siRNA: Mechanism of action, challenges, and therapeutic approaches
Owing to specific and compelling gene silencing, RNA interference (RNAi) is expected to
become an essential approach in treating a variety of infectious, hemato-oncological …
become an essential approach in treating a variety of infectious, hemato-oncological …
Hidradenitis suppurativa: a comprehensive review
A Alikhan, PJ Lynch, DB Eisen - Journal of the American Academy of …, 2009 - Elsevier
Hidradenitis suppurativa, also known as acne inversa, is a chronic, often debilitating disease
primarily affecting the axillae, perineum, and inframammary regions. Prevalence rates of up …
primarily affecting the axillae, perineum, and inframammary regions. Prevalence rates of up …
The expanding significance of keratin intermediate filaments in normal and diseased epithelia
Intermediate filaments are assembled from a diverse group of evolutionary conserved
proteins and are specified in a tissue-dependent, cell type-dependent, and context …
proteins and are specified in a tissue-dependent, cell type-dependent, and context …
First-in-human mutation-targeted siRNA phase Ib trial of an inherited skin disorder
SA Leachman, RP Hickerson, ME Schwartz… - Molecular Therapy, 2010 - cell.com
The rare skin disorder pachyonychia congenita (PC) is an autosomal dominant syndrome
that includes a disabling plantar keratoderma for which no satisfactory treatment is currently …
that includes a disabling plantar keratoderma for which no satisfactory treatment is currently …
Keratin 16 regulates innate immunity in response to epidermal barrier breach
JC Lessard, S Piña-Paz, JD Rotty… - Proceedings of the …, 2013 - National Acad Sciences
Mutations in the type I keratin 16 (Krt16) and its partner type II keratin 6 (Krt6a, Krt6b) cause
pachyonychia congenita (PC), a disorder typified by dystrophic nails, painful hyperkeratotic …
pachyonychia congenita (PC), a disorder typified by dystrophic nails, painful hyperkeratotic …
Keratin gene mutations in disorders of human skin and its appendages
Keratins, the major structural protein of all epithelia are a diverse group of cytoskeletal
scaffolding proteins that form intermediate filament networks, providing structural support to …
scaffolding proteins that form intermediate filament networks, providing structural support to …
Current RNA-based therapeutics in clinical trials
LY Zhou, Z Qin, YH Zhu, ZY He, T Xu - Current Gene Therapy, 2019 - ingentaconnect.com
Long-term research on various types of RNAs has led to further understanding of diverse
mechanisms, which eventually resulted in the rapid development of RNA-based …
mechanisms, which eventually resulted in the rapid development of RNA-based …
[HTML][HTML] Highly branched poly (β-amino ester) s for gene delivery in hereditary skin diseases
Non-viral gene therapy for hereditary skin diseases is an attractive prospect. However,
research efforts dedicated to this area are rare. Taking advantage of the branched structural …
research efforts dedicated to this area are rare. Taking advantage of the branched structural …
Diseases of the tooth: the genetic and molecular basis of inherited anomalies affecting the dentition
MT Cobourne, PT Sharpe - Wiley Interdisciplinary Reviews …, 2013 - Wiley Online Library
In humans, inherited variation in the number, size, and shape of teeth within the dentitions
are relatively common, while rarer defects of hard tissue formation, including amelogenesis …
are relatively common, while rarer defects of hard tissue formation, including amelogenesis …
A review of the clinical phenotype of 254 patients with genetically confirmed pachyonychia congenita
MJ Eliason, SA Leachman, B Feng… - Journal of the American …, 2012 - Elsevier
BACKGROUND: Pachyonychia congenita (PC) is a group of autosomal dominant
keratinizing disorders caused by a mutation in one of 4 keratin genes. Previous classification …
keratinizing disorders caused by a mutation in one of 4 keratin genes. Previous classification …