
Simon Waddington
@ProfSimonWad
Followers
1K
Following
11K
Media
928
Statuses
6K
Professor of Gene Therapy, University College London 🧬🚲🛠
London, England
Joined August 2011
Now live - our review of neurodegeneration in lysosomal storage disease - comparing and contrasting disease mechanisms; emerging evidence of unifying convergence
onlinelibrary.wiley.com
There are currently at least 70 characterised lysosomal storage diseases (LSD) resultant from inherited single-gene defects. Of these, at least 30 present with central nervous system (CNS) neurodeg...
1
3
6
Keeping with the new principles of this place where positivity is rewarded: there is a lovely microblogging site hosting my 6th in a series of superwomen in the history of genetics. Rosalind Franklin - prodigious talent @_BSGCT.
0
0
2
Check out our translational research to develop a gene therapy for a childhood mitochondrial disease (@Mito_Therapy @Dr_RKarda)
0
1
4
Non-biology photo here. This is the Britannia Hotel in Manchester £50/night- (lecturing at Manchester Metropolitan). Manchester is a beautiful place. @DrNLK0 @drol007 @helencooksley
2
0
9
RT @ZLAsgarian: Check out our work at #ESGCT2024 :.•My poster #P0479 on our patented viral vector GT for metabolic diseases.•Ala'a Siam #OR….
0
5
0
RT @drelliechilcott: Come and work @UCL_IfWH with myself and @Dr_RKarda on a hugely exciting preclinical gene therapy project! Research as….
0
6
0
With @jerry760 and team at @dukenus @kkh_sg doing what I do best - frowning while looking down a microscope #iugt #genetherapy @UCL_IfWH @ProfAhadRahim @DrGiuliaMassaro @Dr_RKarda @PrenatalTherapy @DrNLK0
0
4
17
RT @Dr_RickPrivo: I'm pleased to share that I will be speaking at The Festival of Genomics & Biodata, taking place on the 29th-30th of Janu….
0
5
0
RT @CavernomaIre: 👋We have an update for you on the 'Novel Gene Therapy' research. For which Cavernoma Ireland is funding partner alongside….
0
2
0
RT @JBaruteau37171: The group is expanding! 👇 . New exciting post-doctoral researcher position available to study astrocytic GABA metabolis….
0
6
0
RT @Dr_RKarda: Our new pre-print paper on “AAV9-mediated targeting of natural antisense transcript as a novel treatment for Dravet Syndrome….
biorxiv.org
Dravet syndrome (DS) is a severe childhood onset developmental and epileptic encephalopathy which leads to life-long disability. Symptoms usually manifest in the first year of life and include...
0
14
0
RT @jantinaod: We have a new pre-print paper! “AAV9-mediated targeting of natural antisense transcript as a novel treatment for Dravet Synd….
biorxiv.org
Dravet syndrome (DS) is a severe childhood onset developmental and epileptic encephalopathy which leads to life-long disability. Symptoms usually manifest in the first year of life and include...
0
1
0
RT @DrHelenONeill: Today we are hosting our Inaugural Reproduction Symposium at @TheCrick and have invited University partners to present t….
0
5
0
RT @AnnaluciaDarbey: Muscles 💪 and AAV 🦠 - what more could you ask for? . Our preprint highlighting strategies for effective transduction o….
biorxiv.org
Viral vector technologies based on adeno-associated virus (AAV) have demonstrated promising ability to deliver genetic cargo to a range of organs in vivo, with several novel candidates showing...
0
12
0