[HTML][HTML] Deafness: from genetic architecture to gene therapy

C Petit, C Bonnet, S Safieddine - Nature Reviews Genetics, 2023 - nature.com
Progress in deciphering the genetic architecture of human sensorineural hearing
impairment (SNHI) or loss, and multidisciplinary studies of mouse models, have led to the …

[HTML][HTML] Usher syndrome: Hearing loss, retinal degeneration and associated abnormalities

P Mathur, J Yang - Biochimica et Biophysica Acta (BBA)-Molecular Basis …, 2015 - Elsevier
Usher syndrome (USH), clinically and genetically heterogeneous, is the leading genetic
cause of combined hearing and vision loss. USH is classified into three types, based on the …

Comprehensive rare variant analysis via whole-genome sequencing to determine the molecular pathology of inherited retinal disease

KJ Carss, G Arno, M Erwood, J Stephens… - The American Journal of …, 2017 - cell.com
Inherited retinal disease is a common cause of visual impairment and represents a highly
heterogeneous group of conditions. Here, we present findings from a cohort of 722 …

[HTML][HTML] Gene therapy restores auditory and vestibular function in a mouse model of Usher syndrome type 1c

B Pan, C Askew, A Galvin, S Heman-Ackah… - Nature …, 2017 - nature.com
Because there are currently no biological treatments for hearing loss, we sought to advance
gene therapy approaches to treat genetic deafness. We focused on Usher syndrome, a …

[HTML][HTML] Initial sequencing and analysis of the human genome

US DOE Joint Genome Institute: Hawkins Trevor 4 … - Nature, 2001 - nature.com
The human genome holds an extraordinary trove of information about human development,
physiology, medicine and evolution. Here we report the results of an international …

Shaping the intestinal brush border

SW Crawley, MS Mooseker, MJ Tyska - Journal of Cell Biology, 2014 - rupress.org
Epithelial cells from diverse tissues, including the enterocytes that line the intestinal tract,
remodel their apical surface during differentiation to form a brush border: an array of actin …

PDZ domains: structural modules for protein complex assembly

AY Hung, M Sheng - Journal of Biological Chemistry, 2002 - ASBMB
PDZ domains1 are modular protein interaction domains that play a role in protein targeting
and protein complex assembly. Once termed Discs-large homology regions (DHRs) or …

Congenital hyperinsulinism: current trends in diagnosis and therapy

JB Arnoux, V Verkarre, C Saint-Martin… - Orphanet journal of rare …, 2011 - Springer
Congenital hyperinsulinism (HI) is an inappropriate insulin secretion by the pancreatic β-
cells secondary to various genetic disorders. The incidence is estimated at 1/50, 000 live …

addendum: A universal scaling law for atomic diffusion in condensed matter

M Dzugutov - Nature, 2001 - nature.com
The human genome holds an extraordinary trove of information about human development,
physiology, medicine and evolution. Here we report the results of an international …

Usher syndrome 1D and nonsyndromic autosomal recessive deafness DFNB12 are caused by allelic mutations of the novel cadherin-like gene CDH23

JM Bork, LM Peters, S Riazuddin, SL Bernstein… - The American Journal of …, 2001 - cell.com
Genes causing nonsyndromic autosomal recessive deafness (DFNB12) and deafness
associated with retinitis pigmentosa and vestibular dysfunction (USH1D) were previously …