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Scientific Reports
|
March 21, 2020
Longitudinal phenotype development in a minipig model of neurofibromatosis type 1
Johanna Uthoff, Jared Larson, Takashi S Sato, et al.
Biorxiv : the Preprint Server for Biology
|
October 4, 2023
Sortilin inhibition treats multiple neurodegenerative lysosomal storage disorders
Hannah G Leppert, Joelle T Anderson, Kaylie J Timm, et al.
The Journal of Biological Chemistry
|
May 10, 2021
Endolysosomal N-glycan processing is critical to attain the most active form of the enzyme acid alpha-glucosidase
Nithya Selvan, Nickita Mehta, Suresh Venkateswaran, et al.
Progress in Neurobiology
|
March 22, 2020
A multimodal approach to identify clinically relevant biomarkers to comprehensively monitor disease progression in a mouse model of pediatric neurodegenerative disease
Tyler B Johnson, Jon J Brudvig, Kimmo K Lehtimäki, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
October 4, 2020
AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease
Tyler B Johnson, Katherine A White, Jon J Brudvig, et al.
Clinical and Translational Science
|
June 27, 2024
NF1<sup>+/ex42del</sup> miniswine model the cellular disruptions and behavioral presentations of NF1-associated cognitive and motor impairment
Vicki J Swier, Katherine A White, Pedro L Negrão de Assis, et al.
The Journal of Clinical Investigation
|
December 15, 2025
Corrigendum to A CLN6-CLN8 complex recruits lysosomal enzymes at the ER for Golgi transfer
Lakshya Bajaj, Jaiprakash Sharma, Alberto di Ronza, et al.
Molecular Therapy. Methods & Clinical Development
|
March 5, 2021
Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice
Katherine A White, Hemanth R Nelvagal, Timothy A Poole, et al.
The Journal of Clinical Investigation
|
June 30, 2020
A CLN6-CLN8 complex recruits lysosomal enzymes at the ER for Golgi transfer
Lakshya Bajaj, Jaiprakash Sharma, Alberto di Ronza, et al.
Frontiers in Genetics
|
April 10, 2023
Early postnatal administration of an AAV9 gene therapy is safe and efficacious in CLN3 disease
Tyler B Johnson, Jon J Brudvig, Shibi Likhite, et al.
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of 9
Search research articles
Search
Showing results (61-70 of 84) with videos related to
Sort By:
Page
of 9
Scientific Reports
|
March 21, 2020
Longitudinal phenotype development in a minipig model of neurofibromatosis type 1
Johanna Uthoff, Jared Larson, Takashi S Sato, et al.
Biorxiv : the Preprint Server for Biology
|
October 4, 2023
Sortilin inhibition treats multiple neurodegenerative lysosomal storage disorders
Hannah G Leppert, Joelle T Anderson, Kaylie J Timm, et al.
The Journal of Biological Chemistry
|
May 10, 2021
Endolysosomal N-glycan processing is critical to attain the most active form of the enzyme acid alpha-glucosidase
Nithya Selvan, Nickita Mehta, Suresh Venkateswaran, et al.
Progress in Neurobiology
|
March 22, 2020
A multimodal approach to identify clinically relevant biomarkers to comprehensively monitor disease progression in a mouse model of pediatric neurodegenerative disease
Tyler B Johnson, Jon J Brudvig, Kimmo K Lehtimäki, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
October 4, 2020
AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease
Tyler B Johnson, Katherine A White, Jon J Brudvig, et al.
Clinical and Translational Science
|
June 27, 2024
NF1<sup>+/ex42del</sup> miniswine model the cellular disruptions and behavioral presentations of NF1-associated cognitive and motor impairment
Vicki J Swier, Katherine A White, Pedro L Negrão de Assis, et al.
The Journal of Clinical Investigation
|
December 15, 2025
Corrigendum to A CLN6-CLN8 complex recruits lysosomal enzymes at the ER for Golgi transfer
Lakshya Bajaj, Jaiprakash Sharma, Alberto di Ronza, et al.
Molecular Therapy. Methods & Clinical Development
|
March 5, 2021
Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice
Katherine A White, Hemanth R Nelvagal, Timothy A Poole, et al.
The Journal of Clinical Investigation
|
June 30, 2020
A CLN6-CLN8 complex recruits lysosomal enzymes at the ER for Golgi transfer
Lakshya Bajaj, Jaiprakash Sharma, Alberto di Ronza, et al.
Frontiers in Genetics
|
April 10, 2023
Early postnatal administration of an AAV9 gene therapy is safe and efficacious in CLN3 disease
Tyler B Johnson, Jon J Brudvig, Shibi Likhite, et al.
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of 9