Results 101 to 110 of about 144,727 (384)

Cellular unfolded protein response against viruses used in gene therapy

open access: yesFrontiers in Microbiology, 2014
Viruses are excellent vehicles for gene therapy due to their natural ability to infect and deliver the cargo to specific tissues with high efficiency.
Dwaipayan eSen   +3 more
doaj   +1 more source

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

open access: yesNature Biotechnology, 2016
Many genetic liver diseases in newborns cause repeated, often lethal, metabolic crises. Gene therapy using nonintegrating viruses such as adeno-associated virus (AAV) is not optimal in this setting because the nonintegrating genome is lost as developing ...
Yang Yang   +11 more
semanticscholar   +1 more source

NAD+‐Boosters Improve Mitochondria Quality Control In Parkinson's Disease Models Via Mitochondrial UPR

open access: yesAdvanced Science, EarlyView.
Treatment of MPTP‐incubated cells with NAD+‐boosters increase the UPRmt/mitophagy‐related mitochondria quality control (MQC). Disturbed plasma UPRmt‐mitophagy‐mediated MQC profiles in PD patient samples. NMN inhibits motor deficit and forestalls neuropathology phenotypes of PD mice, which is required the atf4‐medicated UPRmt pathway.
Shuoting Zhou   +16 more
wiley   +1 more source

Global Connectivity and Function of Descending Spinal Input Revealed by 3D Microscopy and Retrograde Transduction [PDF]

open access: yes, 2018
The brain communicates with the spinal cord through numerous axon tracts that arise from discrete nuclei, transmit distinct functions, and often collateralize to facilitate the coordination of descending commands.
Blackmore, Murray G.   +4 more
core   +2 more sources

AAV‐mediated gene transfer of DNase I in the liver of mice with colorectal cancer reduces liver metastasis and restores local innate and adaptive immune response

open access: yesMolecular Oncology, 2020
Liver metastasis is the main cause of colorectal cancer (CRC)‐related death. Neutrophil extracellular traps (NETs) play important roles in CRC progression.
Yujia Xia   +14 more
semanticscholar   +1 more source

LRRC8A Regulates Outer Hair Cell Volume and Electromotility and is Required for Hearing

open access: yesAdvanced Science, EarlyView.
This study identifies LRRC8A‐dependent volume‐regulated anion channels (VRACs) as essential for cochlear outer hair cells' electromotility and auditory signal amplification. LRRC8A deficiency disrupts cell volume control, impairs auditory sensitivity, and causes deafness, while targeted LRRC8A re‐expression restores auditory function.
Shengnan Wang   +15 more
wiley   +1 more source

AAV cis-regulatory sequences are correlated with ocular toxicity

open access: yesProceedings of the National Academy of Sciences of the United States of America, 2019
Significance Adeno-associated viral (AAV) gene therapy is becoming an important therapeutic modality, especially for ocular diseases, due to its efficiency of gene delivery and relative lack of pathogenicity. However, AAV sometimes can cause inflammation
W. Xiong   +9 more
semanticscholar   +1 more source

Brain‐Wide Spatiotemporally Distinct Traveling Waves Drive Anxiety‐Like Behaviors in Mice

open access: yesAdvanced Science, EarlyView.
J.Liu et al. reveal the complete propagation dynamics of cortical traveling waves, from local circuitry initiation to long‐range propagation, and identify their functional relevance in modulating anxiety‐like behaviors and underlying cellular neuroplasticity in mice.
Jiaming Liu   +15 more
wiley   +1 more source

An amplified promoter system for targeted expression of calcium indicator proteins in the cerebellar cortex

open access: yesFrontiers in Neural Circuits, 2012
Recording of identified neuronal network activity using genetically encoded calcium indicators (GECIs) requires labeling that is cell type-specific and bright enough for the detection of functional signals.
Bernd eKuhn   +10 more
doaj   +1 more source

Beyond Critical Period Learning: Striatal FoxP2 Affects the Active Maintenance of Learned Vocalizations in Adulthood. [PDF]

open access: yes, 2019
In humans, mutations in the transcription factor forkhead box P2 (FOXP2) result in language disorders associated with altered striatal structure. Like speech, birdsong is learned through social interactions during maturational critical periods, and it ...
Day, Nancy F   +3 more
core  

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