre-assessment of cutaneous gene therapy
Fernando Larcher et al.
MOLECULAR THERAPY (2007)
Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells
Fulvio Mavilio et al.
NATURE MEDICINE (2006)
High-affinity binding of the NC1 domain of collagen VII to laminin 5 and collagen IV
R Brittingham et al.
BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS (2006)
Equal potency of gammaretroviral and lentiviral SIN vectors for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells
A Schambach et al.
MOLECULAR THERAPY (2006)
A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency
S Hacein-Bey-Abina et al.
NEW ENGLAND JOURNAL OF MEDICINE (2003)
A preclinical model for the analysis of genetically modified human skin in vivo
M Del Rio et al.
HUMAN GENE THERAPY (2002)
In vivo assessment of gene delivery to keratinocytes by lentiviral vectors
U Kuhn et al.
JOURNAL OF VIROLOGY (2002)
Systematic determination of the packaging limit of lentiviral vectors
M Kumar et al.
HUMAN GENE THERAPY (2001)