Results 111 to 120 of about 125,197 (343)
Smart Nanotechnologies for Multimodal Neuromodulation and Brain Interfacing
Advanced Science, EarlyView.Recent advances in smart nanotechnologies are expanding the toolbox for brain interfacing, from wireless neuromodulation and high‐resolution sensing to targeted delivery within the central nervous system. By combining responsive nanomaterials with bioinspired design, these platforms enable multimodal interactions with neurons and glia, while also ...
Tommaso Curiale +6 more
wiley +1 more source
Advanced Science, EarlyView.A novel transcriptional module involving CsYABBY3 and CsAS1 is identified to regulate cannabinoid biosynthesis and trichome development. These factors mutually activate each other and form a protein complex via a conserved residue, acting synergistically to amplify metabolic flux through a coordinate feed‐forward mechanism.
Xuewen Zhu +18 more
wiley +1 more source
Therapy for Myhre Syndrome: Goals, Misconceptions, and Current Agents
American Journal of Medical Genetics Part C: Seminars in Medical Genetics, EarlyView.ABSTRACT Myhre Syndrome (MYHRS, MIM #139210) is a rare, multisystem connective tissue disorder caused by recurrent heterozygous gain‐of‐function pathogenic variants in the SMAD4 gene, a key player in TGF‐β signaling and a regulator of extracellular matrix homeostasis.
Alessandro De Falco +2 more
wiley +1 more source
Insights into the kinetics of siRNA-mediated gene silencing from live-cell and live-animal bioluminescent imaging [PDF]
, 2006 Small interfering RNA (siRNA) molecules are potent effectors of post-transcriptional gene silencing. Using noninvasive bioluminescent imaging and a mathematical model of siRNA delivery and function, the effects of target-specific and treatment-specific ...
Bartlett, Derek W., Davis, Mark E.
core +1 more source
Annals of Neurology, EarlyView.Objective SOD1 is the second most frequently mutated gene in European patients with amyotrophic lateral sclerosis (ALS). Given the recent authorization of SOD1‐targeted antisense oligonucleotides for SOD1‐ALS, prompt screening for SOD1 mutations in patients with ALS patients is highly recommended.
Delia Gagliardi +9 more
wiley +1 more source
Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy
Skeletal Muscle, 2011 Antisense oligonucleotides are short nucleic acids designed to bind to specific messenger RNAs in order to modulate splicing patterns or inhibit protein translation.
Goyenvalle Aurélie, Davies Kay E
doaj +1 more source
CD47 antisense oligonucleotide treatment attenuates obesity and its-associated metabolic dysfunction [PDF]
, 2023 Taesik Gwag +5 more
openalex +1 more source
Annals of Neurology, EarlyView.Objective Pathogenic variants in GNAO1 cause a spectrum of epilepsy, movement disorders, and developmental impairment. Clinical heterogeneity complicates prognosis and therapeutic development. We present the first longitudinal natural history study of GNAO1‐related disorders (GNAO1‐RD) to delineate phenotypic trajectories. Methods Sixty‐six individuals
Jana Domínguez‐Carral +52 more
wiley +1 more source
Gene Therapy Applications in Gastroenterology and Hepatology
Canadian 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