Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Correction of recessive dystrophic epidermolysis bullosa by homology-directed repair-mediated genome editing.

Molecular therapy : the journal of the American Society of Gene Therapy - 2 Jun 2021

Bonafont Jose, Mencía Angeles, Chacón-Solano Esteban, Srifa Wai, Vaidyanathan Sriram, Romano Rosa, Garcia Marta, Hervás-Salcedo Rosario, Ugalde Laura, Duarte Blanca, Porteus Matthew H, Del Rio Marcela, Larcher Fernando, Murillas Rodolfo

Abstract excerpt

Genome-editing technologies that enable the introduction of precise changes in DNA sequences have the potential to lead to a new class of treatments for genetic diseases. Epidermolysis bullosa (EB) is a group of rare genetic disorders characterized by extreme skin fragility. The recessive dystrophic subtype of EB (RDEB), which has one of the most severe phenotypes, is caused by mutations in COL7A1. In this study,...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Collagen Type VII
Share to CRISPR-Cas Systems

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Dependovirus

Share to Epidermolysis Bullosa Dystrophica

Share to Gene Editing

Share to Gene Transfer Techniques

Share to Keratinocytes

Share to Recombinational DNA Repair

Source

Open in PubMed Open full text

Identifiers

PMID: 33609734
DOI: 10.1016/j.ymthe.2021.02.019
PMCID: PMC8178438

Genes

COL7A1
CD34

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 4 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Correction of recessive dystrophic epidermolysis bullosa by homology-directed repair-mediated genome editing.PMID 33609734Read ↗

COL7A1 Editing via CRISPR/Cas9 in Recessive Dystrophic Epidermolysis Bullosa.Semantic proximity Read ↗

Predictable CRISPR/Cas9-Mediated COL7A1 Reframing for Dystrophic Epidermolysis Bullosa.Semantic proximity Read ↗

TALEN-based gene correction for epidermolysis bullosa.Semantic proximity Read ↗

Gene editing toward the use of autologous therapies in recessive dystrophic epidermolysis bullosa.Semantic proximity Read ↗

Base edited skin equivalents with clinical potential for ex vivo correction of recessive dystrophic epidermolysis bullosa.Semantic proximity Read ↗

COL7A1 Editing via RNA Trans-Splicing in RDEB-Derived Skin Equivalents.Semantic proximity Read ↗

Efficient in vivo gene editing using ribonucleoproteins in skin stem cells of recessive dystrophic epidermolysis bullosa mouse model.Semantic proximity Read ↗

Deletion of a Pathogenic Mutation-Containing Exon of COL7A1 Allows Clonal Gene Editing Correction of RDEB Patient Epidermal Stem CellsSemantic proximity Read ↗

Gene Therapy for Dystrophic Epidermolysis Bullosa.Semantic proximity Read ↗

CRISPR/Cas9-mediated Generation of <i>COL7A1</i> -deficient Keratinocyte Model of Recessive Dystrophic Epidermolysis BullosaSemantic proximity Read ↗

Human COL7A1-corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa.Semantic proximity Read ↗

Paired nicking-mediated COL17A1 reframing for junctional epidermolysis bullosa.Semantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.