Showing results (11-20 of 67) with videos related to

Sort By:
Pageof 7
Molecular Therapy. Nucleic Acids|June 19, 2017
Antisense Oligonucleotides Promote Exon Inclusion and Correct the Common c.-32-13T>G GAA Splicing Variant in Pompe DiseaseErik van der Wal, Atze J Bergsma, Joon M Pijnenburg, et al.
Analytical Chemistry|March 25, 2015
Absolute quantification of the total and antidrug antibody-bound concentrations of recombinant human α-glucosidase in human plasma using protein G extraction and LC-MS/MSKees J Bronsema, Rainer Bischoff, W W M Pim Pijnappel, et al.
Journal of Proteome Research|September 8, 2009
In-depth profiling of post-translational modifications on the related transcription factor complexes TFIID and SAGANikolai Mischerikow, Gianpiero Spedale, A F Maarten Altelaar, et al.
Molecular Therapy. Methods & Clinical Development|August 11, 2020
Ready for Repair? Gene Editing Enters the Clinic for the Treatment of Human DiseaseMartijn P T Ernst, Mike Broeders, Pablo Herrero-Hernandez, et al.
Annals of Translational Medicine|August 9, 2019
Restoring the regenerative balance in neuromuscular disorders: satellite cell activation as therapeutic target in Pompe diseaseGerben J Schaaf, Rodrigo Canibano-Fraile, Tom J M van Gestel, et al.
Molecular Therapy. Nucleic Acids|September 14, 2016
From Cryptic Toward Canonical Pre-mRNA Splicing in Pompe Disease: a Pipeline for the Development of Antisense OligonucleotidesAtze J Bergsma, Stijn Lm In 't Groen, Frans W Verheijen, et al.
Skeletal Muscle|February 23, 2024
A knock down strategy for rapid, generic, and versatile modelling of muscular dystrophies in 3D-tissue-engineered-skeletal muscleStijn L M In 't Groen, Marnix Franken, Theresa Bock, et al.
Cancer Research|May 13, 2010
A role for BAF57 in cell cycle-dependent transcriptional regulation by the SWI/SNF chromatin remodeling complexNasun Hah, Annemieke Kolkman, Donald D Ruhl, et al.
Nature Biotechnology|March 11, 2003
Engineering the mouse genome with bacterial artificial chromosomes to create multipurpose allelesGiuseppe Testa, Youming Zhang, Kristina Vintersten, et al.
The Journal of Pediatrics|May 14, 2022
Effect of Anti-Iduronate 2-Sulfatase Antibodies in Patients with Mucopolysaccharidosis Type II Treated with Enzyme Replacement TherapyAudrey A M Vollebregt, Marianne Hoogeveen-Westerveld, George J Ruijter, et al.
Pageof 7