Results 171 to 180 of about 124,170 (327)

Scavenger receptor-mediated delivery of antisense mini-exon phosphorothioate oligonucleotide to Leishmania-infected macrophages [PDF]

, 1997
Gautam Chaudhuri
openalex   +1 more source

Upregulation of Functional Kv11.1 Isoform Expression by Inhibition of Intronic Polyadenylation with Antisense Morpholino Oligonucleotides

, 2012
The KCNH2 gene encodes the Kv11.1 potassium channel that conducts the rapidly activating delayed rectifier current in the heart. KCNH2 pre-mRNA undergoes alternative processing; intron 9 splicing leads to the formation of a functional, full-length Kv11 ...
Gong, Qiuming, Stump, Matthew R., Zhou, Zhengfeng   +2 more
core  

State‐of‐the‐Art on Model‐Informed Drug Development Approaches for Pediatric Rare Diseases

CPT: Pharmacometrics &Systems Pharmacology, EarlyView.
ABSTRACT Pediatric rare diseases present unique challenges for drug development due to small patient populations, ethical constraints on clinical trial design, and limited prospectively defined natural history data. Model‐Informed Drug Development (MIDD) has emerged as a powerful paradigm to address these challenges by leveraging quantitative methods ...
Rajesh Krishna, Amitava Mitra, Matthew L. Zierhut, Lilly East, Chandra Durairaj   +4 more
wiley   +1 more source

RNA Therapeutics: Focus on Antisense Oligonucleotides in the Nervous System. [PDF]

Biomol Ther (Seoul)
Ertural B, Çiçek BN, Kurnaz IA.
europepmc   +1 more source

Inhibition of kinesin synthesis and rapid anterograde axonal transport in vivo by an antisense oligonucleotide

, 1993
Anil Amaratunga, Peter J. Morin, K S Kosik, Richard E. Fine   +3 more
openalex   +1 more source

Sustained reduction of neointima with c-myc antisense oligonucleotides in saphenous vein grafts [PDF]

, 1998
John D. Mannion, Michael L. Ormont, Michael G. Magno, James E. O’Brien, Yi Shi, Andrew Zalewski   +5 more
openalex   +1 more source

Transforming Pediatric Rare Disease Drug Development: Enhancing Clinical Trials and Regulatory Evidence With Virtual Patients

CPT: Pharmacometrics &Systems Pharmacology, EarlyView.
ABSTRACT Drug development in pediatric rare diseases is complicated by practical and ethical constraints on clinical trial design, stemming from small, highly heterogeneous, and vulnerable patient populations. Virtual patients (VPs) created with machine‐learning (ML), mechanistically driven computational approaches, or hybrids thereof, have the ...
Fianne Sips, Marco Virgolin, Giuseppe Pasculli, Federico Reali, Alessio Paris, Annette Janus, Yann Godfrin, Daniel Röshammar, Luca Marchetti, Jane Knöchel   +9 more
wiley   +1 more source

Programmable Single-Stranded Circular Antisense Oligonucleotides for Multitarget Gene Therapy. [PDF]

JACS Au
Pan Y, Li X, Zhu C, Wang M, Wu Y, Xu R, Wu J, Chen R, Jiang Y, Tian B, Dong Y, Liu D.   +11 more
europepmc   +1 more source

Modeling and Simulation Identifies Endocytosis Uptake Rate and Fraction Unbound as Important Predictors of Oligonucleotide Pharmacokinetics

CPT: Pharmacometrics &Systems Pharmacology, EarlyView.
ABSTRACT Therapeutic oligonucleotides (TOs) represent an emerging modality, which offers a promising alternative treatment option, particularly for intracellular targets. The two types of TOs, antisense oligonucleotides (ASO) and small interfering RNAs (siRNAs), distribute highly into tissues, especially into the liver and the kidneys.
Felix Stader, Abdallah Derbalah, Adriana Zyla, Cong Liu, Iain Gardner, Armin Sepp   +5 more
wiley   +1 more source

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

Nucleic Acids Res
Kang S, Lee D, Hwang G, Lee K, Kang M.
europepmc   +1 more source