Gene Therapy for Epidermolysis Bullosa.

Animals Autoantigens / genetics Cell Adhesion Molecules / genetics Cell Culture Techniques / methods Clinical Trials as Topic Cloning, Molecular Collagen Type VII / genetics Disease Models, Animal Epidermal Cells / metabolism Epidermolysis Bullosa / genetics Gene Transfer Techniques Genetic Engineering / methods Genetic Therapy / adverse effects Genetic Vectors / genetics Graft Rejection / immunology Humans Mutation Non-Fibrillar Collagens / genetics Retroviridae / genetics Transplantation, Autologous / adverse effects Treatment Outcome Kalinin Collagen Type XVII

Journal

The Journal of investigative dermatology
ISSN: 1523-1747
Titre abrégé: J Invest Dermatol
Pays: United States
ID NLM: 0426720

Informations de publication

Date de publication:
06 2019
Historique:
received: 27 04 2018
revised: 06 11 2018
accepted: 17 11 2018
pubmed: 10 5 2019
medline: 10 5 2020
entrez: 10 5 2019
Statut: ppublish

Résumé

Epidermolysis bullosa is a family of diseases characterized by blistering and fragility of the skin in response to mechanical trauma. Advances in our understanding of epidermolysis bullosa pathophysiology have provided the necessary foundation for the first clinical trials of gene therapy for junctional and dystrophic epidermolysis bullosa. These therapies show that gene therapy is both safe and effective, with the potential to correct the molecular and clinical phenotype of patients with epidermolysis bullosa. Improvements in gene delivery and in preventing immune reactions will be among the challenges that lie ahead during further therapeutic development.

Identifiants

DOI: 10.1016/j.jid.2018.11.036 PMID: 31068252
pubmed: 31068252
pii: S0022-202X(19)30131-9
doi: 10.1016/j.jid.2018.11.036
pii:
doi:

Substances chimiques

Autoantigens 0
Cell Adhesion Molecules 0
Collagen Type VII 0
Non-Fibrillar Collagens 0

Types de publication

Journal Article Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't Research Support, U.S. Gov't, Non-P.H.S. Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

1221-1226

Subventions

Organisme : NIAMS NIH HHS
ID : R13 AR009431
Pays : United States

Informations de copyright

Copyright © 2019 The Authors. Published by Elsevier Inc. All rights reserved.

Auteurs

M Peter Marinkovich (MP)

Department of Dermatology, Program in Epithelial Biology, Stanford University School of Medicine, Stanford, California, USA; Department of Dermatology, Palo Alto Veterans Affairs Medical Center, Palo Alto, California, USA. Electronic address: mpm@stanford.edu.

Jean Y Tang (JY)

Department of Dermatology, Program in Epithelial Biology, Stanford University School of Medicine, Stanford, California, USA.

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Classifications MeSH

questionsmedicales.fr › Eucaryotes › Animaux › Gene Therapy for Epidermolysis Bullosa.
questionsmedicales.fr › Facteurs biologiques › Antigènes › Autoantigènes › Gene Therapy for Epidermolysis Bullosa.
questionsmedicales.fr › Facteurs biologiques › Antigènes › Antigènes de surface › Molécules d'adhérence cellulaire › Gene Therapy for Epidermolysis Bullosa.
questionsmedicales.fr › Techniques d'investigation › Techniques in vitro › Techniques de culture › Techniques de culture cellulaire › Gene Therapy for Epidermolysis Bullosa.
questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Caractéristiques des études épidémiologiques › Études cliniques comme sujet › Essais cliniques comme sujet › Gene Therapy for Epidermolysis Bullosa.
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Clonage moléculaire › Gene Therapy for Epidermolysis Bullosa.
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Scléroprotéines › Protéines de la matrice extracellulaire › Collagène › Collagènes non fibrillaires › Collagène de type VII › Gene Therapy for Epidermolysis Bullosa.
questionsmedicales.fr › Techniques d'investigation › Modèles théoriques › Modèles biologiques › Modèles animaux de maladie humaine › Gene Therapy for Epidermolysis Bullosa.
questionsmedicales.fr › Cellules › Cellules épidermiques › Gene Therapy for Epidermolysis Bullosa.
questionsmedicales.fr › Maladies de la peau et du tissu conjonctif › Maladies de la peau › Dermatoses vésiculobulleuses › Épidermolyse bulleuse › Gene Therapy for Epidermolysis Bullosa.
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Techniques de transfert de gènes › Gene Therapy for Epidermolysis Bullosa.
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Gene Therapy for Epidermolysis Bullosa.
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Thérapie génétique › Gene Therapy for Epidermolysis Bullosa.
questionsmedicales.fr › Phénomènes génétiques › Structures génétiques › Vecteurs génétiques › Gene Therapy for Epidermolysis Bullosa.
questionsmedicales.fr › Phénomènes du système immunitaire › Immunologie en transplantation › Réaction de l'hôte contre le greffon › Rejet du greffon › Gene Therapy for Epidermolysis Bullosa.
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Gene Therapy for Epidermolysis Bullosa.
questionsmedicales.fr › Phénomènes génétiques › Variation génétique › Mutation › Gene Therapy for Epidermolysis Bullosa.
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Scléroprotéines › Protéines de la matrice extracellulaire › Collagène › Collagènes non fibrillaires › Gene Therapy for Epidermolysis Bullosa.
questionsmedicales.fr › Virus › Virus à ARN › Retroviridae › Gene Therapy for Epidermolysis Bullosa.
questionsmedicales.fr › Procédures de chirurgie opératoire › Transplantation › Transplantation autologue › Gene Therapy for Epidermolysis Bullosa.
questionsmedicales.fr › Qualité, accès, évaluation des soins de santé › Qualité des soins de santé › Mécanismes d'évaluation des soins de santé › Évaluation des résultats et des processus en soins de santé › Évaluation de résultat (soins) › Résultat thérapeutique › Gene Therapy for Epidermolysis Bullosa.