Results 41 to 50 of about 127,002 (321)

Silencing disease genes in the laboratory and the clinic

, 2011
Synthetic nucleic acids are commonly used laboratory tools for modulating gene expression and have the potential to be widely used in the clinic. Progress towards nucleic acid drugs, however, has been slow and many challenges remain to be overcome before
Corey, David R., Watts, Jonathan K.
core   +1 more source

HLA-G: expression in human keratinocytes in vitro and in human skin in vivo [PDF]

, 1994
Classical, polymorphic major histocompatibility complex class I molecules are expressed on most nucleated cells.They present peptides at the cell surface and, thus, enable the immune system to scan peptides for their antigenicity.
Ulbrecht, M., Rehberger, B., Strobel, I., Messer, Gerald, Kind, P., Degitz, K., Bieber, T., Weiss, Elisabeth H.   +7 more
core   +2 more sources

Evidence Favoring a Positive Feedback Loop for Physiologic Auto Upregulation of hnRNP-E1 during Prolonged Folate Deficiency in Human Placental Cells [PDF]

, 2017
Background: Previously, we determined that heterogeneous nuclear ribonucleoprotein E1 (hnRNP-E1) functions as an intracellular physiologic sensor of folate deficiency.
Antony, Aśok C., Hansen, Deborah K., Jayaram, Hiremagalur N., Khan, Rehana A., Kusumanchi, Praveen, Stabler, Sally P., Tang, Ying-Sheng, Xiao, Suhong   +7 more
core   +1 more source

Transport Oligonucleotides—A Novel System for Intracellular Delivery of Antisense Therapeutics

Molecules, 2020
Biological activity of antisense oligonucleotides (asON), especially those with a neutral backbone, is often attenuated by poor cellular accumulation. In the present proof-of-concept study, we propose a novel delivery system for asONs which implies the ...
Oleg V. Markov, Anton V. Filatov, Maxim S. Kupryushkin, Ivan V. Chernikov, Olga A. Patutina, Anton A. Strunov, Elena L. Chernolovskaya, Valentin V. Vlassov, Dmitrii V. Pyshnyi, Marina A. Zenkova   +9 more
doaj   +1 more source

Polymerase-endonuclease amplification reaction for large-scale enzymatic production of antisense oligonucleotide [PDF]

, 2009
Synthetic oligonucleotides are contaminated with highly homologous failure sequences. Oligonucleotide synthesis is difficult to scale up because it requires expensive equipments, hazardous chemicals, and tedious purification process.
Deming Gou, Xiaolong Wang
core   +1 more source

Micro-RNA family that modulates fibrosis and uses thereof [PDF]

, 2017
The present invention relates to the identification of a microRNA family, designated miR-29a-c, that is a key regulator of fibrosis in cardiac tissue. The inventors show that members of the miR-29 family are down-regulated in the heart tissue in response
Olson, Eric N., van Rooij, Eva
core   +1 more source

Exploratory Analysis of ELP1 Expression in Whole Blood From Patients With Familial Dysautonomia

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Background Familial dysautonomia (FD) is a hereditary neurodevelopmental disorder caused by aberrant splicing of the ELP1 gene, leading to a tissue‐specific reduction in ELP1 protein expression. Preclinical models indicate that increasing ELP1 levels can mitigate disease manifestations.
Alejandra González‐Duarte, Lucy Norcliffe‐Kaufmann, Maria Luisa Cotrina, Zenith Khan, Kaia Dalamo, Patricio Millar Vernetti, Matthew Lawless, Elisabetta Morini, Monica Salani, Marla Weetall, Jana Narasimhan, Agostino G. Rocha, Susan A. Slaugenhaupt, Horacio Kaufmann   +13 more
wiley   +1 more source

Efficient inhibition of HIV-1 expression by LNA modified antisense oligonucleotides and DNAzymes targeted to functionally selected binding sites

Retrovirology, 2007
Background A primary concern when targeting HIV-1 RNA by means of antisense related technologies is the accessibility of the targets. Using a library selection approach to define the most accessible sites for 20-mer oligonucleotides annealing within the ...
Berkhout Ben, Wengel Jesper, Haasnoot Joost, Jakobsen Martin R, Kjems Jørgen   +4 more
doaj   +1 more source

Steric antisense inhibition of AMPA receptor Q/R editing reveals tight coupling to intronic editing sites and splicing [PDF]

, 2012
Adenosine-to-Inosine (A-to-I) RNA editing is a post-transcriptional mechanism, evolved to diversify the transcriptome in metazoa. In addition to wide-spread editing in non-coding regions protein recoding by RNA editing allows for fine tuning of protein ...
Ales Balik, Altschul, Andrew C. Penn, Arzumanov, Arzumanov, Balik, Bass, Birney, Brusa, Carver, Daniel, Darty, Dominski, Egebjerg, Eisen, Enstero, Feldmeyer, Gallo, Ge, Greger, Hamada, Heasman, Higuchi, Higuchi, Hogg, Ingo H. Greger, Jepson, Jubin, Katoh, Kent, Kwak, Laurencikiene, Maas, Moulton, Peng, Reenan, Rice, Rosenthal, Rueter, Ryman, Schoft, Sommer, Stephens, Summerton, Summerton, Summerton, Tariq, Traynelis, Valente, Venkatesh, Yamashita, Yang   +51 more
core   +1 more source

Effectiveness and Safety of Nusinersen and Risdiplam in Spinal Muscular Atrophy: A Systematic Review

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Spinal Muscular Atrophy (SMA) is a rare genetic disorder marked by progressive muscle weakness and mobility loss. It has a profound physical, emotional and social impact on patients and caregivers, requiring comprehensive medical and supportive care.
Amin Mehrabian, Peter Auguste, Amy Grove, Anna Brown, Janette Parr, Mubarak Patel, Furqan Butt, Jeremiah Donoghue, Mehdi Yousefi, Jo Parsons   +9 more
wiley   +1 more source