Results 51 to 60 of about 102,021 (242)

In Vivo Cytosolic Delivery of Biomolecules into Neurons for Super‐Resolution Imaging and Genome Modification

Advanced Science, EarlyView.
The N1 peptide specifically targets neurons, enabling cytosolic delivery of fluorescent dyes and proteins for super‐resolution imaging and functional genetic modification. Abstract Efficient delivery of biomolecules into neurons has significant impacts on therapeutic applications in the central nervous system (CNS) and fundamental neuroscience research.
Xiaoqian Ge, Joseph B. Wekselblatt, Scott Elmore, Bo Wang, Tongtong Wang, Renjinming Dai, Tingting Zhang, Harsh Dave, Mohammadaref Ghaderi, Athul Raj Anilkumar, Bill Wang, Shashank R. Sirsi, Jung‐Mo Ahn, Mikhail G. Shapiro, Yuki Oka, Carlos Lois, Zhenpeng Qin   +16 more
wiley   +1 more source

ALS antisense oligonucleotides [PDF]

Science-Business eXchange, 2013
Isis and three academic groups are developing antisense oligonucleotide therapeutics for the most common cause of ALS. The drugs target hexanucleotide repeat expansions in C9orf72 and mitigate neurotoxicity in vitro. Animal models are not yet available.
openaire   +2 more sources

Delivery of Antisense Oligonucleotides to the Cornea

Nucleic Acid Therapeutics, 2020
Antisense oligonucleotides (ASOs) are synthetic nucleic acids that recognize complementary RNA sequences inside cells and modulate gene expression. In this study, we explore the feasibility of ASO delivery to the cornea. We used quantitative polymerase chain reaction to test the efficacy of a benchmark ASO targeting a noncoding nuclear RNA, Metastasis ...
Xin Gong, Venkateswara Mootha, David R. Corey, Frank Rigo, John D. Hulleman, Viet Q. Chau, Thahza P. Prakash, Jiaxin Hu, Rafael Ufret-Vincenty   +8 more
openaire   +4 more sources

Aptamers in oncology: a diagnostic perspective [PDF]

, 2008
Nucleic acid sequences can produce a wide variety of three-dimensional conformations. Some of these structural forms are able to interact with proteins and small molecules with high affinity and specificity.
Khan, Huma, Missailidis, Sotiris
core  

Antisense-mediated exon skipping: a therapeutic strategy for titin-based dilated cardiomyopathy [PDF]

, 2015
Frameshift mutations in the TTN gene encoding titin are a major cause for inherited forms of dilated cardiomyopathy (DCM), a heart disease characterized by ventricular dilatation, systolic dysfunction, and progressive heart failure. To date, there are no
Alessandra, Moretti, Alexander, Goedel, Annemieke Aartsma, Rus, Brenda, Gerull, Daniel, Sinnecker, Elvira, Parrotta, John J., Atherton, Julie, Mcgaughran, Karl Ludwig, Laugwitz, Katja, Metzger, Ludwig, Thierfelder, Luna Simona, Pane, Martin, Schaller, Matthias, Mann, Meinrad Paul, Gawaz, Michael, Gramlich, Murgia, Marta, Qifeng, Zhou, Richard P., Harvey, Siegfried, Labeit, Sonja, Sch\uf6tterl, Thomas, Brade, Zhifen, Chen   +22 more
core   +1 more source

In Situ and Real‐Time Multi‐Modality Imaging Guided Orderly Triple‐Therapy of Tumors with a Multifunctional Nanodrug

Advanced Science, EarlyView.
A multifunctional AuNR@SiO2@MnO2@ DNA prodrugs (ASMD) nanodrug is designed to establish an in situ and real‐time photoacoustic (PA)/magnetic resonance (MR)/fluorescence (FL) multi‐modality imaging guided orderly photothermal therapy (PTT)/chemodynamic therapy (CDT)/gene therapy (GT) triple‐therapy of tumor.
Chaoyi Yang, Yuexuan Meng, Ying An, Jing Jia, Yuru Wang, Guangming Li, Yiran Li, Shan Wu, Chengyao Geng, Yunlong Chen, Huangxian Ju   +10 more
wiley   +1 more source

New Transferrin Receptor‐Targeting Conjugate Effectively Delivers DNA to Mouse Brain

Angewandte Chemie, EarlyView.
We present a novel RNA aptamer targeting the transferrin receptor (TfR), conjugated with DSPE lipid, which achieves remarkably efficient brain uptake, reaching brain‐to‐serum ratios as high as 6.5. Through fluorescence imaging and polymerase‐chain reaction analyses, we verify that this new conjugate successfully delivers a substantial amount of DNA ...
Min‐sun Song, Adrian H. Bustos, Lise Bastue, Justas Mikutavicius, Piotr Swiderski, Kelly J. Clemens, Nagy Habib, John Rossi, Kira Astakhova   +8 more
wiley   +2 more sources

Tissue pharmacokinetics of antisense oligonucleotides

Molecular Therapy - Nucleic Acids
Pharmacokinetics (PK) of antisense oligonucleotides (ASOs) is characterized by rapid distribution from plasma to tissue and slow terminal plasma elimination driven by re-distribution from tissue. Quantitative understanding of tissue PK and RNA knockdown for various ASO chemistries, conjugations, and administration routes is critical for successful drug
Erica Bäckström, Alessandro Bonetti, Per Johnsson, Stefan Öhlin, Anders Dahlén, Patrik Andersson, Shalini Andersson, Peter Gennemark   +7 more
openaire   +3 more sources

Subunit Stoichiometry of a Heteromultimeric G protein-coupled Inward-rectifier K^+ Channel [PDF]

, 1996
We investigated the stoichiometry of the heteromultimeric G protein-coupled inward-recitfier K^+ channel (GIRK) formed from GIRK1 and GIRK4 subunits. Multimeric GIRK constructs with several concatenated channel subunits were expressed in Xenopus oocytes.
Dougherty, Dennis A., Lester, Henry A., Silverman, Scott K.   +2 more
core  

Biochemical Properties of a Decoy Oligodeoxynucleotide Inhibitor of STAT3 Transcription Factor. [PDF]

, 2018
Cyclic STAT3 decoy (CS3D) is a second-generation, double-stranded oligodeoxynucleotide (ODN) that mimics a genomic response element for signal transducer and activator of transcription 3 (STAT3), an oncogenic transcription factor.
Grandis, Jennifer R, Ha, Patrick K, Johnson, Daniel E, Lee, David S, Lee, David S, O'Keefe, Rachel A   +5 more
core   +2 more sources