Results 231 to 240 of about 266,614 (380)

Application of antisense oligonucleotide drugs in amyotrophic lateral sclerosis and Huntington's disease. [PDF]

Transl Neurodegener
Ou K, Jia Q, Li D, Li S, Li XJ, Yin P.
europepmc   +1 more source

Antisense oligonucleotides to Cux-1, a Cut-related homeobox gene, cause increased apoptosis in mouse embryonic kidney cultures. [PDF]

, 1997
Susan E. Quaggin, Herman Yeger, Peter Igarashi   +2 more
openalex   +1 more source

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

AmNA-Modified Antisense Oligonucleotide Targeting MCM8 as a Cancer-Specific Chemosensitizer for Platinum Compounds. [PDF]

Cancer Sci
Uchibori Y, Suekuni M, Kokaji Y, Yoshida K, Kiyono T, Kasahara Y, Fujita M.   +6 more
europepmc   +1 more source

Oligonucleotides Tethered to a Short Polyguanylic Acid Stretch Are Targeted to Macrophages: Enhanced Antiviral Activity of a Vesicular Stomatitis Virus-Specific Antisense Oligonucleotide [PDF]

, 1999
Vikram Prasad, Shehla Hashim, Amitabha Mukhopadhyay, Sandip K. Basu, Rajendra P. Roy   +4 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

Anti-TNF-α antisense-oligonucleotide-conjugated PLG nanoparticles protect transplanted islets. [PDF]

Mol Ther Methods Clin Dev
Bealer EJ, Padgaonkar N, Crumley K, Saito E, Beekman Z, DeKorte A, Prakash TP, Revenko A, Shea LD.   +8 more
europepmc   +1 more source

Design, biochemical, biophysical and biological properties of cooperative antisense oligonucleotides [PDF]

, 1995
Ekambar R. Kandimalla, Adrienne Manning, Christopher S. Lathan, Randal A. Byrn, Sudhir Agrawal   +4 more
openalex   +1 more source

Update on Gene Therapy in the Treatment of Hereditary Hearing Loss

Sensory Neuroscience, EarlyView.
Gene therapy is a promising therapeutic approach for genetic disorders, including three major gene therapy strategies: gene replacement, gene suppression, and gene editing. After transmitting genes to the inner ear through appropriate pathways, repair the cochlea in order to achieve the goal of treating hereditary hearing loss.
Weiwei He, Shanying Han, Xinghong Liu, Xiaotong Huang, Wen Yang, Xinghua Tang, Chuan Dong, Jiangang Fan   +7 more
wiley   +1 more source

Antisense oligonucleotide treatment in a preterm infant with early-onset SCN2A developmental and epileptic encephalopathy. [PDF]

Nat Med
Wagner M, Berecki G, Fazeli W, Nussbaum C, Flemmer AW, Frizzo S, Heer F, Heinen F, Horton R, Jacotin H, Motel W, Spar B, Klein C, Siegel C, Hübener C, Stöcklein S, Paolini M, Staudt M, Tacke M, Wolff M, Petrou S, Souza M, Borggraefe I.   +22 more
europepmc   +1 more source