Results 171 to 180 of about 510,296 (316)

Exploring AAV‐Mediated Gene Therapy for Inner Ear Diseases: from Preclinical Success to Clinical Potential

open access: yesAdvanced Science, EarlyView.
Current preclinical studies of AAV‐mediated gene therapy explore different strategies based on the characteristics of inner ear diseases. For genetic hearing loss, approaches include the replacement of a “good gene,” removal of a “bad gene,” or direct correction of mutations through base editing.
Fan Wu   +7 more
wiley   +1 more source

Report on International Scientific Conference “Filatov Memorial Lecture-2015” dedicated to 140th Birth Anniversary of Academician V.P. Filatov

open access: yesJournal of Ophthalmology, 2015
N.V. Pasyechnikova   +4 more
doaj   +1 more source

The burden of illness in thyroid eye disease: current state of the evidence. [PDF]

open access: yesFront Ophthalmol (Lausanne)
Tamhankar MA   +7 more
europepmc   +1 more source

Long‐Lasting Auditory and Vestibular Recovery Following Gene Replacement Therapy in a Novel Usher Syndrome Type 1c Mouse Model

open access: yesAdvanced Science, EarlyView.
This study shows that gene replacement therapy using the AAV2/Anc80L65 virus can successfully restore hearing and balance in Ush1c knockout mice. The treatment leads to lasting improvements in both auditory and vestibular functions, highlighting its potential as a therapeutic approach for genetic hearing loss and vestibular disorders in humans ...
Weinan Du   +13 more
wiley   +1 more source

Rituximab for a rare pediatric case of concurrent thyroid eye disease and myasthenia gravis. [PDF]

open access: yesTher Adv Endocrinol Metab
Zhang H   +5 more
europepmc   +1 more source

Secret in the eyes - fish eye disease

open access: yesIndian Journal of Nephrology, 2016
P Rajesh, PK Maharana, Vaibhav Ingle
openaire   +3 more sources

Exome Sequencing Reveals the Genetic Architecture of Non‐syndromic Orofacial Clefts and Identifies BOC as a Novel Causal Gene

open access: yesAdvanced Science, EarlyView.
Nonsyndromic orofacial clefts (NSOFCs) are the most common craniofacial defects. Exome sequencing of 214 sporadic cases sheds new light on its genetic architecture and identifies many candidate pathogenic variants. Furthermore, functional studies establish BOC as a novel causal gene and reveal an unusual two‐locus model of inheritance via the epistatic
Qing He   +16 more
wiley   +1 more source

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