Prenatal Therapy in Developmental Disorders: Drug Targeting via Intra-Amniotic Injection to Treat X-Linked Hypohidrotic Ectodermal Dysplasia
| Autor: | Katharina Hermes, AnhThu Dang, Pascal Schneider, Kenneth M. Huttner, Holm Schneider, Peter Krieg |
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| Rok vydání: | 2014 |
| Předmět: |
Drug
Amniotic fluid Offspring media_common.quotation_subject Pharmacology toxicology Dermatology Bioinformatics Biochemistry Injections Mice 03 medical and health sciences Drug Delivery Systems 0302 clinical medicine Pharmacokinetics Intra-amniotic injection Animals Medicine Genetics(clinical) Pharmacology (medical) Hypohidrotic ectodermal dysplasia Molecular Biology Genetics (clinical) 030304 developmental biology media_common Medicine(all) 0303 health sciences Ectodermal Dysplasia 1 Anhidrotic business.industry Transplacental Prenatal Care General Medicine Cell Biology Ectodysplasins medicine.disease Mice Mutant Strains Human genetics 3. Good health Disease Models Animal Treatment Outcome Prenatal therapy Targeted drug delivery 030220 oncology & carcinogenesis Poster Presentation Drug delivery business |
| Zdroj: | The Journal of investigative dermatology Orphanet Journal of Rare Diseases Journal of Investigative Dermatology, vol. 134, no. 12, pp. 2985-2987 |
| DOI: | 10.1038/jid.2014.264 |
| Popis: | Background Disorders that irremediably affect fetuses make early stage therapies desirable. X-linked hypohidrotic ectodermal dysplasia (XLHED), the most common inherited disorder of ectoderm development affecting the skin and its appendages, glands, and teeth, is caused by a lack of the signaling molecule ectodysplasin A1 (EDA1). In the Tabby XLHED mouse model, repeated intravenous administration of EDA1 to pregnant mice has been shown to correct the developmental abnormalities in the offspring. Maternal drug administration, however, exposes mothers to potential drug toxicity and is limited by the variability in transplacental drug delivery. Alternative approaches to fetal treatment should entail low risk drug delivery with reproducible pharmacokinetics. We hypothesized that a single injection of an EDA1 replacement molecule into the amniotic fluid could allow sustained drug exposure at levels sufficient for correction of XLHED. |
| Databáze: | OpenAIRE |
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