E-LIB Suche - Ergebnisse für: Baruteau, J.

1

mRNA therapy corrects defective glutathione metabolism and ..:

Gurung, S ; Timmermand, OV ; Perocheau, D...
https://discovery.ucl.ac.uk/id/eprint/10178381/1/STM.pdf. , 2024

2

Liver-directed gene therapy for inherited metabolic disease:

Baruteau, J ; Brunetti-Pierri, N ; Gissen, P
https://discovery.ucl.ac.uk/id/eprint/10185756/1/Liver%20directed%20gene%20therapy%20for%20inherited%20metabolic%20diseases.pdf. , 2024

Link: https://discovery.ucl.ac..

3

Pyruvate carboxylase deficiency type C; variable presentati..:

Bernhardt, I ; Van Dorp, L ; Dixon, M...
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC10764197/. , 2023

Link: http://www.ncbi.nlm.nih...

4

Assessment of pre-clinical liver models based on their abil..:

Westhaus, A ; Cabanes Creus, M ; Dilworth, K.L...
http://edoc.mdc-berlin.de/23212/1/23212oa.pdf. , 2023

Link: http://edoc.mdc-berlin.d..

5

Gene therapy restores dopamine transporter expression and a..:

Ng, J ; Barral, S ; De La Fuente Barrigon, C...
doi:10.1126/scitranslmed.aaw1564. , 2022

Link: https://doi.org/10.1126/..

6

Direct replacement of oral sodium benzoate with glycerol ph..:

Yeo, M ; Rehsi, P ; Dorman, M...
https://discovery.ucl.ac.uk/id/eprint/10145660/1/JIMD%20Reports%20-%202022%20-%20Yeo.pdf. , 2022

Link: https://discovery.ucl.ac..

7

Disentangling molecular and clinical stratification pattern..:

Tebani, A ; Sudrié-Arnaud, B ; Dabaj, I...
https://discovery.ucl.ac.uk/id/eprint/10126669/1/Tebani_et_2021_JMG.pdf. , 2021

Link: https://discovery.ucl.ac..

8

ASL expression in ALDH1A1+ neurons in the substantia nigra ..:

Lerner, S ; Eilam, R ; Adler, L...
https://discovery.ucl.ac.uk/id/eprint/10133933/1/Chainey-AlonsoBerbotto2021_Article_AStructuredMethodicalProcessFo.pdf. , 2021

Link: https://discovery.ucl.ac..

9

The exosome journey: from biogenesis to uptake and intracel..:

Gurung, S ; Perocheau, D ; Touramanidou, L.
https://discovery.ucl.ac.uk/id/eprint/10127020/1/s12964-021-00730-1.pdf. , 2021

Link: https://discovery.ucl.ac..

10

Gene Therapy for Lysosomal Storage Disorders: Ongoing Studi..:

Massaro, G ; Geard, AF ; Liu, W...
https://discovery.ucl.ac.uk/id/eprint/10126517/1/biomolecules-11-00611-v2.pdf. , 2021

Link: https://discovery.ucl.ac..

11

Novel therapies for mucopolysaccharidosis type III:

Seker Yilmaz, B ; Davison, J ; Jones, SA.
https://discovery.ucl.ac.uk/id/eprint/10110949/1/Baruteau_J%20of%20Inher%20Metab%20Disea%20-%202020%20-%20Seker%20Yilmaz%20-%20Novel%20therapies%20for%20mucopolysaccharidosis%20type%20III.pdf. , 2021

Link: https://discovery.ucl.ac..

12

Undiagnosed Phenylketonuria Can Exist Everywhere: Results F..:

van Wegberg A. M. J ; Trefz F ; Gizewska M...
info:eu-repo/semantics/altIdentifier/pmid/34474089. , 2021

Link: https://hdl.handle.net/1..

13

Clinical applications for exosomes: are we there yet?:

Perocheau, D ; Touramanidou, L ; Gurung, S..
https://discovery.ucl.ac.uk/id/eprint/10124283/7/Baruteau_bph.15432.pdf. , 2021

Link: https://discovery.ucl.ac..

14

Gene therapy restores dopamine transporter expression and a..:

Ng, J ; Barral, S ; Barrigon, CDLF...
https://discovery.ucl.ac.uk/id/eprint/10130034/1/Waddington_Science%20translational%20medicine%20Gene%20therapy%20for%20DTDS%20accepted%20manuscript.pdf. , 2021

Link: https://discovery.ucl.ac..

15

Safety and efficacy of an engineered hepatotropic AAV gene ..:

Baruteau, J ; Cunningham, SC ; Yilmaz, BS...
https://discovery.ucl.ac.uk/id/eprint/10136674/1/Baruteau_Safety%20and%20efficacy%20of%20an%20engineered%20hepatotropic%20AAV%20gene%20therapy%20for%20ornithine%20transcarbamylase%20deficiency%20in%20cynomolgus%20monkeys_VoR.pdf. , 2021

Link: https://discovery.ucl.ac..