Results 251 to 260 of about 992,612 (392)

Single Administration of AAV‐mAtp6v1b2 Gene Therapy Rescues Hearing and Vestibular Disorders Caused by Atp6v1b2‐Induced Lysosomal Dysfunction in Hair Cells

open access: yesAdvanced Science, EarlyView.
Wei et al. establish a hair cell‐specific conditional knockout mouse model (Atp6v1b2fl/fl;Atoh1Cre/+), and demonstrate the importance of Atp6v1b2 for hair cell through maintaining the survival of lysosomes. A single administration of AAV‐ie‐Eh3‐mAtp6v1b2 through scala media at P0‐P2 realizes function compensation and restores hearing and balance ...
Gege Wei   +15 more
wiley   +1 more source

Discovery of papyifuran A as an unusual cembrane diterpenoid from Boswellia papyrifera resin reveals EEF2 as a potent new drug target for fibrosis of multiple organs

open access: yesActa Pharmaceutica Sinica B
Chronic kidney disease (CKD) affects 8%–15% of the population globally and can cause renal failure, partly due to lack of effective treatments and drug targets.
Madhu Babu Sura   +3 more
doaj  

GDC: Integration of Multi‐Omic and Phenotypic Resources to Unravel the Genetic Pathogenesis of Hearing Loss

open access: yesAdvanced Science, EarlyView.
Overview of the Genetic Deafness Commons (GDC), integrating data from the Chinese Deafness Genetics Consortium (CDGC) and 51 public databases. The GDC provides tools for variant search, functional predictions, and gene‐disease visualization, offering insights into 201 hearing loss genes and facilitating novel gene discovery and clinical applications ...
Hui Cheng   +11 more
wiley   +1 more source

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