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Prenatal therapy in developmental disorders: drug targeting via intra-amniotic injection to treat X-linked hypohidrotic ectodermal dysplasia.

作者信息

Hermes Katharina, Schneider Pascal, Krieg Peter, Dang AnhThu, Huttner Kenneth, Schneider Holm

机构信息

German Competence Centre for Children with Ectodermal Dysplasias, Department of Pediatrics, University of Erlangen-Nürnberg, Erlangen, Germany.

Department of Biochemistry, University of Lausanne, Lausanne, Switzerland.

出版信息

J Invest Dermatol. 2014 Dec;134(12):2985-2987. doi: 10.1038/jid.2014.264. Epub 2014 Jun 20.


DOI:10.1038/jid.2014.264
PMID:24950237
Abstract
摘要

相似文献

[1]
Prenatal therapy in developmental disorders: drug targeting via intra-amniotic injection to treat X-linked hypohidrotic ectodermal dysplasia.

J Invest Dermatol. 2014-12

[2]
Prenatal Correction of X-Linked Hypohidrotic Ectodermal Dysplasia.

N Engl J Med. 2018-4-26

[3]
[Clinical and molecular study in a child with X-linked hypohidrotic ectodermal dysplasia].

Arch Argent Pediatr. 2015-12-1

[4]
[X-linked hypohidrotic ectodermal dysplasia:a case report].

Zhonghua Er Ke Za Zhi. 2013-9

[5]
Case of X-linked hypohidrotic ectodermal dysplasia with a novel EDA missense mutation.

J Dermatol. 2015-9

[6]
[Prenatal diagnosis of a fetus with X-linked hypohidrotic ectodermal dysplasia].

Zhonghua Yi Xue Yi Chuan Xue Za Zhi. 2020-11-10

[7]
Bilateral amastia in a female with X-linked hypohidrotic ectodermal dysplasia.

Br J Dermatol. 2014-9

[8]
A new EDA gene mutation in a family of X-linked hypohidrotic ectodermal dysplasia.

J Dermatol Sci. 2014-5

[9]
Phenotypic heterogeneity and mutational spectrum in a cohort of 45 Italian males subjects with X-linked ectodermal dysplasia.

Clin Genet. 2015-4

[10]
Novel nonsense mutation of the EDA gene in a Chinese family with X-linked hypohidrotic ectodermal dysplasia.

J Dermatol. 2014-11

引用本文的文献

[1]
Attitudes of female carriers of X-linked hypohidrotic ectodermal dysplasia towards prenatal treatment and their decisions during a pregnancy with a male fetus.

Orphanet J Rare Dis. 2025-4-15

[2]
Investigation of the protein corona and biodistribution profile of polymeric nanoparticles for intra-amniotic delivery.

Biomaterials. 2025-9

[3]
Ectodermal Dysplasia - An Overview and Update.

Indian Dermatol Online J. 2024-4-23

[4]
X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED): A Case Report and Overview of the Diagnosis and Multidisciplinary Modality Treatments.

Cureus. 2023-6-13

[5]
A Causal Treatment for X-Linked Hypohidrotic Ectodermal Dysplasia: Long-Term Results of Short-Term Perinatal Ectodysplasin A1 Replacement.

Int J Mol Sci. 2023-4-12

[6]
Spotlight on Genetic Kidney Diseases: A Call for Drug Delivery and Nanomedicine Solutions.

ACS Nano. 2023-4-11

[7]
Protocol for the Phase 2 EDELIFE Trial Investigating the Efficacy and Safety of Intra-Amniotic ER004 Administration to Male Subjects with X-Linked Hypohidrotic Ectodermal Dysplasia.

Genes (Basel). 2023-1-6

[8]
The Role of Ectodysplasin A on the Ocular Surface Homeostasis.

Int J Mol Sci. 2022-12-10

[9]
Ectodysplasin A1 Deficiency Leads to Osteopetrosis-like Changes in Bones of the Skull Associated with Diminished Osteoclastic Activity.

Int J Mol Sci. 2022-10-13

[10]
Ectodermal dysplasias: New perspectives on the treatment of so far immedicable genetic disorders.

Front Genet. 2022-9-6

本文引用的文献

[1]
Early respiratory and ocular involvement in X-linked hypohidrotic ectodermal dysplasia.

Eur J Pediatr. 2013-4-4

[2]
Systematic evaluation of exertional hyperthermia in children and adolescents with hypohidrotic ectodermal dysplasia: an observational study.

Pediatr Res. 2011-9

[3]
Prevalence and prevention of severe complications of hypohidrotic ectodermal dysplasia in infancy.

Early Hum Dev. 2010-6-1

[4]
Molecular aspects of hypohidrotic ectodermal dysplasia.

Am J Med Genet A. 2009-9

[5]
Conserved features and evolutionary shifts of the EDA signaling pathway involved in vertebrate skin appendage development.

Mol Biol Evol. 2008-5

[6]
A totally sex-linked gene in the house mouse.

Nature. 1952-4-19

[7]
Permanent correction of an inherited ectodermal dysplasia with recombinant EDA.

Nat Med. 2003-5

[8]
Distribution of the IgG Fc receptor, FcRn, in the human fetal intestine.

Pediatr Res. 2003-2

[9]
In utero gene transfer of human factor IX to fetal mice can induce postnatal tolerance of the exogenous clotting factor.

Blood. 2003-2-15

[10]
Sustained delivery of therapeutic concentrations of human clotting factor IX--a comparison of adenoviral and AAV vectors administered in utero.

J Gene Med. 2002

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