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ENPP1-Fc prevents mortality and vascular calcifications in rodent model of generalized arterial calcification of infancy

Generalized arterial calcification of infancy (GACI) is a terminal disease caused by the ENPP1 enzyme deficiency. Here, Albrigh et al. show that ENPP1 enzyme replacement therapy prevents the ectopic calcifications and mortality in mice with GACI, suggesting a novel treatment for vascular calcification in humans.

Ronald A. Albright
Paul Stabach
Demetrios T. Braddock
ResearchOpen Access01 Dec 2015
Nature Communications

Volume: 6, P: 1-11
Structure and dynamics of KH domains from FBP bound to single-stranded DNA

Demetrios T. Braddock
John M. Louis
G. Marius Clore
Research28 Feb 2002
Nature

Volume: 415, P: 1051-1056
Direct targeting of amplified gene loci for proapoptotic anticancer therapy

Amplified loci in cancer genomes are directly targeted with triplex-forming oligonucleotides.

Meetu Kaushik Tiwari
Daniel A. Colon-Rios
Faye A. Rogers
Research28 Oct 2021
Nature Biotechnology

Volume: 40, P: 325-334
In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery

Gene editing approaches are widely used for correcting mutations, but their application is largely limited to cells and not living animals. Here the authors show that in vivoγPNA-mediated editing of a β-globin mutation is promoted by SCF and leads to sustained normalization of blood haemoglobin levels β-thalassemic mice.

Raman Bahal
Nicole Ali McNeer
Peter M. Glazer
ResearchOpen Access26 Oct 2016
Nature Communications

Volume: 7, P: 1-14
Metformin suppresses gluconeogenesis by inhibiting mitochondrial glycerophosphate dehydrogenase

Metformin treatment of rats at physiologically relevant doses inhibits the redox shuttle enzyme mitochondrial glycerophosphate dehydrogenase.

Anila K. Madiraju
Derek M. Erion
Gerald I. Shulman
Research21 May 2014
Nature

Volume: 510, P: 542-546
MicroRNA silencing for cancer therapy targeted to the tumour microenvironment

A novel anti-microRNA delivery platform that targets the acidic tumour microenvironment, in which a chosen anti-miRNA is coupled to a peptide that can transport the anti-miRNA across cell membranes specifically in an acidic environment.

Christopher J. Cheng
Raman Bahal
Frank J. Slack
Research17 Nov 2014
Nature

Volume: 518, P: 107-110

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ENPP1-Fc prevents mortality and vascular calcifications in rodent model of generalized arterial calcification of infancy

Structure and dynamics of KH domains from FBP bound to single-stranded DNA

Direct targeting of amplified gene loci for proapoptotic anticancer therapy

In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery

Metformin suppresses gluconeogenesis by inhibiting mitochondrial glycerophosphate dehydrogenase

MicroRNA silencing for cancer therapy targeted to the tumour microenvironment

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