@AberdareNic this will take some time and is not going to impact what is due to go to clinical trials now, but hopefully the following generation of therapies.
English
FrattaLab
76 posts
FrattaLab
@FrattaLab
FrattaLab @UCLIoN working on #RNA biology of #ALS and #SBMA and #Kennedy's disease
Inscrit le Kasım 2019
200 Abonnements970 Abonnés
@AberdareNic thanks Nicola. Others developing gene therapies can incorporate this technology to make them safer. We also hope this extra safety and reduced toxicity will allow all of us to be bolder and try "stronger" gene therapy approaches for sporadic ALS.
English
Even to my limited understanding, this seems very significant. I am in complete awe of the minds that develop such incredible technology, and the work and commitment it must take. I’d love to find out more about what this means for the future @FrattaLab
FrattaLab@FrattaLab
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/sc… @UCLIoN @TheCrick @OscarWilkins16 @MaxZYJChien
English
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
English
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/sc…
@UCLIoN @TheCrick @OscarWilkins16 @MaxZYJChien
English
FrattaLab retweeté
#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/s4159…
English
@BrielmaierL @PeteHarley95 @jbneuro we definitely look forward to independent confirmation. same ASOs can be used by anybody for all such experiments.
English
@FrattaLab @PeteHarley95 @jbneuro Since these ASOs are patented, will independent confirmation occur?
Or is that not necessary?
English
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/10.110…
English
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/res…
English
FrattaLab retweeté
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/res…
English
FrattaLab retweeté
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/article/…
English
FrattaLab retweeté
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
English
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/10.110…
biorxiv.org/content/10.110…
English
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/10.110…
English
FrattaLab retweeté
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/sh… 1/9
English
See the full thread from brilliant first author @OscarWilkins16
Exceptional collaborators on this, including @ClaireLePichon @ule_lab @MaxZYJChien Jo Wlaschin
@UCLIoN @TheCrick
Oscar Wilkins@OscarWilkins16
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/10.110…
English
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/10.110…
English
FrattaLab retweeté
Our latest @NeuroCellPress reveals widespread mislocalisation of proteins & mRNAs in ALS. This is partly reversed by VCP ATPase inhibition: cell.com/neuron/pdfExte… @UCLIoN @mndassoc @The_MRC @ListerInstitute @ojziff @harleyinthehood @erist_yiran @JacobNeeves @PataniLab @TheCrick
English
FrattaLab retweeté
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/sh… 1/5
English
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/se…
English
FrattaLab retweeté
