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Brandon A Wustman

Showing results (11-20 of 18) with videos related to

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Nature Biotechnology|June 2, 2025
Dual SORT LNPs for multi-organ base editingMinjeong Kim, Eunice S Song, Joseph C Chen, et al.
The Journal of Neuroscience : the Official Journal of the Society for Neuroscience|April 13, 2012
Lysosomal dysfunction in a mouse model of Sandhoff disease leads to accumulation of ganglioside-bound amyloid-β peptideSerene Keilani, Yi Lun, Anthony C Stevens, et al.
Plos One|July 19, 2014
The pharmacological chaperone AT2220 increases the specific activity and lysosomal delivery of mutant acid alpha-glucosidase, and promotes glycogen reduction in a transgenic mouse model of Pompe diseaseRichie Khanna, Allan C Powe, Yi Lun, et al.
The FEBS Journal|February 13, 2010
The pharmacological chaperone isofagomine increases the activity of the Gaucher disease L444P mutant form of beta-glucosidaseRichie Khanna, Elfrida R Benjamin, Lee Pellegrino, et al.
Neurotherapeutics : the Journal of the American Society for Experimental Neurotherapeutics|July 20, 2014
A GCase chaperone improves motor function in a mouse model of synucleinopathyFranziska Richter, Sheila M Fleming, Melanie Watson, et al.
Proceedings of the National Academy of Sciences of the United States of America|April 28, 2025
Inhaled <i>DNAI1</i> mRNA therapy for treatment of primary ciliary dyskinesiaMirko Hennig, Rumpa B Bhattacharjee, Ishita Agarwal, et al.
Plos One|August 8, 2015
Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused AgalsidaseDavid G Warnock, Daniel G Bichet, Myrl Holida, et al.
Science (New York, N.Y.)|June 13, 2024
In vivo editing of lung stem cells for durable gene correction in miceYehui Sun, Sumanta Chatterjee, Xizhen Lian, et al.
Pageof 2

Showing results (11-20 of 18) with videos related to

Sort By:
Pageof 2
You have reached the last page of results.This site can display upto 18 results.
Nature Biotechnology|June 2, 2025
Dual SORT LNPs for multi-organ base editingMinjeong Kim, Eunice S Song, Joseph C Chen, et al.
The Journal of Neuroscience : the Official Journal of the Society for Neuroscience|April 13, 2012
Lysosomal dysfunction in a mouse model of Sandhoff disease leads to accumulation of ganglioside-bound amyloid-β peptideSerene Keilani, Yi Lun, Anthony C Stevens, et al.
Plos One|July 19, 2014
The pharmacological chaperone AT2220 increases the specific activity and lysosomal delivery of mutant acid alpha-glucosidase, and promotes glycogen reduction in a transgenic mouse model of Pompe diseaseRichie Khanna, Allan C Powe, Yi Lun, et al.
The FEBS Journal|February 13, 2010
The pharmacological chaperone isofagomine increases the activity of the Gaucher disease L444P mutant form of beta-glucosidaseRichie Khanna, Elfrida R Benjamin, Lee Pellegrino, et al.
Neurotherapeutics : the Journal of the American Society for Experimental Neurotherapeutics|July 20, 2014
A GCase chaperone improves motor function in a mouse model of synucleinopathyFranziska Richter, Sheila M Fleming, Melanie Watson, et al.
Proceedings of the National Academy of Sciences of the United States of America|April 28, 2025
Inhaled <i>DNAI1</i> mRNA therapy for treatment of primary ciliary dyskinesiaMirko Hennig, Rumpa B Bhattacharjee, Ishita Agarwal, et al.
Plos One|August 8, 2015
Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused AgalsidaseDavid G Warnock, Daniel G Bichet, Myrl Holida, et al.
Science (New York, N.Y.)|June 13, 2024
In vivo editing of lung stem cells for durable gene correction in miceYehui Sun, Sumanta Chatterjee, Xizhen Lian, et al.
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