@FrattaLab

FrattaLab @UCLIoN working on #RNA biology of #ALS and #SBMA and #Kennedy's disease

frattalab.org
Joined November 2019
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  • Following200
  • Followers960
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Great perspective from Yi Zeng and Aaron Gitler. Wonderful collaborations and long journey with @ClaireLePichon and @ule_lab. And funding to start and now continue the work from UCL NgTP/@SRausingTrust @The_MRC @mndassoc @MNDoddie5 @TheCrick
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Out @ScienceMagazine! New tech to activate ALS gene therapies only in the right cells at the right time - Increased safety to have more options for ALS. We do it by taking advantage of cryptic splicing. science.org/doi/10.1126/scie… @UCLIoN @TheCrick @OscarWilkins16 @MaxZYJChien

Creation of de novo cryptic splicing for ALS and FTD precision medicine

Loss of function of the RNA-binding protein TDP-43 (TDP-LOF) is a hallmark of amyotrophic lateral sclerosis (ALS) and other neurodegenerative disorders. Here we describe TDP-REG, which exploits the...

science.org
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#RepeatExpansionDisorders are up to three times more common than current estimates. Underdiagnosis or reduced penetrance? by our wonderful dream team @kriski_ibanez @mattzano @chrsclrksn + Bharati Jadhav & Andy Sharp nature.com/articles/s41591-0…
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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 @PeteHarley95 in great collaboration with @jbneuro biorxiv.org/content/10.1101/…

Loss of TDP-43 induces synaptic dysfunction that is rescued by UNC13A splice-switching ASOs

TDP-43 loss of function induces multiple splicing changes, including a cryptic exon in the amyotrophic lateral sclerosis and fronto-temporal lobar degeneration risk gene UNC13A , leading to nonsens...

biorxiv.org
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New postdoc opportunity to work on splicing and ALS therapeutics @TheCrick and @UCLIoN. Basic molecular work with true translational potential. 4 days still to apply. jobs.ac.uk/job/DGJ551/resear…
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We're looking for someone with a creative mindset and excellent modern molecular biology skills (DNA manipulation, Illumina, Nanopore etc) to join our team at @ucl and @TheCrick in the @FrattaLab developing candidate gene therapies for ALS. Apply here: jobs.ac.uk/job/DGJ551/resear…
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Why small mindedness is having big consequences @Brain1878 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/artic…
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FrattaLab retweeted
Ruepp Lab@RueppLab27 Feb 2024
Do you love #RNA and #neuroscience? Are you looking for a #postdocposition on #ALS and gene therapy? Join @RueppLab @UKDRI @KingsIoPPN and @FrattaLab @UCLIoN for a collaborative pre-clinical project on a novel gene therapy approach for ALS. More info here: tinyurl.com/2cpcbsp3
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This work was led by @SamBryce_Smith and a joint effort with @mariasecrier 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/10.1101/… biorxiv.org/content/10.1101/…

TDP-43 nuclear loss in FTD/ALS causes widespread alternative polyadenylation changes

In frontotemporal dementia and amyotrophic lateral sclerosis, the RNA-binding protein TDP-43 is depleted from the nucleus. TDP-43 loss leads to cryptic exon inclusion but a role in other RNA proces...

biorxiv.org
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TDP-43 loss causes mis-splicing in ALS/FTD. We now show it also induces widespread cryptic 3'UTRs. This increases RNA stability, translation and function of ELK1 and other TFs. Also provides novel targets for biomarker and therapeutics development. biorxiv.org/content/10.1101/…

TDP-43 loss induces extensive cryptic polyadenylation in ALS/FTD

Nuclear depletion and cytoplasmic aggregation of the RNA-binding protein TDP-43 is the hallmark of ALS, occurring in over 97% of cases. A key consequence of TDP-43 nuclear loss is the de-repression...

biorxiv.org
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Really excited about our latest preprint, where we uncovered a neuroprotective role for neuronal polyunsaturated fatty acids in FTD and ALS. Led by @AshlingGiblin and @AlexCammack biorxiv.org/cgi/content/shor… 1/9
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See the full thread from brilliant first author @OscarWilkins16 Exceptional collaborators on this, including @ClaireLePichon @ule_lab @MaxZYJChien Jo Wlaschin @UCLIoN @TheCrick
Oscar Wilkins@OscarWilkins1616 Nov 2023
New BioRxiv paper on using cryptic splicing to unlock precision gene therapy activation for ALS, FTD & other TDP-opathies! Key contributions @MaxZYJChien Josette Wlaschin +many others from @ClaireLePichon @ule_lab @FrattaLab labs and more. A thread 🧵 1/13 biorxiv.org/content/10.1101/…

Creation of de novo cryptic splicing for ALS/FTD precision medicine

A system enabling the expression of therapeutic proteins specifically in diseased cells would be transformative, providing greatly increased safety and the possibility of pre-emptive treatment. Here...

biorxiv.org
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Want to deliver gene therapies broadly, but activate them only in diseased neurons? TDP-REG "hacks" TDP cryptic mis-splicing to drive expression only WHEN and WHERE needed! Less toxicity, wider therapeutic window, new options for sporadic ALS therapies. biorxiv.org/content/10.1101/…

Creation of de novo cryptic splicing for ALS/FTD precision medicine

A system enabling the expression of therapeutic proteins specifically in diseased cells would be transformative, providing greatly increased safety and the possibility of pre-emptive treatment. Here...

biorxiv.org
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FrattaLab retweeted
PataniLab@PataniLab21 Jul 2023
Our latest @NeuroCellPress reveals widespread mislocalisation of proteins & mRNAs in ALS. This is partly reversed by VCP ATPase inhibition: cell.com/neuron/pdfExtended/… @UCLIoN @mndassoc @The_MRC @ListerInstitute @ojziff @harleyinthehood @erist_yiran @JacobNeeves @PataniLab @TheCrick
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Check out our new preprint on generation and characterisation of C9orf72 polyGR and polyPR knock-in mice. A huge effort over many years by @milioto_carmelo and many collaborators incl. @marcaurelbusche @soyonhonglab biorxiv.org/cgi/content/shor… 1/5
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We are recruiting a lab manager! If interested in helping shape a growing lab and directly contribute to molecular and cellular translational research in ALS, in London, please apply below: ucl.ac.uk/work-at-ucl/search…
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Pleased to share this first-author review, out now in @MolNeuro! '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 🧠🧪
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