Suchergebnisse - "Oligonucleotides"

Improved targeted delivery of antisense oligonucleotide with an antibody mask.

Autoren: Hsia HE Zanini C Simonneau C et al.

Quelle: Nucleic acids research [Nucleic Acids Res] 2025 Oct 28; Vol. 53 (20).

Publikationsart: Journal Article

Info zur Zeitschrift: Publisher: Oxford University Press Country of Publication: England NLM ID: 0411011 Publication Model: Print Cited Medium: Internet ISSN: 1362-4962 (Electronic) Linking ISSN: 03051048 NLM ISO Abbreviation: Nucleic Acids Res Subsets: MEDLINE

MeSH-Schlagworte: Oligonucleotides, Antisense*/chemistry , Oligonucleotides, Antisense*/administration & dosage , Oligonucleotides, Antisense*/genetics , Receptors, Transferrin*/immunology , Receptors, Transferrin*/metabolism , Immunoconjugates*/chemistry , Antibodies*/chemistry, Humans ; Blood-Brain Barrier/metabolism ; Oligonucleotides/chemistry ; Animals ; Transcytosis ; Mice ; Click Chemistry ; Phosphorothioate Oligonucleotides/chemistry

Pharmacokinetics of therapies approved for spinal muscular atrophy: A narrative review of current evidence.

Autoren: Sel EK Gkrinia EMM Roncato R et al.

Körperschaften: Sel EK Gkrinia EMM Roncato R et al.

Quelle: The Journal of international medical research [J Int Med Res] 2025 Nov; Vol. 53 (11), pp. 3000605251397777. Date of Electronic Publication: 2025 Nov 26.

Publikationsart: Journal Article; Review

Info zur Zeitschrift: Publisher: Sage Publications Country of Publication: England NLM ID: 0346411 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1473-2300 (Electronic) Linking ISSN: 03000605 NLM ISO Abbreviation: J Int Med Res Subsets: MEDLINE

MeSH-Schlagworte: Muscular Atrophy, Spinal*/drug therapy , Muscular Atrophy, Spinal*/genetics , Muscular Atrophy, Spinal*/therapy , Muscular Atrophy, Spinal*/pathology , Muscular Atrophy, Spinal*/metabolism , Oligonucleotides*/pharmacokinetics , Oligonucleotides*/therapeutic use , Oligonucleotides*/administration & dosage , Genetic Therapy*/methods , Biological Products*/pharmacokinetics , Biological Products*/therapeutic use, Humans ; Oligonucleotides, Antisense/pharmacokinetics ; Oligonucleotides, Antisense/therapeutic use ; Survival of Motor Neuron 1 Protein/genetics ; Motor Neurons/metabolism ; Motor Neurons/pathology ; Motor Neurons/drug effects ; Azo Compounds ; Pyrimidines ; Recombinant Fusion Proteins

Antibody-oligonucleotide conjugates in cancer therapy: Potential and Promise.

Autoren: Meng Q Yang M Xing F et al.

Quelle: Critical reviews in oncology/hematology [Crit Rev Oncol Hematol] 2025 Nov; Vol. 215, pp. 104858. Date of Electronic Publication: 2025 Jul 18.

Publikationsart: Journal Article; Review

Info zur Zeitschrift: Publisher: Elsevier Scientific Publishers Country of Publication: Netherlands NLM ID: 8916049 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1879-0461 (Electronic) Linking ISSN: 10408428 NLM ISO Abbreviation: Crit Rev Oncol Hematol Subsets: MEDLINE

MeSH-Schlagworte: Neoplasms*/drug therapy , Neoplasms*/therapy , Neoplasms*/genetics , Immunoconjugates*/therapeutic use , Immunoconjugates*/chemistry , Immunoconjugates*/pharmacology , Oligonucleotides*/therapeutic use , Oligonucleotides*/chemistry, Humans ; Animals ; Oligonucleotides, Antisense/therapeutic use

Pharmacokinetics and Safety of Pelacarsen, a GalNAc 3 -Conjugated Antisense Oligonucleotide Targeting Apo(a), in Participants With Mild Hepatic Impairment.

Autoren: Yan JH Taylor AJ Clough T et al.

Quelle: Clinical and translational science [Clin Transl Sci] 2025 Oct; Vol. 18 (10), pp. e70344.

Publikationsart: Journal Article; Clinical Trial, Phase I

Info zur Zeitschrift: Publisher: WileyBlackwell Pub Country of Publication: United States NLM ID: 101474067 Publication Model: Print Cited Medium: Internet ISSN: 1752-8062 (Electronic) Linking ISSN: 17528054 NLM ISO Abbreviation: Clin Transl Sci Subsets: MEDLINE

MeSH-Schlagworte: Acetylgalactosamine* , Oligonucleotides, Antisense*/pharmacology, Adolescent ; Adult ; Aged ; Female ; Humans ; Male ; Middle Aged ; Young Adult ; Area Under Curve ; Injections, Subcutaneous ; Severity of Illness Index ; Oligonucleotides

Donidalorsen Treatment of Hereditary Angioedema in Patients Previously on Long-Term Prophylaxis.

Autoren: Riedl MA Bernstein JA Jacobs JS et al.

Quelle: The journal of allergy and clinical immunology. In practice [J Allergy Clin Immunol Pract] 2025 Sep; Vol. 13 (9), pp. 2381-2389.e3. Date of Electronic Publication: 2025 Jul 17.

Publikationsart: Journal Article; Clinical Trial, Phase III; Multicenter Study

Info zur Zeitschrift: Publisher: Elsevier Inc Country of Publication: United States NLM ID: 101597220 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2213-2201 (Electronic) NLM ISO Abbreviation: J Allergy Clin Immunol Pract Subsets: MEDLINE

MeSH-Schlagworte: Angioedemas, Hereditary*/drug therapy , Oligonucleotides, Antisense*/therapeutic use , Oligonucleotides, Antisense*/adverse effects, Humans ; Male ; Female ; Middle Aged ; Adult ; Quality of Life ; Aged ; Treatment Outcome ; Complement C1 Inhibitor Protein/therapeutic use ; Antibodies, Monoclonal, Humanized/therapeutic use ; Young Adult ; Oligonucleotides

ApoC-III as Therapeutic Target: Is it Primetime for Clinical Use?

Autoren: Biolo M Galimberti F Portinari C et al.

Quelle: Current atherosclerosis reports [Curr Atheroscler Rep] 2025 Jul 18; Vol. 27 (1), pp. 72. Date of Electronic Publication: 2025 Jul 18.

Publikationsart: Journal Article; Review

Info zur Zeitschrift: Publisher: Springer Country of Publication: United States NLM ID: 100897685 Publication Model: Electronic Cited Medium: Internet ISSN: 1534-6242 (Electronic) Linking ISSN: 15233804 NLM ISO Abbreviation: Curr Atheroscler Rep Subsets: MEDLINE

MeSH-Schlagworte: Apolipoprotein C-III*/antagonists & inhibitors , Apolipoprotein C-III*/metabolism , Hypertriglyceridemia*/drug therapy , Oligonucleotides, Antisense*/therapeutic use, Humans ; RNA, Small Interfering/therapeutic use ; Oligonucleotides/therapeutic use ; Pancreatitis/prevention & control

Effect of olezarsen on lipoprotein-associated ApoC-III levels in patients with familial chylomicronemia syndrome.

Autoren: Yang X Alexander VJ Xia S et al.

Quelle: Atherosclerosis [Atherosclerosis] 2025 Sep; Vol. 408, pp. 120462. Date of Electronic Publication: 2025 Jul 21.

Publikationsart: Clinical Trial, Phase III; Journal Article; Randomized Controlled Trial; Research Support, Non-U.S. Gov't

Info zur Zeitschrift: Publisher: Elsevier Country of Publication: Ireland NLM ID: 0242543 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1879-1484 (Electronic) Linking ISSN: 00219150 NLM ISO Abbreviation: Atherosclerosis Subsets: MEDLINE

MeSH-Schlagworte: Apolipoprotein C-III*/blood , Hyperlipoproteinemia Type I*/drug therapy , Hyperlipoproteinemia Type I*/blood , Hyperlipoproteinemia Type I*/genetics , Oligonucleotides, Antisense*/therapeutic use, Adult ; Female ; Humans ; Male ; Middle Aged ; Young Adult ; Apolipoprotein A-I/blood ; Apolipoprotein B-100/blood ; Apolipoprotein B-48/blood ; Double-Blind Method ; Lipoprotein(a)/blood ; Treatment Outcome ; Triglycerides/blood ; Oligonucleotides

SCR Disease Name: Familial hyperchylomicronemia syndrome

ASOptimizer: optimizing chemical diversity of antisense oligonucleotides through deep learning.

Autoren: Kang S Lee D Hwang G et al.

Quelle: Nucleic acids research [Nucleic Acids Res] 2025 Jul 07; Vol. 53 (W1), pp. W39-W44.

Publikationsart: Journal Article

Info zur Zeitschrift: Publisher: Oxford University Press Country of Publication: England NLM ID: 0411011 Publication Model: Print Cited Medium: Internet ISSN: 1362-4962 (Electronic) Linking ISSN: 03051048 NLM ISO Abbreviation: Nucleic Acids Res Subsets: MEDLINE

MeSH-Schlagworte: Deep Learning* , Oligonucleotides, Antisense*, Humans ; Oligonucleotides/chemistry ; Computer Systems ; Software

Clinical applications of oligonucleotides for cancer therapy.

Autoren: DeFranciscis V Amabile G Kortylewski M

Quelle: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2025 Jun 04; Vol. 33 (6), pp. 2705-2718. Date of Electronic Publication: 2025 Mar 05.

Publikationsart: Journal Article; Review

Info zur Zeitschrift: Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE

MeSH-Schlagworte: Neoplasms*/therapy , Neoplasms*/genetics , Oligonucleotides*/therapeutic use , Oligonucleotides*/genetics , Genetic Therapy*/methods, Humans ; Immunotherapy/methods ; Clinical Trials as Topic ; Animals ; Oligonucleotides, Antisense/therapeutic use ; Drug Delivery Systems

Hepatotoxicity Reduction Profiles of Antisense Oligonucleotides Containing Amido-Bridged Nucleic Acid and 2'-O,4'-C-Spirocyclopropylene Bridged Nucleic Acid.

Autoren: Kawanobe T Asano S Kandori H et al.

Quelle: Nucleic acid therapeutics [Nucleic Acid Ther] 2025 Jun; Vol. 35 (3), pp. 114-124. Date of Electronic Publication: 2025 Mar 26.

Publikationsart: Journal Article

Info zur Zeitschrift: Publisher: Mary Ann Liebert, Inc Country of Publication: United States NLM ID: 101562758 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2159-3345 (Electronic) Linking ISSN: 21593337 NLM ISO Abbreviation: Nucleic Acid Ther Subsets: MEDLINE

MeSH-Schlagworte: Oligonucleotides, Antisense*/pharmacology , Oligonucleotides, Antisense*/chemistry , Oligonucleotides, Antisense*/genetics , Liver*/drug effects , Liver*/pathology , Liver*/metabolism , Nucleic Acids*/chemistry , Spiro Compounds*/chemistry, Animals ; Mice ; Male ; Oligonucleotides

Imetelstat in myeloid malignancies: current data and future directions.

Autoren: Bidikian A Bewersdorf JP Kewan T et al.

Quelle: Expert review of anticancer therapy [Expert Rev Anticancer Ther] 2025 May; Vol. 25 (5), pp. 517-528. Date of Electronic Publication: 2025 Mar 28.

Publikationsart: Journal Article; Review

Info zur Zeitschrift: Publisher: Taylor & Francis Country of Publication: England NLM ID: 101123358 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1744-8328 (Electronic) Linking ISSN: 14737140 NLM ISO Abbreviation: Expert Rev Anticancer Ther Subsets: MEDLINE

MeSH-Schlagworte: Myelodysplastic Syndromes*/drug therapy , Myelodysplastic Syndromes*/pathology , Telomerase*/antagonists & inhibitors , Oligonucleotides, Antisense*/pharmacology , Oligonucleotides, Antisense*/administration & dosage , Oligonucleotides, Antisense*/adverse effects, Humans ; Animals ; Oligonucleotides

Novel Therapeutics for Familial Chylomicronemia Syndrome.

Autoren: Izar MC Fonseca FAH

Quelle: Current atherosclerosis reports [Curr Atheroscler Rep] 2025 Apr 21; Vol. 27 (1), pp. 51. Date of Electronic Publication: 2025 Apr 21.

Publikationsart: Journal Article; Review

Info zur Zeitschrift: Publisher: Springer Country of Publication: United States NLM ID: 100897685 Publication Model: Electronic Cited Medium: Internet ISSN: 1534-6242 (Electronic) Linking ISSN: 15233804 NLM ISO Abbreviation: Curr Atheroscler Rep Subsets: MEDLINE

MeSH-Schlagworte: Hyperlipoproteinemia Type I*/therapy , Hyperlipoproteinemia Type I*/genetics , Hyperlipoproteinemia Type I*/drug therapy , Oligonucleotides, Antisense*/therapeutic use , Apolipoprotein C-III*/genetics , Apolipoprotein C-III*/antagonists & inhibitors, Humans ; Angiopoietin-Like Protein 3 ; RNA, Small Interfering/therapeutic use ; Angiopoietin-like Proteins ; Genetic Therapy/methods ; Oligonucleotides

SCR Disease Name: Familial hyperchylomicronemia syndrome

Familial chylomicronemia syndrome and treatments to target hepatic APOC3 mRNA.

Autoren: Brinton EA Eckel RH Gaudet D et al.

Quelle: Atherosclerosis [Atherosclerosis] 2025 Apr; Vol. 403, pp. 119114. Date of Electronic Publication: 2025 Jan 29.

Publikationsart: Journal Article; Review; Research Support, Non-U.S. Gov't

Info zur Zeitschrift: Publisher: Elsevier Country of Publication: Ireland NLM ID: 0242543 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1879-1484 (Electronic) Linking ISSN: 00219150 NLM ISO Abbreviation: Atherosclerosis Subsets: MEDLINE

MeSH-Schlagworte: Apolipoprotein C-III*/genetics , Apolipoprotein C-III*/metabolism , Hyperlipoproteinemia Type I*/genetics , Hyperlipoproteinemia Type I*/therapy , Hyperlipoproteinemia Type I*/blood , Hyperlipoproteinemia Type I*/drug therapy , Oligonucleotides, Antisense*/therapeutic use , RNA, Messenger*/metabolism , RNA, Messenger*/genetics , Liver*/metabolism , Liver*/drug effects , Genetic Therapy*/methods, Humans ; RNA, Small Interfering/therapeutic use ; Triglycerides/blood ; Animals ; Oligonucleotides/therapeutic use ; Treatment Outcome

SCR Disease Name: Familial hyperchylomicronemia syndrome

Mixed-mode separation of antisense oligonucleotides using a single column with complementary anion-exchange and hydrophobic interaction chromatography approaches.

Autoren: Kazarian AA Lee JH Mitasev B et al.

Quelle: Journal of chromatography. A [J Chromatogr A] 2025 Jan 11; Vol. 1740, pp. 465581. Date of Electronic Publication: 2024 Dec 05.

Publikationsart: Journal Article

Info zur Zeitschrift: Publisher: Elsevier Country of Publication: Netherlands NLM ID: 9318488 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1873-3778 (Electronic) Linking ISSN: 00219673 NLM ISO Abbreviation: J Chromatogr A Subsets: MEDLINE

MeSH-Schlagworte: Hydrophobic and Hydrophilic Interactions* , Oligonucleotides, Antisense*/chemistry , Oligonucleotides, Antisense*/isolation & purification, Chromatography, Ion Exchange/methods ; Phosphorothioate Oligonucleotides/isolation & purification ; Phosphorothioate Oligonucleotides/chemistry

DNA nanotechnology-based strategies for minimising hybridisation-dependent off-target effects in oligonucleotide therapies.

Autoren: Li X Hu H Wang H et al.

Quelle: Materials horizons [Mater Horiz] 2025 Mar 04; Vol. 12 (5), pp. 1388-1412. Date of Electronic Publication: 2025 Mar 04.

Publikationsart: Journal Article; Review

Info zur Zeitschrift: Publisher: Royal Society of Chemistry Country of Publication: England NLM ID: 101623537 Publication Model: Electronic Cited Medium: Internet ISSN: 2051-6355 (Electronic) Linking ISSN: 20516347 NLM ISO Abbreviation: Mater Horiz Subsets: MEDLINE

MeSH-Schlagworte: Oligonucleotides*/therapeutic use , Oligonucleotides*/chemistry , Nanotechnology*/methods , Genetic Therapy*/methods , DNA*/chemistry, Humans ; Animals ; Oligonucleotides, Antisense/therapeutic use ; Oligonucleotides, Antisense/chemistry ; Nucleic Acid Hybridization ; CRISPR-Cas Systems

An Improved P(V) Thio-Oligonucleotide Synthesis Platform.

Autoren: Nassir M Gherardi L Redman RL et al.

Quelle: Organic letters [Org Lett] 2025 Jan 10; Vol. 27 (1), pp. 97-102. Date of Electronic Publication: 2024 Dec 19.

Publikationsart: Journal Article

Info zur Zeitschrift: Publisher: American Chemical Society Country of Publication: United States NLM ID: 100890393 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1523-7052 (Electronic) Linking ISSN: 15237052 NLM ISO Abbreviation: Org Lett Subsets: MEDLINE

MeSH-Schlagworte: Oligonucleotides*/chemistry , Oligonucleotides*/chemical synthesis, Molecular Structure ; Phosphorothioate Oligonucleotides/chemistry ; Phosphorothioate Oligonucleotides/chemical synthesis ; Phosphorus/chemistry ; Stereoisomerism ; Sulfur/chemistry ; Bromine/chemistry

Safety and efficacy of antisense oligonucleotides on triglyceride, apolipoprotein C-III, and other lipid parameters levels in hypertriglyceridemia; a network meta-analysis of randomized controlled trials.

Autoren: Mahmoud A Abdelsayed K Mohamed AA et al.

Quelle: Lipids in health and disease [Lipids Health Dis] 2025 Mar 22; Vol. 24 (1), pp. 109. Date of Electronic Publication: 2025 Mar 22.

Publikationsart: Journal Article; Network Meta-Analysis

Info zur Zeitschrift: Publisher: BioMed Central Country of Publication: England NLM ID: 101147696 Publication Model: Electronic Cited Medium: Internet ISSN: 1476-511X (Electronic) Linking ISSN: 1476511X NLM ISO Abbreviation: Lipids Health Dis Subsets: MEDLINE

MeSH-Schlagworte: Apolipoprotein C-III*/blood , Apolipoprotein C-III*/antagonists & inhibitors , Hypertriglyceridemia*/drug therapy , Hypertriglyceridemia*/blood , Triglycerides*/blood , Oligonucleotides, Antisense*/therapeutic use , Oligonucleotides, Antisense*/adverse effects, Humans ; Randomized Controlled Trials as Topic ; Oligonucleotides

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

Autoren: Tripathy RK Pande AH

Quelle: Pharmaceutical research [Pharm Res] 2025 Feb; Vol. 42 (2), pp. 219-236. Date of Electronic Publication: 2025 Feb 15.

Publikationsart: Journal Article; Review

Info zur Zeitschrift: Publisher: Kluwer Academic/Plenum Publishers Country of Publication: United States NLM ID: 8406521 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1573-904X (Electronic) Linking ISSN: 07248741 NLM ISO Abbreviation: Pharm Res Subsets: MEDLINE

MeSH-Schlagworte: Oligonucleotides*/chemistry , Oligonucleotides*/administration & dosage , Oligonucleotides*/therapeutic use , Single-Domain Antibodies*/chemistry , Immunoconjugates*/chemistry, Humans ; Animals ; Drug Delivery Systems/methods ; Oligonucleotides, Antisense

Progress and Future Directions in Exon Skipping and Inclusion Therapies: The Landscape of Oligonucleotide-Based Genetic Medicine.

Autoren: Anwar S Yokota T

Quelle: Methods in molecular biology (Clifton, N.J.) [Methods Mol Biol] 2025; Vol. 2964, pp. 3-21.

Publikationsart: Journal Article; Review

Info zur Zeitschrift: Publisher: Humana Press Country of Publication: United States NLM ID: 9214969 Publication Model: Print Cited Medium: Internet ISSN: 1940-6029 (Electronic) Linking ISSN: 10643745 NLM ISO Abbreviation: Methods Mol Biol Subsets: MEDLINE

MeSH-Schlagworte: Exons*/genetics , Genetic Therapy*/methods , Genetic Therapy*/trends , Oligonucleotides*/therapeutic use , Oligonucleotides*/genetics, Humans ; Precision Medicine/methods ; Muscular Dystrophy, Duchenne/genetics ; Muscular Dystrophy, Duchenne/therapy ; Muscular Atrophy, Spinal/genetics ; Muscular Atrophy, Spinal/therapy ; RNA Splicing ; Oligonucleotides, Antisense/therapeutic use

Quantifying and mitigating motor phenotypes induced by antisense oligonucleotides in the central nervous system.

Autoren: Moazami MP Rembetsy-Brown JM Sarli SL et al.

Quelle: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2024 Dec 04; Vol. 32 (12), pp. 4401-4417. Date of Electronic Publication: 2024 Oct 28.

Publikationsart: Journal Article

Info zur Zeitschrift: Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE

MeSH-Schlagworte: Oligonucleotides, Antisense*/pharmacology , Oligonucleotides, Antisense*/administration & dosage , Phenotype*, Animals ; Mice ; Central Nervous System/drug effects ; Central Nervous System/metabolism ; Male ; Humans ; Phosphorothioate Oligonucleotides/pharmacology ; Phosphorothioate Oligonucleotides/chemistry ; Motor Activity/drug effects