FrattaLab
@frattalab
FrattaLab @UCLIoN working on #RNA biology of #ALS and #SBMA and #Kennedy's disease
ID: 1190230623849459712
https://www.frattalab.org 01-11-2019 11:35:16
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Pleased to share this first-author review, out now in Molecular Neurodegeneration! 'The era of cryptic exons: implications for ALS-FTD' We discuss recent discoveries & the exciting opportunities that cryptic exons offer for the development of novel therapies & biomarkers in #ALS #MND #FTD 🧠🧪
Boosting muscle BDNF rescues impaired in vivo axonal transport of signalling endosomes in CMT2D mice: insight.jci.org/articles/view/… UCL Queen Square Institute of Neurology UCL Brain Sciences UK Dementia Research Institute #AxonalTransport #CMT #MotorNeuron #NMJ #PeripheralNerve CMT United Kingdom HereditaryNeuropathy CMT Research Foundation Muscular Dystrophy UK TREAT-NMD
Check out our new preprint on generation and characterisation of C9orf72 polyGR and polyPR knock-in mice. A huge effort over many years by Carmelo Milioto and many collaborators incl. Marc Aurel Busche Soyon Hong biorxiv.org/cgi/content/sh… 1/5
Our latest Neuron reveals widespread mislocalisation of proteins & mRNAs in ALS. This is partly reversed by VCP ATPase inhibition: cell.com/neuron/pdfExte… UCL Queen Square Institute of Neurology MND Association Medical Research Council The Lister Institute of Preventive Medicine Oliver Ziff Jasmine Harley @erist_yiran Jacob Neeves PataniLab The Crick
See the full thread from brilliant first author Oscar Wilkins Exceptional collaborators on this, including Claire Le Pichon ule_lab Max Z. Y. J. Chien Jo Wlaschin UCL Queen Square Institute of Neurology The Crick
Really excited about our latest preprint, where we uncovered a neuroprotective role for neuronal polyunsaturated fatty acids in FTD and ALS. Led by Ashling Giblin and Alex Cammack biorxiv.org/cgi/content/sh… 1/9
This work was led by Sam Bryce-Smith and a joint effort with Maria Secrier and many others. Parallel efforts from the Gitler and La Spada labs also identify the link between TDP-43 and 3'UTR changes and out now: biorxiv.org/content/10.110… biorxiv.org/content/10.110…
Do you love #RNA and #neuroscience? Are you looking for a #postdocposition on #ALS and gene therapy? Join Ruepp Lab UK Dementia Research Institute Institute of Psychiatry, Psychology & Neuroscience and FrattaLab UCL Queen Square Institute of Neurology for a collaborative pre-clinical project on a novel gene therapy approach for ALS. More info here: tinyurl.com/2cpcbsp3
Why small mindedness is having big consequences Brain I live under its shadow. I suspect most of you do too. It is the great mountain of small things. The higher it gets, the bigger the shadow it casts: a malignant darkness that pervades our lives. academic.oup.com/brain/article/…
Age-dependent and compartment-specific changes in mitochondrial homeostasis & cytoplasmic biophysics in ageing PNS neurons: onlinelibrary.wiley.com/doi/10.1111/ac… Big Aging Cell study & effort from Alessio Vagnoni UCL Queen Square Institute of Neurology Francesca Mattedi Sandy Richter Emily The MNP Lab #Ageing
We find TDP-43 loss to cause presynaptic defects, which are rescued by ASOs correcting a single cryptic exon in UNC13A. Surprising effect of just one cryptic exon and promising strategy for ALS! Matt Keuss Pete Harley in great collaboration with Juan Burrone Lab biorxiv.org/content/10.110…
Very nice work combining strategies for AAV targeting to muscle from Annalucia Darbey of the FrattaLab: biorxiv.org/content/10.110… UCL Queen Square Institute of Neurology UCL Brain Sciences The Crick UCL Div of Surgery UK Dementia Research Institute