3 Photos and videos
4 Oct 2024
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
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4 Oct 2024
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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FrattaLab retweeted
1 Oct 2024
#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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24 Jun 2024
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/…
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13 Mar 2024
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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FrattaLab retweeted
8 Mar 2024
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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FrattaLab retweeted
2 Mar 2024
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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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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24 Jan 2024
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/…
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24 Jan 2024
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/…
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FrattaLab retweeted
18 Jan 2024
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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16 Nov 2023
See the full thread from brilliant first author @OscarWilkins16
Exceptional collaborators on this, including @ClaireLePichon @ule_lab @MaxZYJChien Jo Wlaschin
@UCLIoN @TheCrick
16 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/…
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16 Nov 2023
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/…
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FrattaLab retweeted
21 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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FrattaLab retweeted
19 Jul 2023
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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21 Jun 2023
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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FrattaLab retweeted
15 Mar 2023
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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16 Mar 2023
Well done anna-leigh!! @annaleighbrown2
14 Mar 2023
Congratulations to PhD student @annaleighbrown2 from @UCL, who is awarded the Jean Corsan Prize for the best scientific paper published by a PhD student!
Read more about our prize winners here 👇 alzheimersresearchuk.org/lon…
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20 Feb 2023
Kennedy's disease, caused by a CAG expansion, is considered rare (1:33K males). We found a 1:3K mutation frequency in >110K X chromosomes, predicting a 1:6.8K prevalence. Why this discrepancy? Reduced penetrance or misdiagnosis?
check our paper @Brain1878
doi.org/10.1093/brain/awad05…
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20 Feb 2023
This is work from brilliant @mattzano and @kriski_ibanez from @arianna_tucci Lab.
Made possible thanks to the great resources from @GenomicsEngland , @gnomad_project, Bryan Traynor and Project MinE @_Makeityours
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