E-LIB Suche - Ergebnisse für: Garanto, Alejandro

1

Preclinical Development of Antisense Oligonucleotides to Re..:

Suárez-Herrera, Nuria ; Li, Catherina H. Z. ; Leijsten, Nico...
Cells. 13 (2024) 7 - p. 601 , 2024

Link: https://doi.org/10.3390/..

2

Efficacy, biodistribution and safety comparison of chemical..:

Vázquez-Domínguez, Irene ; Anido, Alejandro Allo ; Duijkers, Lonneke...
Nucleic Acids Research. , 2024

Link: https://doi.org/10.1093/..

3

Proof-of-concept for multiple AON delivery by a single U7sn..:

Suárez-Herrera, Nuria ; Riswick, Iris B. ; Vázquez-Domínguez, Irene...
Molecular Therapy. 32 (2024) 3 - p. 837-851 , 2024

Link: https://doi.org/10.1016/..

4

Generation of hiPSC lines from four pyridoxine-dependent ep..:

Schuurmans, Imke M.E. ; van Karnebeek, Clara D.M. ; Hoogendoorn, Anita D.M...
Stem Cell Research. 79 (2024) - p. 103480 , 2024

Link: https://doi.org/10.1016/..

5

Generation of hiPSC lines from four glutaric aciduria type ..:

Schuurmans, Imke M.E. ; van Karnebeek, Clara D.M. ; Hoogendoorn, Anita D.M....
Stem Cell Research. 79 (2024) - p. 103481 , 2024

Link: https://doi.org/10.1016/..

6

Generation of an induced pluripotent stem cell line carryin..:

Schuurmans, Imke M.E. ; Wu, Ka M. ; van Karnebeek, Clara D.M...
Stem Cell Research. 71 (2023) - p. 103173 , 2023

Link: https://doi.org/10.1016/..

7

Whole genome sequencing for USH2A-associated disease reveal..:

Reurink, Janine ; Weisschuh, Nicole ; Garanto, Alejandro...
Human Genetics and Genomics Advances. 4 (2023) 2 - p. 100181 , 2023

Link: https://doi.org/10.1016/..

8

Exploring genotype–phenotype correlations in glutaric acidu..:

Schuurmans, Imke M. E. ; Dimitrov, Bianca ; Schröter, Julian...
Journal of Inherited Metabolic Disease. 46 (2023) 3 - p. 371-390 , 2023

Link: https://doi.org/10.1002/..

9

Comparative Clustering (CompaCt) of eukaryote complexomes i..:

van Strien, Joeri ; Evers, Felix ; Lutikurti, Madhurya...
PLOS Computational Biology. 19 (2023) 8 - p. e1011090 , 2023

Link: https://doi.org/10.1371/..

10

Generation of an iPSC line (RMCGENi020-A) from a patient wi..:

Suárez-Herrera, Nuria ; Leijsten, Nico ; Albert, Silvia...
Stem Cell Research. 73 (2023) - p. 103252 , 2023

Link: https://doi.org/10.1016/..

11

CRISPR-Cas9 correction of a nonsense mutation in LCA5 rescu..:

Afanasyeva, Tess A.V. ; Athanasiou, Dimitra ; Perdigao, Pedro R.L....
Molecular Therapy - Methods & Clinical Development. 29 (2023) - p. 522-531 , 2023

Link: https://doi.org/10.1016/..

12

Gene augmentation of LCA5-associated Leber congenital amaur..:

Faber, Siebren ; Mercey, Olivier ; Junger, Katrin...
JCI Insight. 8 (2023) 10 - p. , 2023

Link: https://doi.org/10.1172/..

13

Progress and harmonization of gene editing to treat human d..:

Cavazza, Alessia ; Hendel, Ayal ; Bak, Rasmus O....
Molecular Therapy - Nucleic Acids. 34 (2023) - p. 102066 , 2023

Link: https://doi.org/10.1016/..

14

Generation of an induced pluripotent stem cell line carryin..:

Schuurmans, Imke M.E. ; Wu, Ka M. ; van Karnebeek, Clara D.M...
Stem Cell Research. 69 (2023) - p. 103069 , 2023

Link: https://doi.org/10.1016/..

15

In Vitro Skeletal Muscle Model of PGM1 Deficiency Reveals A..:

Conte, Federica ; Ashikov, Angel ; Mijdam, Rachel...
International Journal of Molecular Sciences. 24 (2023) 9 - p. 8247 , 2023

Link: https://doi.org/10.3390/..