Results 11 to 20 of about 47,774 (288)

Gene and Antisense Delivery in Alcoholism Research [PDF]

open access: yesAlcoholism: Clinical and Experimental Research, 2002
This article represents the proceedings of a symposium at the 2001 annual meeting of the Research Society on Alcoholism in Montreal, Canada. Drs. Yedy Israel and Fulton Crews were organizers and co‐chairpersons. The presentations were (1) Introduction to the symposium, by Yedy Israel; (2) Gene delivery to the brain, by Fulton T. Crews; (3) Gene therapy
Israel Jacard, Yedy   +12 more
openaire   +4 more sources

Targeted delivery of antisense oligonucleotides by molecular conjugates [PDF]

open access: yesSomatic Cell and Molecular Genetics, 1992
Antisense oligonucleotides efficiently inhibit gene expression in vitro; however, the successful therapeutic application of this technology in vivo will require the development of improved delivery systems. In this report we describe a technique that efficiently delivers antisense oligonucleotides into cells using molecular conjugates.
Bunnell, Bruce A.   +2 more
openaire   +4 more sources

Cationic polyelectrolyte-mediated delivery of antisense morpholino oligonucleotides for exon-skipping in vitro and in mdx mice

open access: yesInternational Journal of Nanomedicine, 2015
Mingxing Wang, Bo Wu, Jason D Tucker, Peijuan Lu, Qilong Lu Department of Neurology, McColl-Lockwood Laboratory for Muscular Dystrophy Research, Cannon Research Center, Carolinas Medical Center, Charlotte, NC, USA Abstract: In this study, we ...
Wang M, Wu B, Tucker JD, Lu P, Lu Q
doaj   +1 more source

Bolaamphiphile-based nanocomplex delivery of phosphorothioate gapmer antisense oligonucleotides as a treatment for Clostridium difficile

open access: yesInternational Journal of Nanomedicine, 2016
John P Hegarty,1 Jacek Krzeminski,2 Arun K Sharma,2 Diana Guzman-Villanueva,3 Volkmar Weissig,3 David B Stewart Sr1 1Deparment of Surgery, Pennsylvania State University College of Medicine Hershey, PA, USA; 2Department of Pharmacology, Penn State ...
Hegarty JP   +5 more
doaj   +1 more source

Antisense RNA Design, Delivery, and Analysis [PDF]

open access: yes, 2022
This open access volume gathers a variety of models, delivery systems, and approaches that can be used to assess RNA technology for exploiting antisense as a therapeutic intervention.
core   +5 more sources

Self-transfecting GMO-PMO chimera targeting Nanog enable gene silencing in vitro and suppresses tumor growth in 4T1 allografts in mouse

open access: yesMolecular Therapy: Nucleic Acids, 2023
Phosphorodiamidate morpholino oligonucleotide (PMO)-based antisense reagents cannot enter cells without the help of a delivery technique, which limits their clinical applications. To overcome this problem, self-transfecting guanidinium-linked morpholino (
Ujjal Das   +8 more
doaj   +1 more source

Advances in antisense oligonucleotide development for target identification, validation, and as novel therapeutics [PDF]

open access: yes, 2008
Antisense oligonucleotides (As-ODNs) are single stranded, synthetically prepared strands of deoxynucleotide sequences, usually 18–21 nucleotides in length, complementary to the mRNA sequence of the target gene.
Alirio J. Melendez   +3 more
core   +1 more source

Duchenne Muscular Dystrophy: From Diagnosis to Therapy

open access: yesMolecules, 2015
Duchenne muscular dystrophy (DMD) is an X-linked inherited neuromuscular disorder due to mutations in the dystrophin gene. It is characterized by progressive muscle weakness and wasting due to the absence of dystrophin protein that causes degeneration of
Maria Sofia Falzarano   +3 more
doaj   +1 more source

Polymersome delivery of siRNA and antisense oligonucleotides [PDF]

open access: yesJournal of Controlled Release, 2009
siRNA and antisense oligonucleotides, AON, have similar size and negative charge and are often packaged for in vitro delivery with cationic lipids or polymers-but exposed positive charge is problematic in vivo. Here we demonstrate loading and functional delivery of RNAi and AON with non-ionic, nano-transforming polymersomes.
Younghoon, Kim   +11 more
openaire   +2 more sources

Poly(ester amine) Composed of Polyethylenimine and Pluronic Enhance Delivery of Antisense Oligonucleotides In Vitro and in Dystrophic mdx Mice

open access: yesMolecular Therapy: Nucleic Acids, 2016
A series of poly(esteramine)s (PEAs) constructed from low molecular weight polyethyleneimine (LPEI) and Pluronic were evaluated for the delivery of antisense oligonuclotides (AOs), 2′-O-methyl phosphorothioate RNA (2′-OMePS) and phosphorodiamidate ...
Mingxing Wang   +5 more
doaj   +1 more source

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