Thursday, December 15, 2022

Design and Delivery of SINEUP: A New Modular Tool to Increase Protein Translation

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Arnoldi M, Zarantonello G, Espinoza S, Gustincich S, Di Leva F, Biagioli M.; Methods Mol Biol. 2022;2434:63-87. doi: 10.1007/978-1-0716-2010...

Recurrent repeat expansions in human cancer genomes

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Erwin, G.S., Gürsoy, G., Al-Abri, R. et al.; Nature (2022). doi:10.1038/s41586-022-05515-1  Expansions of tandem DNA repeats (TRs) are known...

Researchers may have found a new path for halting cancer cell production

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Stanford Medicine; December 14, 2022;  The project began not with cancer, but with a rare, neurodegenerative disease without a cure, Friedre...
Wednesday, December 14, 2022

Kaleibe launched by Replay to target genetic brain disorders with gene therapy

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14-12-2022​. Replay, a genome writing company reprogramming biology by writing and delivering big DNA, has announced the launch of Kaleibe, ...

G-rich motifs within phosphorothioate-based antisense oligonucleotides (ASOs) drive activation of FXN expression through indirect effects

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Feng Wang, Ezequiel Calvo-Roitberg, Julia M Rembetsy-Brown, Minggang Fang, Jacquelyn Sousa, Zachary J Kartje, Pranathi Meda Krishnamurthy, J...
Monday, December 12, 2022

Retinal and Visual Pathways Involvement in Carriers of Friedreich’s Ataxia

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Ziccardi, L.; Barbano, L.; Antonelli, G.; Cioffi, E.; Di Renzo, A.; Gioiosa, V.; Marcotulli, C.; Grzybowski, A.; Casali, C.; Parisi, V. ; Di...
Sunday, December 11, 2022

Double blind trial of a deuterated form of linoleic acid (RT001) in Friedreich ataxia

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David R. Lynch, Katherine D. Mathews, Susan Perlman, Theresa Zesiewicz, Sub Subramony, Omid Omidvar, Adam P. Vogel, Ana Krtolica, Nadia Litt...

Ataxia Rating Scales: Content Analysis by Linking to the International Classification of Functioning, Disability and Health

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Etoom, M.; Jahan, A.M.; Alghwiri, A.; Lena, F.; Modugno, N.; Healthcare 2022, 10, 2459. doi:10.3390/healthcare10122459 The content analysis ...

Clinical Drug-Drug Interaction Between Vatiquinone, a 15-Lipoxygenase Inhibitor, and Rosuvastatin, a Breast Cancer Resistance Protein Substrate

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Lee, L., Murase, K., Ma, J. and Thoolen, M. (2022); Clin Pharmacol Drug Dev. doi:10.1002/cpdd.1199 These results demonstrate that vatiquinon...
Saturday, December 10, 2022

Ataxia Rating Scales Reflect Patient Experience: an Examination of the Relationship Between Clinician Assessments of Cerebellar Ataxia and Patient-Reported Outcomes

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Michele H. Potashman, Miranda L. Mize, Melissa W. Beiner, Samantha Pierce, Vladimir Coric & Jeremy D. Schmahmann; Cerebellum (2022). doi...