Search Results - "Antisense oligonucleotide"

A Bioanalytical Liquid Chromatography Tandem Mass Spectrometry Approach for the Quantification of a Novel Antisense Oligonucleotide Designed for Parkinson’s Disease: A Rat Brain Biodistribution Study

Palaiologou, Anastasia^aff1, Naki, Marianna^{aff2, aff3}, Pantazopoulou, Marina^aff4, Kattan, Fedon-Giasin^aff2, Stefanis, Leonidas^aff4, Doxakis, Epaminondas^aff2, Tamvakopoulos, Constantin^aff1

In Vivo Evaluation of Exon 51 Skipping in hDMD/Dmd- null Mice

Sheri, Narin^Aff3, Yokota, Toshifumi^Aff3

Asakura, Atsushi^Aff2, Walker, John M.^Aff1

Estimating meaningful differences in measures of neuropathic impairment, health-related quality of life, and nutritional status in patients with hereditary transthyretin amyloidosis

Folkvaljon, Folke, Gertz, Morie, Gillmore, Julian D., Khella, Sami, Masri, Ahmad, Maurer, Mathew S., Cruz, Márcia Waddington, Wixner, Jonas, Chen, Jersey, Reicher, Barry, Kwoh, Jesse, Yarlas, Aaron, Berk, John L.

electronic

Exon-Skipping for a Pathogenic COL6A1 Variant in Ullrich Congenital Muscular Dystrophy

Aguti, Sara^Aff4, Guirguis, Fady^{Aff4, Aff5}, Bönnemann, Carsten^Aff5, Muntoni, Francesco^{Aff4, Aff6}, Bolduc, Véronique^Aff5, Zhou, Haiyan^{Aff6, Aff7}

Maruyama, Rika^Aff2, Yokota, Toshifumi^Aff3, Walker, John M.^Aff1

Delivery of Antisense Oligonucleotides to the Mouse Retina

Garanto, Alejandro^{Aff4, Aff5}

Arechavala-Gomeza, Virginia^Aff2, Garanto, Alejandro^Aff3, Walker, John M.^Aff1

Tofersen for SOD1 amyotrophic lateral sclerosis: a systematic review and meta-analysis

Hamad, Abdullah Ashraf^{Aff1, IDs10072025079942_cor1}, Alkhawaldeh, Ibraheem M.^{Aff2, Aff3}, Nashwan, Abdulqadir J.^{Aff4, Aff5}, Meshref, Mostafa, Imam, Yahia^{Aff7, Aff8, Aff9}

Antisense Oligonucleotide Embedded Context Responsive Nanoparticles Derived from Synthetic Ionizable Lipids for lncRNA Targeted Therapy of Breast Cancer

Sarkar, Sourav^aff1, Moitra, Parikshit^aff2, Bera, Sayan^aff1, Bhattacharya, Santanu^{aff1, aff3, aff4, aff5}

Imaging of CPP Delivery Mechanisms of Oligonucleotides

El Boujnouni, Najoua^{Aff3, Aff4}, van Asbeck, Alexander H.^Aff3, Dieker, Jürgen^Aff3, Wansink, Derick G.^Aff4, Brock, Roland^{Aff3, Aff5}

Langel, Ülo^Aff2, Walker, John M.^Aff1

Efficacy and safety of olezarsen in lowering apolipoprotein C-III and triglycerides in healthy Japanese Americans.

Karwatowska-Prokopczuk, Ewa, Lesogor, Anastasia, Yan, Jing-He, Hoenlinger, Angelika, Margolskee, Alison, Li, Lu, Tsimikas, Sotirios

application/pdf

Coordination-Driven Self-Assembly of Metal Ion–Antisense Oligonucleotide Nanohybrids for Chronic Bacterial Infection Therapy

Li, Anqi^aff1, Zhang, Yan^aff1, Wan, Li^aff1, Peng, Rujue^aff1, Zhang, Xuan^aff1, Guo, Qiuyan^aff1, Xu, Shan^aff1, Qiao, Dan^aff1, Zheng, Pengwu^aff1, Li, Na^aff2, Zhu, Wufu^aff1, Pan, Qingshan^aff1

New Frontiers for Site-Directed RNA Editing: Harnessing Endogenous ADARs

Merkle, Tobias^Aff4, Stafforst, Thorsten^Aff4

Picardi, Ernesto^Aff2, Pesole, Graziano^Aff3, Walker, John M.^Aff1

Development of LNA Gapmer Oligonucleotide-Based Therapy for ALS/FTD Caused by the C9orf72 Repeat Expansion

Sathyaprakash, Chaitra^Aff4, Manzano, Raquel^Aff5, Varela, Miguel A.^Aff5, Hashimoto, Yasumasa^Aff6, Wood, Matthew J. A.^Aff5, Talbot, Kevin^Aff4, Aoki, Yoshitsugu^Aff6

Yokota, Toshifumi^Aff2, Maruyama, Rika^Aff3, Walker, John M.^Aff1

Targeting Polyadenylation for Retention of RNA at Chromatin

Ntini, Evgenia^{Aff3, Aff4}, Vang Ørom, Ulf Andersson^Aff5

Ørom, Ulf Andersson Vang^Aff2, Walker, John M.^Aff1

MST3 and MST4 : critical regulators of liver lipid partitioning and hepatocarcinogenesis : new therapeutic targets for the treatment of non-alcoholic fatty liver disease and hepatocellular carcinoma / Mara Caputo.

Caputo, Mara, 1993-

Book , 2023

Nanobody-Oligonucleotide Conjugates (NucleoBodies): The Next Frontier in Oligonucleotide Therapy

Tripathy, Rajan K., Pande, Abhay H.^{Aff1, IDs1109502503829z_cor1}

Progress and prospects in antisense oligonucleotide-mediated exon skipping therapies for Duchenne muscular dystrophy

Chwalenia, Katarzyna^{Aff1, Aff2}, Wood, Matthew J. A.^{Aff1, Aff2, Aff3}, Roberts, Thomas C.^{Aff1, Aff2, Aff3, IDs10974024096882_cor1}

Nano-Regulation of Gene Expression in Chlamydomonas reinhardtii: Harnessing AuNPs for Remotely Switchable Lipid Biosynthesis via Antisense Oligonucleotides

Rafiei, Nahid^{aff1, aff2, aff3}, Alishah Aratboni, Hossein^{aff2, aff3}, Alemzadeh, Abbas^aff1, Saavedra-Alonso, Santiago^aff3, Razi, Hooman^aff1, Morones-Ramírez, José Rubén^{aff2, aff3}

A Modular Albumin-Oligonucleotide Biomolecular Assembly for Delivery of Antisense Therapeutics

Elkhashab, Marwa^aff1, Dilek, Yeter^aff3, Foss, Morten^aff2, Creemers, Laura B.^aff3, Howard, Kenneth A.^aff1

Long-term CSF responses in adult patients with spinal muscular atrophy type 2 or 3 on treatment with nusinersen

Cebulla, Gina^{Aff1, Aff2}, Hai, Ling^{Aff1, Aff2}, Warnken, Uwe, Güngör, Cansu, Hoffmann, Dirk C.^{Aff1, Aff2}, Korporal-Kuhnke, Mirjam, Wildemann, Brigitte, Wick, Wolfgang^{Aff1, Aff2}, Kessler, Tobias^{Aff1, Aff2, IDs00415025129847_cor1}, Weiler, Markus^{Aff3, IDs00415025129847_cor2}

Pwp1 inhibition impairs the development and early lineage commitment of mouse preimplantation embryos

Takuto YAMAMOTO, Atsushi TAKASU, Yasuhiro ISUMI, Satoshi MASHIKO, Daiki SHIKATA, Shinnosuke HONDA, Naojiro MINAMI, Shuntaro IKEDA

electronic resource

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