ReviewThe Journal of investigative dermatology2012
Novel molecular therapies for heritable skin disorders.
Review in The Journal of investigative dermatology, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
22 citing papers in PubMed.
- Challenges in Treating Genodermatoses: New Therapies at the Horizon.Frontiers in pharmacology · 2021Review
- Ex Vivo COL7A1 Correction for Recessive Dystrophic Epidermolysis Bullosa Using CRISPR/Cas9 and Homology-Directed Repair.Molecular therapy. Nucleic acids · 2018Article
- Rapid generation of Col7a1Laboratory investigation; a journal of technical methods and pathology · 2017Article
- Design and Characterization of a Human Monoclonal Antibody that Modulates Mutant Connexin 26 Hemichannels Implicated in Deafness and Skin Disorders.Frontiers in molecular neuroscience · 2017Article
- Identification of translational dermatology research priorities in the U.K.: results of an electronic Delphi exercise.The British journal of dermatology · 2015Article
- From marrow to matrix: novel gene and cell therapies for epidermolysis bullosa.Molecular therapy : the journal of the American Society of Gene Therapy · 2015Review
- A living band-aid for epidermolysis bullosa.Blood transfusion = Trasfusione del sangue · 2015Article
- Bone marrow stromal cells as immunomodulators. A primer for dermatologists.Journal of dermatological science · 2015Review
- In vivo gene silencing following non-invasive siRNA delivery into the skin using a novel topical formulation.Journal of controlled release : official journal of the Controlled Release Society · 2014Article
- Modulating the stem cell niche for tissue regeneration.Nature biotechnology · 2014Review
- Cell therapy in dermatology.Cold Spring Harbor perspectives in medicine · 2014Review
- An overview of alopecias.Cold Spring Harbor perspectives in medicine · 2014Review
- Highly rapid and efficient conversion of human fibroblasts to keratinocyte-like cells.The Journal of investigative dermatology · 2014Article
- Advances in understanding and treating dystrophic epidermolysis bullosa.F1000prime reports · 2014Review
- Allogeneic blood and bone marrow cells for the treatment of severe epidermolysis bullosa: repair of the extracellular matrix.Lancet (London, England) · 2013Review
- Defining the extracellular matrix using proteomics.International journal of experimental pathology · 2013Review
- Generation of 3D skin equivalents fully reconstituted from human induced pluripotent stem cells (iPSCs).PloS one · 2013Article
- [New developments in hereditary blistering skin diseases].Der Hautarzt; Zeitschrift fur Dermatologie, Venerologie, und verwandte Gebiete · 2013Article
- Molecular therapeutics for heritable skin diseases.The Journal of investigative dermatology · 2012Review
- Progress towards genetic and pharmacological therapies for keratin genodermatoses: current perspective and future promise.Experimental dermatology · 2012Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
Abstract
Tremendous progress has been made in the past two decades in molecular genetics of heritable skin diseases, and pathogenic mutations have been identified in as many as 500 distinct human genes. This progress has resulted in improved diagnosis with prognostic implications, has refined genetic counseling, and has formed the basis for prenatal and presymptomatic testing and preimplantation genetic diagnosis. However, there has been relatively little progress in developing effective and specific treatments for these often devastating diseases. However, very recently, a number of novel molecular strategies, including gene therapy, cell-based approaches, and protein replacement therapy, have been explored for the treatment of these conditions. This overview will focus on the prototypic heritable blistering disorders, epidermolysis bullosa, and related keratinopathies, in which significant progress has been made recently toward treatment, and it will illustrate how some of the translational research therapies have already entered the clinical arena.
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.