Personalized Development of Antisense Oligonucleotides for Exon Skipping Restores Type XVII Collagen Expression in Junctional Epidermolysis Bullosa

Consensus re‐classification of inherited epidermolysis bullosa and other disorders with skin fragility

(2020) C. Has et al.   BRITISH JOURNAL OF DERMATOLOGY

A cancer stem cell-like phenotype is associated with miR-10b expression in aggressive squamous cell carcinomas

(2020) Monika Wimmer et al.   Cell Communication and Signaling

Molecular Therapeutics in Development for Epidermolysis Bullosa: Update 2020

(2020) Cristina Has et al.   Molecular Diagnosis & Therapy

Translational perspectives to treat Epidermolysis bullosa – where do we stand?

(2020) Christine Prodinger et al.   EXPERIMENTAL DERMATOLOGY

QR-313, an antisense oligonucleotide, shows therapeutic efficacy for treatment of dominant and recessive dystrophic epidermolysis bullosa: a preclinical study

(2020) Olivier Bornert et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

Small molecule drug development for rare genodermatoses – evaluation of the current status in epidermolysis bullosa

(2020) Verena Wally et al.   Orphanet Journal of Rare Diseases

Deep-intronic ABCA4 variants explain missing heritability in Stargardt disease and allow correction of splice defects by antisense oligonucleotides

(2019) Riccardo Sangermano et al.   GENETICS IN MEDICINE

Gene Therapy for Epidermolysis Bullosa

(2019) M. Peter Marinkovich et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

The assembly and evaluation of antisense oligonucleotides applied in exon skipping for titin-based mutations in dilated cardiomyopathy

(2019) Julia Kelley Hahn et al.   JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY

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

Recent Advances and Perspectives in Liposomes for Cutaneous Drug Delivery

(2018) Amanda C. Carita et al.   CURRENT MEDICINAL CHEMISTRY

Diacerein orphan drug development for epidermolysis bullosa simplex: A phase 2/3 randomized, placebo-controlled, double-blind clinical trial

(2018) Verena Wally et al.   JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY

An ex-vivo RNA trans -splicing strategy to correct human generalized severe epidermolysis bullosa simplex

(2018) P. Peking et al.   BRITISH JOURNAL OF DERMATOLOGY

Precision Medicine through Antisense Oligonucleotide-Mediated Exon Skipping

(2018) Dunhui Li et al.   TRENDS IN PHARMACOLOGICAL SCIENCES

Low-dose calcipotriol can elicit wound closure, anti-microbial, and anti-neoplastic effects in epidermolysis bullosa keratinocytes

(2018) Christina Guttmann-Gruber et al.   Scientific Reports

FDA-Approved Oligonucleotide Therapies in 2017

(2017) Cy A. Stein et al.   MOLECULAR THERAPY

Antisense oligonucleotides promote exon inclusion and correct the common c.-32-13T > G (IVS1) GAA splicing variant in iPS-derived skeletal muscle cells from Pompe patients

(2017) W. Pijnappel et al.   NEUROMUSCULAR DISORDERS

Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues

(2017) Annemieke Aartsma-Rus et al.   Nucleic Acid Therapeutics

Exploiting the p53 Pathway for Therapy

(2017) Chit Fang Cheok et al.   Cold Spring Harbor Perspectives in Medicine

Amelioration of junctional epidermolysis bullosa due to exon skipping

(2016) C. Kowalewski et al.   BRITISH JOURNAL OF DERMATOLOGY

An elastic liposomal formulation for RNAi-based topical treatment of skin disorders: Proof-of-concept in the treatment of psoriasis

(2016) Eline Desmet et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS

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

Epidemiology of Inherited Epidermolysis Bullosa Based on Incidence and Prevalence Estimates From the National Epidermolysis Bullosa Registry

(2016) Jo-David Fine   JAMA Dermatology

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

Molecular architecture and function of the hemidesmosome

(2015) Gernot Walko et al.   CELL AND TISSUE RESEARCH

Hereditary epidermolysis bullosa

(2015) Martin Laimer et al.   JOURNAL DER DEUTSCHEN DERMATOLOGISCHEN GESELLSCHAFT

Exon-Skipping Antisense Oligonucleotides to Correct Missplicing in Neurogenetic Diseases

(2014) Kavitha Siva et al.   Nucleic Acid Therapeutics

Molecular mechanisms of phenotypic variability in junctional epidermolysis bullosa

(2011) D. Kiritsi et al.   JOURNAL OF MEDICAL GENETICS

Extracutaneous manifestations and complications of inherited epidermolysis bullosa

(2009) Jo-David Fine et al.   JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY

Extracutaneous manifestations and complications of inherited epidermolysis bullosa

(2009) Jo-David Fine et al.   JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY