New Fellowship for Graduate Students from the FARA Grant Program |
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The Graduate Research Fellowship is competitive fellowship that provides 1 to 3 years of support for individuals engaged in graduate study focused on FA leading to a Doctor of Philosophy (Ph.D.) degree (or equivalent). Applicants need to be matriculated in the chosen graduate school and have a chosen thesis adviser at the time of application and provide proof of passing the qualifying exam, if applicable. The research proposal must focus on projects that fall within FARA’s Research Priorities, as outlined here. Preliminary data are encouraged but not required. Maximum budget request is $50,000 per year. LOI deadline is March 15, 2023. For more information, visit FARA's Grant Program page. LOIs can be submitted here.
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The FARA Fellow Program
The FARA Fellow Program recognizes young investigators who will commit their early career to FA research and provides them with opportunities to grow their profile and network with other investigators.
- Both new and existing postdoctoral trainees with fewer than 6 years working as a postdoctoral trainee, accounting for career disruptions, and fewer than 4 years working in an FA lab as a postdoctoral trainee, can be nominated to receive the award.
- Nominations must be submitted by the trainee’s PI or prospective PI and consist of the trainee’s CV, documentation providing evidence that the PI can support the trainee’s salary for 2 years and a letter briefly describing how the trainee’s qualities and research accomplishments will support FARA's mission of developing treatments and a cure for FA.
- The award consists of $10,000 USD and financial support to attend one scientific conference per year and the FARA & CHOP FA Symposium in Philadelphia.
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As FARA Fellows, trainees will be invited to attend round table discussions with other Fellows, present at the FARA Forum and at the FARA Flash Talks, and contribute their FA knowledge and results of their research to the FA CollaMap.
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CDMRP Funding Opportunities Announced |
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The FY23 Congressional budget signed on December 29, 2022 listed “Hereditary Ataxia” among the topics areas eligible for funding in 2023.
The 2023 funding opportunities within the Peer-Reviewed Medical Research Program can be seen here.
FARA and the National Ataxia Foundation are hosting an informational webinar on these funding opportunities on March 2 at 12:00pm ET. Please register here.
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Funding Opportunities
Clinical Trial Award (CTA)
- Pre-Application (Preproposal): April 12, 2023 5:00 p.m. ET
- Application: July 19, 2023 11:59 p.m. ET
Discovery Award (DA)
- Pre-Application (Letter of Intent): March 29, 2023 5:00 p.m. ET
- Application: April 26, 2023 11:59 p.m. ET
Focused Program Award (FPA)
- Pre-Application (Preproposal): April 12, 2023 5:00 p.m. ET
- Application: July 19, 2023 11:59 p.m. ET
Investigator-Initiated Research Award (IIRA)
- Pre-Application (Letter of Intent): April 19, 2023 5:00 p.m. ET
- Application: May 31, 2023 11:59 p.m. ET
Lifestyle and Behavioral Health Interventions Research Award (LBIRA)
- Pre-Application (Letter of Intent): April 19, 2023 5:00 p.m. ET
- Application: May 31, 2023 11:59 p.m. ET
Technology/Therapeutic Development Award (TTDA)
- Pre-Application (Letter of Intent): April 19, 2023 5:00 p.m. ET
- Application: May 31, 2023 11:59 p.m. ET
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FARA Flash Talk Series 2023 - Call for Abstracts |
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During the month of May, to celebrate FA Awareness Month, FARA will host the annual Flash Talks series. These webinars will feature presentations from graduate students, postdocs, and residents involved in FA research. This is a great chance for FA patients, caregivers, and friends to learn about FARA funded research and get to know and interact with junior investigators involved in FA research.
We encourage all postdocs and grad students working in the FA field to express their interest in participating by submitting a short abstract and filling out this form by March 1, 2023. Speakers will be notified by March 21, 2023. The schedule of talks will be finalized in April and the webinar series will take place in May. The most voted presentations will be recognized with an award.
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Recent FARA Funded FA Publications
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Neurobehavioral deficits of mice expressing a low level of G127V mutant frataxin
Fil D, Conley RL, Zuberi AR, Lutz CM, Gemelli T, Napierala M, Napierala JS.
This work aimed to determine the effect of the G130V (murine G127V) mutation on Fxn expression and to define its multi-system impact in vivo. CRISPR/Cas9 was used to introduce the G127V missense mutation in the Fxn coding sequence and homozygous mice (FxnG127V/G127V) were generated. The endogenous Fxn G127V protein is detected at much lower levels in all tissues analyzed from FxnG127V/G127V mice compared to age and sex-matched WT mice without differences in Fxn transcript levels. FxnG127V/G127V mice are significantly smaller than WT counterparts, but perform similarly in most neurobehavioral tasks.
FXN gene methylation determines carrier status in Friedreich ataxia
Lam C, Gilliam KM, Rodden LN, Schadt KA, Lynch DR, Bidichandani S.
In this paper, the authors explored if the FRDA differentially methylated region (FRDA-DMR) in intron 1, which is hypermethylated in cis with the GAA triplet-repeat (GAA-TRE), effectively detects heterozygous GAA-TRE. FXN DNA methylation reliably detects the GAA-TRE in the heterozygous state and offers a robust alternative strategy to diagnose FRDA due to compound heterozygosity and to identify asymptomatic heterozygous carriers of the GAA-TRE.
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Drosophila melanogaster frataxin: protein crystal and predicted solution structure with identification of the iron-binding regions
Rodrigues AV, Batelu S, Hinton TV, Rotondo J, Thompson L, Brunzelle JS, Stemmler TL.
The goal of this report is to provide structural details of the Drosophila melanogaster frataxin ortholog (Dfh), using both X-ray crystallography and nuclear magnetic resonance (NMR) spectroscopy, in order to provide the foundational insight needed to understand the structure-function correlation of the protein.
Cardiovascular Research in Friedreich Ataxia: Unmet Needs and Opportunities
Payne RM.
This review describes the current basic and clinical understanding of the FRDA heart, and most importantly, identify major gaps in knowledge that represent new directions and opportunities for research.
Quantitative Oculomotor Assessment in Hereditary Ataxia: Discriminatory Power, Correlation with Severity Measures, and Recommended Parameters for Specific Genotypes
Garces P, Antoniades CA, Sobanska A, Kovacs N, Ying SH, Gupta AS, Perlman S, Szmulewicz DJ, Pane C, Németh AH, Jardim LB, Coarelli G, Dankova M, Traschütz A, Tarnutzer AA.
This work aids in understanding the current knowledge of quantitative oculomotor parameters in hereditary ataxias, and identifies gaps for validation as potential trial outcome measures in specific ataxia genotypes.
A wearable motion capture suit and machine learning predict disease progression in Friedreich's ataxia
Kadirvelu B, Gavriel C, Nageshwaran S, Chan JPK, Nethisinghe S, Athanasopoulos S, Ricotti V, Voit T, Giunti P, Festenstein R, Faisal AA.
Here the author captured full-body movement kinematics from patients with wearable sensors, enabling them to define digital behavioral features. The work demonstrates how data-derived wearable biomarkers can track personal disease trajectories and indicates the potential of such biomarkers for substantially reducing the duration or size of clinical trials testing disease-modifying therapies and for enabling behavioral transcriptomics.
Protoporphyrin IX Binds to Iron(II)-Loaded and to Zinc-Loaded Human Frataxin
Bernardo-Seisdedos G, Schedlbauer A, Pereira-Ortuzar T, Mato JM, Millet O.
Here, the authors used NMR spectroscopy, Molecular Dynamics, and Docking approaches to demonstrate new roles of frataxin and showed that that frataxin also binds Zn2+ in a structurally similar way to Fe2+, but with lower affinity. Both Fe2+-loaded and Zn2+-loaded frataxins specifically associate to protoporphyrin IX with micromolar affinity, while apo-frataxin does not bind to the porphyrin.
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FARA Newly Awarded Research Grants
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General Research Grants
Massimo Pandolfo & Stefano Stifani (McGill University)
An induced pluripotent stem cell model to investigate microglia in Friedreich ataxia
Andre Nussenzweig (NIH)
Mechanism of repeat expansion in Friedreich's ataxia
Benoit D’Autreaux (CNRS-CEA-Université Paris-Saclay)
Discovery of frataxin substitute drug candidates for the treatment of Friedreich’s ataxia
Thiago Rezende (University of Campinas) & Ian Harding (Monash University)
An optimized deep learning-based method for cerebellum segmentation
Postdoctoral Fellowship
Lisa Romano (University of Florida)
Repeat associated non-AUG (RAN) proteins in FA: contribution to disease and therapeutic opportunities
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The FARA Forum is a monthly webinar featuring investigators who have been awarded FARA grants. The webinar is open to FARA grant awardees and it is held on the second Tuesday of every month. Here are the next dates:
March 14, 2023 12:00 PM ET
Marco Carpenter - Children's Hospital of Philadelphia
April 11, 2023 12:00PM ET
Ricardo Mouro Pinto - Massachusetts General Hospital, Harvard Medical School
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FARA Research Grants: General Research Grants, Postdoctoral Fellowships and Research Awards –Next LOI deadline is August 15, 2023; Graduate Research fellowship – LOI deadline is March 15, 2023. See FARA grant program priorities here.
Funding opportunities for rare disease research
FDA launched the FDA Rare Neurodegenerative Disease Grant Program when the Accelerating Access to Critical Therapies for ALS was enacted. The agency awards grants and contracts to public and private entities to cover costs of research and development of interventions intended to prevent, diagnose, mitigate, treat or cure ALS and other rare neurodegenerative diseases in adults and children. Click here to learn more.
NIH funding opportunities, R03, Small Grant Program
The purpose of this funding opportunity announcement is to solicit applications for pilot projects to elucidate a role for understudied proteins associated with rare diseases. Awards will support generation of preliminary data and/or tools around eligible understudied protein(s). The next application deadline is March 15, 2023. Click here to learn more.
Other grant opportunities from CIRM can be found here.
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Meetings of Interest to the FA Community |
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ASGCT May 16-20, 2023 Los Angeles, CA
ISSCR June 14-17, 2023 Boston
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Postdoctoral positions at UT Southwestern – Napierala Lab
Two postdoctoral fellow positions are immediately available to work on oligonucleotide-mediated therapeutic approaches for Friedreich’s ataxia (FRDA), one of the most common inherited ataxias, caused by expansion of trinucleotide repeats. The postdoctoral fellow will also be involved in developing approaches for gene replacement therapy for FRDA. Both projects involve work with state-of-the-art induced pluripotent stem cell (iPSC)-based models and/or mouse models of the disease. We are seeking highly motivated scientists to join our multi-disciplinary group at the University of Texas Southwestern Medical Center in Dallas, TX to advance development of cure for FRDA.
The position requires experience utilizing mammalian cell culture models, general molecular and cellular biology techniques and/or experience working with transgenic mouse models. Expertise in culture of iPSCs or in applications involving antisense oligonucleotides, genome editing, viral expression vectors, transcriptional regulation and post-translational histone modifications is desirable but not required.
We offer competitive pay starting at $55,000 per year plus insurance and a comprehensive benefits package. Successful candidates will be eligible for an additional award of $10,000 per year for up to three years. In addition, postdoctoral fellows will be able to participate in two conferences per year (expenses paid).
The Department of Neurology at UT Southwestern Medical Center is a nationally recognized leader in the comprehensive management of neurological conditions. Our vibrant Department comprises more than 75 full-time faculty in 12 subspecialty sections, supporting a spectrum of complex and innovative approaches to neurological care, a broad range of novel research programs, and abundant educational opportunities for trainees. Through collaborations with the Peter O’Donnell Jr. Brain Institute, the Department of Neurology is building state-of-the art facilities for drug and device testing and bringing current and new faculty together for collaborative research on brain disease therapy.
Dallas is an extremely vibrant, cosmopolitan metropolis of more than 10 million people and is home to world class restaurants, art and professional sports. Interested candidates please directly contact Dr. Marek Napierala at marek.napierala@utsouthwstern.edu.
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