Results 131 to 140 of about 270,275 (243)

A morpholino antisense oligonucleotide rescues type I and type III SMA mice

, 2012
The majority of patients affected by Spinal Muscular Atrophy (SMA)are due to homozygous deletions of Survival Motor Neuron gene 1(SMN1) which leads to the absence of functional SMN protein,and results in motor neuron degeneration and muscle weakness.A ...
Zhou, Haiyan, Janghra, Narinder, Anthony, Karen, Muntoni, Francesco, Morgan, Jennifer   +4 more
core  

Nusinersen: the antisense oligonucleotide at the forefront of spinal muscular atrophy treatment. [PDF]

RNA Biol
Singh NN, Ottesen EW, Singh RN.
europepmc   +1 more source

Sintesi e Purificazione di Oligonucleotidi e Nucleosidi 2’- funzionalizzati

, 2009
Oligonucleotides (ON) are commonly used as research reagents to modulate gene expression in cell culture and in animal models. Various chemistries of ON have been synthesized, incorporating modifications to the phosphodiester backbone, sugar or ...
Mari, Lara
core  

WDR91, an endosomal maturation protein, promotes antisense oligonucleotide activity. [PDF]

Mol Ther Nucleic Acids
Chen S, Zheng T, Li D.
europepmc   +1 more source

Inhaled Angiopoietin-Like 4 Antisense Oligonucleotide Therapy for Lung Injury and Fibrosis. [PDF]

Adv Sci (Weinh)
Fan H, Tan Y, Zhang J, Liu X, Qu J, Chua D, Cheng HS, Kim JHS, Liu YX, Qin C, Liu Y, Li D, Qiu J, Chi M, Bi M, Zhang Q, Li Y, Motamedi Joibari M, Nilsson SK, Tan NS, Fan Y, Li L.   +21 more
europepmc   +1 more source

Enhanced anti-tumor effects with microencapsulated c-myc antisense oligonucleotide.

, 1999
A phosphorothioate c-myc antisense oligonucleotide was complexed with zinc and encapsulated into injectable biodegradable microspheres. The efficacy of this novel formulation was compared with intravenous administration of the unencapsulated drug in ...
Lee R, Leonetti C, Geiser T, CALABRETTA, Bruno, Zupi G, Putney SD, Brown J, Cucco C, Skorski T, Zon G.   +9 more
core  

Targeted delivery of an antisense oligonucleotide in the retina: Uptake, distribution, stability, and effect

, 1996
In this article, we describe the preliminary results of the development of an animal model that will enable us to study the effect of photoreceptor-derived debris accumulation on the normal function of the retina in vivo.
Rakoczy, P.E., Lai, M.C., Watson, M., Constable, I., Seydel, U.   +4 more
core  

Antisense Oligonucleotide Therapeutics Targeting Age-Related Diseases. [PDF]

BioDrugs
Lee HG, Kim J, Jang CY, Shin M.
europepmc   +1 more source

Antisense oligonucleotide-mediated knockdown therapy in two infants with severe KCNT1 epileptic encephalopathy. [PDF]

Nat Med
Nakayama T, El Achkar CM, Burbano LE, Quraishi IH, Wu J, Li M, Asami Y, Golinski SR, Sherrill E, Goodlett BD, de Gusmao CM, Friedman DA, Lentucci C, Suslovitch V, Riccardi O, Faour KNW, Kuniholm A, Soucy Verran A, Coffman S, Ahtam B, Zhao B, Chin DH, DiDonato RL, Hu CA, Lopez E, Hills S, Maljevic S, Tran H, Bush LW, Grant PE, Madsen JR, Smith RS, Kaczmarek LK, Berde CB, Yu TW.   +34 more
europepmc   +1 more source

An LNA gapmer antisense oligonucleotide inhibitor of human NNMT. [PDF]

Mol Ther Nucleic Acids
Chen S, Poudel BH, Veedu RN.
europepmc   +1 more source