Molecular Therapeutics in Development for Epidermolysis Bullosa: Update 2020
Published 2020 View Full Article
- Home
- Publications
- Publication Search
- Publication Details
Title
Molecular Therapeutics in Development for Epidermolysis Bullosa: Update 2020
Authors
Keywords
-
Journal
Molecular Diagnosis & Therapy
Volume 24, Issue 3, Pages 299-309
Publisher
Springer Science and Business Media LLC
Online
2020-04-23
DOI
10.1007/s40291-020-00466-7
References
Ask authors/readers for more resources
Related references
Note: Only part of the references are listed.- Consensus re‐classification of inherited epidermolysis bullosa and other disorders with skin fragility
- (2020) C. Has et al. BRITISH JOURNAL OF DERMATOLOGY
- Genomics‐based treatment in a patient with two overlapping heritable skin disorders: Epidermolysis bullosa and acrodermatitis enteropathica
- (2020) Hassan Vahidnezhad et al. HUMAN MUTATION
- Context-Dependent Strategies for Enhanced Genome Editing of Genodermatoses
- (2020) Oliver Patrick March et al. Cells
- Meeting Report: The First Global Congress on Epidermolysis Bullosa, EB2020 London – Toward Treatment and Cure
- (2020) Jemima E. Mellerio et al. JOURNAL OF INVESTIGATIVE DERMATOLOGY
- Clinically Relevant Correction of Recessive Dystrophic Epidermolysis Bullosa by Dual sgRNA CRISPR/Cas9-Mediated Gene Editing
- (2019) Jose Bonafont et al. MOLECULAR THERAPY
- Identification of rigosertib for the treatment of recessive dystrophic epidermolysis bullosa-associated squamous cell carcinoma
- (2019) Velina S. Atanasova et al. CLINICAL CANCER RESEARCH
- Revertant Mosaic Fibroblasts in Recessive Dystrophic Epidermolysis Bullosa
- (2019) K. Twaroski et al. BRITISH JOURNAL OF DERMATOLOGY
- Fibroblast activation and abnormal extracellular matrix remodelling as common hallmarks in three cancer‐prone genodermatoses
- (2019) E. Chacón‐Solano et al. BRITISH JOURNAL OF DERMATOLOGY
- Epidermolysis bullosa: Advances in research and treatment
- (2019) Christine Prodinger et al. EXPERIMENTAL DERMATOLOGY
- Stem cell competition orchestrates skin homeostasis and ageing
- (2019) Nan Liu et al. NATURE
- Collagen VII Expression is Required in Both Keratinocytes and Fibroblasts for Anchoring Fibril Formation in Bilayer Engineered Skin Substitutes
- (2019) Dorothy M. Supp et al. CELL TRANSPLANTATION
- Profound immunodeficiency with severe skin disease explained by concomitant novel CARMIL2 and PLEC1 loss-of-function mutations
- (2019) Maria E. Maccari et al. CLINICAL IMMUNOLOGY
- Gene Therapy for Epidermolysis Bullosa
- (2019) M. Peter Marinkovich et al. JOURNAL OF INVESTIGATIVE DERMATOLOGY
- Natural history of growth and anaemia in children with epidermolysis bullosa: A retrospective cohort study
- (2019) A. Reimer et al. BRITISH JOURNAL OF DERMATOLOGY
- EBGene trial: Patient pre‐selection outcomes for the European GENEGRAFT ex vivo phase I/II gene therapy trial for recessive dystrophic epidermolysis bullosa
- (2019) S. Gaucher et al. BRITISH JOURNAL OF DERMATOLOGY
- Combined anti‐inflammatory and low‐dose antiproliferative therapy for squamous cell carcinomas in recessive dystrophic epidermolysis bullosa
- (2019) A. Reimer et al. JOURNAL OF THE EUROPEAN ACADEMY OF DERMATOLOGY AND VENEREOLOGY
- Natural exon skipping sets the stage for antisense oligonucleotide-mediated exon skipping as therapy for dystrophic epidermolysis bullosa
- (2019) Jeroen Bremer et al. Molecular Therapy-Nucleic Acids
- EGFR inhibition for metastasized cutaneous squamous cell carcinoma in dystrophic epidermolysis bullosa
- (2019) Andrea Diociaiuti et al. Orphanet Journal of Rare Diseases
- CRISPR/Cas9-based targeted genome editing for correction of recessive dystrophic epidermolysis bullosa using iPS cells
- (2019) Joanna Jacków et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Next generation sequencing identifies double homozygous mutations in two distinct genes (EXPH5 and COL17A1 ) in a patient with concomitant simplex and junctional epidermolysis bullosa
- (2018) Hassan Vahidnezhad et al. HUMAN MUTATION
- CRISPR/Cas9-Based Cellular Engineering for Targeted Gene Overexpression
- (2018) Mark Osborn et al. INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
- EB2017—Progress in Epidermolysis Bullosa Research toward Treatment and Cure
- (2018) Jouni Uitto et al. JOURNAL OF INVESTIGATIVE DERMATOLOGY
- Injury- and inflammation-driven skin fibrosis: The paradigm of epidermolysis bullosa
- (2018) Alexander Nyström et al. MATRIX BIOLOGY
- PD-1 Blockade with Cemiplimab in Advanced Cutaneous Squamous-Cell Carcinoma
- (2018) Michael R. Migden et al. NEW ENGLAND JOURNAL OF MEDICINE
- Gentamicin inducesLAMB3nonsense mutation readthrough and restores functional laminin 332 in junctional epidermolysis bullosa
- (2018) Vadim Lincoln et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- 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
- An ex-vivo RNA trans -splicing strategy to correct human generalized severe epidermolysis bullosa simplex
- (2018) P. Peking et al. BRITISH JOURNAL OF DERMATOLOGY
- 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
- Decorin counteracts disease progression in mice with recessive dystrophic epidermolysis bullosa
- (2018) Francesca Cianfarani et al. MATRIX BIOLOGY
- The administration of high-mobility group box 1 fragment prevents deterioration of cardiac performance by enhancement of bone marrow mesenchymal stem cell homing in the delta-sarcoglycan-deficient hamster
- (2018) Takashi Kido et al. PLoS One
- Gentamicin induces functional type VII collagen in recessive dystrophic epidermolysis bullosa patients
- (2017) David T. Woodley et al. JOURNAL OF CLINICAL INVESTIGATION
- 906 Systemic HMGB1 administration ameliorates cutaneous and non-cutaneous manifestations in a dystrophic epidermolysis bullosa model mouse
- (2017) T. Shimbo et al. JOURNAL OF INVESTIGATIVE DERMATOLOGY
- Closure of a Large Chronic Wound through Transplantation of Gene-Corrected Epidermal Stem Cells
- (2017) Johann W. Bauer et al. JOURNAL OF INVESTIGATIVE DERMATOLOGY
- Amlexanox Enhances Premature Termination Codon Read-Through in COL7A1 and Expression of Full Length Type VII Collagen: Potential Therapy for Recessive Dystrophic Epidermolysis Bullosa
- (2017) Velina S. Atanasova et al. JOURNAL OF INVESTIGATIVE DERMATOLOGY
- 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
- An RNA-targeted therapy for dystrophic epidermolysis bullosa
- (2017) Patricia Peking et al. NUCLEIC ACIDS RESEARCH
- 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
- Amelioration of junctional epidermolysis bullosa due to exon skipping
- (2016) C. Kowalewski et al. BRITISH JOURNAL OF DERMATOLOGY
- RNA-based therapies for genodermatoses
- (2016) Olivier Bornert et al. EXPERIMENTAL DERMATOLOGY
- Construction and validation of an RNA trans-splicing molecule suitable to repair a large number of COL7A1 mutations
- (2016) B Tockner et al. GENE THERAPY
- Safety and Wound Outcomes Following Genetically Corrected Autologous Epidermal Grafts in Patients With Recessive Dystrophic Epidermolysis Bullosa
- (2016) Zurab Siprashvili et al. JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
- 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
- Analysis of the functional consequences of targeted exon deletion in COL7A1 reveals prospects for dystrophic epidermolysis bullosa therapy
- (2016) Olivier Bornert et al. MOLECULAR THERAPY
- Nivolumab for Recurrent Squamous-Cell Carcinoma of the Head and Neck
- (2016) Robert L. Ferris et al. NEW ENGLAND JOURNAL OF MEDICINE
- 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
- Antisense Oligonucleotide-mediated Exon Skipping as a Systemic Therapeutic Approach for Recessive Dystrophic Epidermolysis Bullosa
- (2016) Jeroen Bremer et al. Molecular Therapy-Nucleic Acids
- ACOL7A1variant leading to in-frame skipping of exon 15 attenuates disease severity in recessive dystrophic epidermolysis bullosa
- (2015) A. Schwieger-Briel et al. BRITISH JOURNAL OF DERMATOLOGY
- Management of cutaneous squamous cell carcinoma in patients with epidermolysis bullosa: best clinical practice guidelines
- (2015) J.E. Mellerio et al. BRITISH JOURNAL OF DERMATOLOGY
- Losartan ameliorates dystrophic epidermolysis bullosa and uncovers new disease mechanisms
- (2015) A. Nystrom et al. EMBO Molecular Medicine
- Proinflammatory Cytokines and Antiskin Autoantibodies in Patients With Inherited Epidermolysis Bullosa
- (2015) Giuseppina Annicchiarico et al. MEDICINE
- Systemic high-mobility group box 1 administration suppresses skin inflammation by inducing an accumulation of PDGFRα+ mesenchymal cells from bone marrow
- (2015) Eriko Aikawa et al. Scientific Reports
- Monozygotic twins discordant for recessive dystrophic epidermolysis bullosa phenotype highlight the role of TGF-β signalling in modifying disease severity
- (2014) Teresa Odorisio et al. HUMAN MOLECULAR GENETICS
- Mechanisms of Natural Gene Therapy in Dystrophic Epidermolysis Bullosa
- (2014) Dimitra Kiritsi et al. JOURNAL OF INVESTIGATIVE DERMATOLOGY
- Inherited epidermolysis bullosa: Updated recommendations on diagnosis and classification
- (2014) Jo-David Fine et al. JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY
- Aminoglycosides Restore Full-length Type VII Collagen by Overcoming Premature Termination Codons: Therapeutic Implications for Dystrophic Epidermolysis Bullosa
- (2014) Jon Cogan et al. MOLECULAR THERAPY
- Induced pluripotent stem cells from human revertant keratinocytes for the treatment of epidermolysis bullosa
- (2014) N. Umegaki-Arao et al. Science Translational Medicine
- Use of cetuximab as an adjuvant agent to radiotherapy and surgery in recessive dystrophic epidermolysis bullosa with squamous cell carcinoma
- (2013) M. Kim et al. BRITISH JOURNAL OF DERMATOLOGY
- Collagen VII plays a dual role in wound healing
- (2013) Alexander Nyström et al. JOURNAL OF CLINICAL INVESTIGATION
- The Pathogenetic Role of IL-1β in Severe Epidermolysis Bullosa Simplex
- (2013) Verena Wally et al. JOURNAL OF INVESTIGATIVE DERMATOLOGY
- Intravenously Injected Recombinant Human Type VII Collagen Homes to Skin Wounds and Restores Skin Integrity of Dystrophic Epidermolysis Bullosa
- (2013) David T. Woodley et al. JOURNAL OF INVESTIGATIVE DERMATOLOGY
- Successful therapeutic transplantation of revertant skin in epidermolysis bullosa
- (2013) Antoni Gostyński et al. JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY
- A Lack of Premature Termination Codon Read-Through Efficacy of PTC124 (Ataluren) in a Diverse Array of Reporter Assays
- (2013) Stuart P. McElroy et al. PLOS BIOLOGY
- Fibroblast-Derived Dermal Matrix Drives Development of Aggressive Cutaneous Squamous Cell Carcinoma in Patients with Recessive Dystrophic Epidermolysis Bullosa
- (2012) Y.-Z. Ng et al. CANCER RESEARCH
- Complexities of TGF-β Targeted Cancer Therapy
- (2012) Erin C. Connolly et al. International Journal of Biological Sciences
- Natural Gene Therapy May Occur in All Patients with Generalized Non-Herlitz Junctional Epidermolysis Bullosa with COL17A1 Mutations
- (2012) Anna M.G. Pasmooij et al. JOURNAL OF INVESTIGATIVE DERMATOLOGY
- Punch grafting of chronic ulcers in patients with laminin-332–deficient, non-Herlitz junctional epidermolysis bullosa
- (2012) Wing Yan Yuen et al. JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY
- Rescue of nonsense mutations by amlexanox in human cells
- (2012) Sara Gonzalez-Hilarion et al. Orphanet Journal of Rare Diseases
- Kindler syndrome: Extension of FERMT1 mutational spectrum and natural history
- (2011) Cristina Has et al. HUMAN MUTATION
- Molecular mechanisms of phenotypic variability in junctional epidermolysis bullosa
- (2011) D. Kiritsi et al. JOURNAL OF MEDICAL GENETICS
- PDGFR -positive cells in bone marrow are mobilized by high mobility group box 1 (HMGB1) to regenerate injured epithelia
- (2011) K. Tamai et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Cetuximab Therapy of Metastasizing Cutaneous Squamous Cell Carcinoma in a Patient with Severe Recessive Dystrophic Epidermolysis Bullosa
- (2009) Andreas W. Arnold et al. DERMATOLOGY
- Epidermolysis bullosa and the risk of life-threatening cancers: The National EB Registry experience, 1986-2006
- (2008) Jo-David Fine et al. JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY
- EGFR Antagonists in Cancer Treatment
- (2008) Fortunato Ciardiello et al. NEW ENGLAND JOURNAL OF MEDICINE
- A frequent functional SNP in theMMP1promoter is associated with higher disease severity in recessive dystrophic epidermolysis bullosa
- (2007) Matthias Titeux et al. HUMAN MUTATION
Publish scientific posters with Peeref
Peeref publishes scientific posters from all research disciplines. Our Diamond Open Access policy means free access to content and no publication fees for authors.
Learn MoreCreate your own webinar
Interested in hosting your own webinar? Check the schedule and propose your idea to the Peeref Content Team.
Create Now