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Scientific News

FARA funds research progress

In this section, you will find the most recent FA research publications, many of which are funded by FARA, as well as information on upcoming conferences and symposiums. You can search for articles by date using the archive box in the right hand column. To locate FARA Funded or Supported Research, click the hyperlink in the right hand column. You may also search for specific content using key words or phrases in the search button at the top right of your screen. Please be sure to visit other key research sections of our website for information on FARA’s Grant Program and the Treatment Pipeline.

Global Ataxia Advocacy Organizations Unite to Host International Ataxia Research Conference in Washington, DC

On November 14th-16th, the Friedreich’s Ataxia Research Alliance (FARA US), Ataxia UK, fara (Australia), and GoFar (Italy) will host the International Ataxia Research Conference (IARC) in Washington DC.

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Orphan Drugs In Development For The Treatment Of Friedreich's Ataxia: Focus On Omaveloxolone

Nrf2 activators such as omaveloxolone (Omav) modulate antioxidative mechanisms, and thus may be viable therapeutic agents in Friedreich's Ataxia (FRDA). This paper reviews the MOXIe trial (NCT02255435, Reata Pharmaceuticals Inc) and the use of other Nrf2 activators as a viable option in the treatment of FRDA.

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Temporal but not spatial dysmetria relates to disease severity in FA

Features of Friedreich's Ataxia (FA) include proprioceptive and cerebellar deficits leading to impaired muscle coordination and, consequently, dysmetria in force and time of movement. The aim of this study is to characterize dysmetria and its association to functional capacity. Also, the authors examine the neural mechanisms of dysmetria by quantifying the EMG burst area, duration, and time-to-peak of the agonist muscle. 27 individuals with FA and 13 healthy controls (HC) performed the modified Functional Ataxia Rating Scale (mFARS), and goal-directed movements with the ankle. Dysmetria was quantified as position and time error during dorsiflexion. FA individuals exhibited greater time but not position error than HC. Moreover, time error correlated with disease severity and was related to increased agonist EMG burst. Temporal dysmetria is associated to functional capacity, likely due to altered activation of the agonist muscle.

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Quantitative Proteomic and Network Analysis of Differentially Expressed Proteins in PBMC of Friedreich's Ataxia (FRDA) Patients

Given the ongoing drug trials in Friedreich's ataxia (FRDA), identification of reliable and easily accessible biomarkers for monitoring disease progression and therapeutic intervention is a foremost requirement. In this study, comparative proteomic profiling of PBMC proteins from FRDA patients and age- and gender-matched healthy controls was done using 2D-Differential in-Gel Electrophoresis (2D-DIGE). Protein-protein interaction (PPI) was analyzed using BioGRID and STRING pathway analysis tools. Using biological variance analysis (BVA) and LC/MS, the authors found eight differentially expressed proteins with fold change ≥1.5; p ≤ 0.05. Based on their cellular function, the identified proteins showed a strong pathological role in neuroinflammation, cardiomyopathy, compromised glucose metabolism, and iron transport, which are the major clinical manifestations of FRDA. Protein-protein network analysis of differentially expressed proteins with frataxin further supports their involvement in the pathophysiology of FRDA. Considering their crucial role in the cardiac and neurological complications, respectively, the two down-regulated proteins, actin α cardiac muscle 1 (ACTC1) and pyruvate dehydrogenase E1 component subunit β (PDHE1), are suggested as potential prognostic markers for FRDA.

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Refinement of protein Fe(II) binding characteristics utilizing a competition assay exploiting small molecule ferrous chelators

Iron is the most prevalent metal in biology. Its chemical and redox versatility allows it to direct activity of many Fe binding proteins. While iron's biological applications are diverse, challenges inherent in having Fe(II) present at high abundance means cells must ensure delivery to the correct recipient, while also ensuring its chemistry is regulated. Having a detailed understanding of the biophysical characteristics of a protein's iron binding characteristics allows us to understand general cellular metal homeostasis events. Unfortunately, most spectroscopic techniques available to measure metal binding affinity require protein be in a homogeneous state. Homogeneity creates an artificial environment when measuring metal binding since within cells numerous additional metal binding biomolecules compete with the target. Here the authors investigate commercially available Fe(II) chelators with spectral markers coupled to metal binding and release to determine their utility as competitors while measuring aspects of metal binding by apoproteins, during a metal binding competition assay. Adding chelators during apoprotein metal binding mimics heterogeneous metal binding environments present in vivo, and provides a more realistic metal binding affinity measurement. Ferrous chelators explored within this report include: Rhod-5N, Magfura-2, Fura-4F, Fura-2, and TPA (Tris-(2-byridyl-methyl)amine. These chelators were used to calibrate binding affinities for yeast and fly frataxin (Yfh1 and Dfh, respectively).

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