Results 111 to 120 of about 124,170 (327)

Allele‐Specific Regulation of PAXIP1‐AS1 by SMC3/CEBPB at rs112651172 in Psychiatric Disorders Drives Synaptic and Behavioral Dysfunctions in Mice

open access: yesAdvanced Science, EarlyView.
This study identifies a functional noncoding variant, rs112651172 (C/G), that drives allele‐specific expression of PAXIP1‐AS1 in monozygotic twin pairs discordant for schizophrenia and bipolar disorder. The risk G allele enhances CEBPB binding, leading to lncRNA upregulation, CNTNAP3 derepression, and synaptic and behavioral deficits in mice.
Chaoying Ni   +14 more
wiley   +1 more source

Sequence‐Specific Installation of Aryl Groups in RNA via DNA‐Catalyst Conjugates

open access: yesAngewandte Chemie, EarlyView.
DNA oligonucleotides carrying amine nucleophilic catalysts are employed to deliver aryl groups to complementary sites in RNA under physiological conditions. The strategy can be used to sequence‐specifically label protein‐coding RNAs, or to knock down expression selectively, with potential applications in basic transcriptome research and in future ...
Sumon Pratihar   +4 more
wiley   +2 more sources

Gene Therapy Applications in Gastroenterology and Hepatology

open access: yesCanadian Journal of Gastroenterology, 2000
Advantages and disadvantages of viral vectors and nonviral vectors for gene delivery to digestive organs are reviewed. Advances in systems for the introduction of new gene expression are described, including self-deleting retroviral transfer vectors ...
Catherine H Wu, Lanlan Shen, George Y Wu
doaj   +1 more source

High‐Throughput Strategies for Streamlining Lipid Nanoparticle Development Pipeline

open access: yesAdvanced Science, EarlyView.
This review highlights emerging state‐of‐the‐art high‐throughput strategies for optimising lipid nanoparticle formulation. By integrating combinatorial design, characterization, in vitro/in vivo screening, automation, and machine learning into a closed‐loop framework, it provides a roadmap to streamline discovery and accelerate the translation of ...
Lois Lam   +3 more
wiley   +1 more source

ATP‐Powered Signaling Between Artificial and Living Cells

open access: yesAngewandte Chemie, EarlyView.
We introduce an ATP‐powered artificial cell platform capable of selectively and transiently releasing DNA signals to facilitate communication between artificial and living cells. By engineering these DNA signals, we achieve precise activation of cellular functions and regulated delivery in response to ATP levels.
Soumya Sethi   +2 more
wiley   +2 more sources

RNA G‐Quadruplex RIBOTAC‐Mediated Targeted Degradation of lncRNA TERRA

open access: yesAdvanced Science, EarlyView.
This study introduces a selective RNA degradation strategy targeting long noncoding RNA (lncRNA) TERRA, upregulated in ALT (alternative lengthening of telomeres) cancer cells. Using ribonuclease‐targeting chimeras (RIBOTACs), RNase L is recruited to catalytically degrade TERRA, uncovering a novel approach to affect a disease associated lncRNA in ALT ...
Elias Khaskia   +2 more
wiley   +1 more source

Osmotically Tunable Microdroplets Enable Amplification‐Free CRISPR Detection of Gene Doping

open access: yesAdvanced Science, EarlyView.
A novel amplification‐free clustered regularly interspaced short palindromic repeats/CRISPR‐associated protein (CRISPR/Cas) 12a assay is integrated with osmotically shrinkable double emulsion droplets for the rapid and ultrasensitive detection of gene doping.
Jihun Han   +6 more
wiley   +1 more source

m6A Methylation‐Induced Autophagy Impairment by TFEB Regulation in SOD1‐G93A ALS Cell Model

open access: yesAmerican Journal of Medical Genetics Part B: Neuropsychiatric Genetics, EarlyView.
ABSTRACT We investigate the role of m6A RNA methylation in regulating transcription factor EB (TFEB) and its contribution to mitochondrial autophagy (mitophagy) dysfunction in amyotrophic lateral sclerosis (ALS). ALS cell models were used to analyse mitophagy markers and TFEB expression under METTL3 and TFEB modulation, using RT‐qPCR, Western blot ...
Di An   +7 more
wiley   +1 more source

Genetic diagnosis as a tool for personalized treatment of Duchenne muscular dystrophy [PDF]

open access: yes, 2016
Accurate definition of genetic mutations causing Duchenne muscular dystrophy (DMD) has always been relevant in order to provide genetic counseling to patients and families, and helps to establish the prognosis in the case where the distinction between ...
Bello, Luca, Pegoraro, Elena
core  

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