Developing aptamers into therapeutics

White, Rebekah R.; Sullenger, Bruce A.; Rusconi, Christopher P.

doi:10.1172/jci11325

Cited by 268 publications

(201 citation statements)

References 35 publications

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“…In general, DNA oligonucleotides are susceptible to nuclease-mediated degradation in vivo with a half-life varying from 30 min to 60 min [29]. In order to avoid 5' and 3' exonuclease activity, aptamers can be chemically modified at their terminal ends.…”

Section: Plasma Stability Of Aptamer Hea2_3mentioning

confidence: 99%

Biodistribution of Novel 68Ga-Radiolabelled HER2 Aptamers in Mice

Gijs¹,

Becker

Plenevaux

et al. 2016

J Nucl Med Radiat Ther

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Section: Plasma Stability Of Aptamer Hea2_3mentioning

confidence: 99%

Biodistribution of Novel 68Ga-Radiolabelled HER2 Aptamers in Mice

Gijs¹,

Becker

Plenevaux

et al. 2016

J Nucl Med Radiat Ther

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“…This technology has been explored in clinical trials of anticancer and antiviral therapeutics. 51 Moreover, opportunities also exist for therapeutic aptamers as antithrombotic and hemostatic agents. Aptamers that target tissue factor pathway inhibitor have shown similar hemostatic effects in vitro as NASP.…”

Section: Alternative Hemostatic Agentsmentioning

confidence: 99%

Hemophilia: New Protein Therapeutics

Pipe

2010

Hematology

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Therapeutic advances for patients with hemophilia have resulted in reduced mortality, improved joint outcomes, safety from blood-transmitted pathogens, improved quality of life, and a normalized life span in the developed world. The production of recombinant coagulation factors has increased the worldwide capacity for replacement therapy and facilitated aggressive prophylactic therapy. However, this has come at significant cost, and barriers remain to broad application of prophylaxis. Recombinant DNA technology remains a promising platform to develop novel hemophilia therapeutics with improved functional properties to try to overcome some of these remaining barriers. Bioengineering strategies have produced novel therapeutics with increased production efficiency, increased potency and resistance to inactivation, prolonged plasma half-lives, and reduced immunogenicity. Alternative nonbiologic therapies may lead to new treatment paradigms. The current pipeline of new technologies and products is promising and growing with several agents already advancing from preclinical to clinical trials.

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“…54 The term aptamer (from the Latin aptus [bound]) was coined by Ellington and Szostak after their pioneering work originally published in Nature. 55 On the basis of iterative selection techniques, aptamers that bind to essentially any protein or small molecule can be generated.…”

Section: Aptamers or Protein-binding Oligonucleotidesmentioning

confidence: 99%