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Molecular Therapy. Methods & Clinical Development
|
September 2, 2025
Novel transferrin receptor-mediated enzyme replacement therapy efficiently treats myogenic and neurogenic aspects of Pompe disease in mice
Kelly George, Raquel Riley, Shan Zhou, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
December 11, 2025
AAV gene therapy for GBA1-related diseases
Swathi Ayloo, Jae Cheon Ryu, Shih-Ching Chou, et al.
Nature Neuroscience
|
December 19, 2022
Microglia ferroptosis is regulated by SEC24B and contributes to neurodegeneration
Sean K Ryan, Matija Zelic, Yingnan Han, et al.
Communications Biology
|
August 2, 2024
Glucosylceramide synthase modulation ameliorates murine renal pathologies and promotes macrophage effector function in vitro
Agnes Cheong, Florin Craciun, Hervé Husson, et al.
The Journal of Clinical Investigation
|
June 19, 2025
Cross-species efficacy of AAV-mediated ARSA replacement for metachromatic leukodystrophy
Shyam Ramachandran, Jeffery Ardinger, Jie Bu, et al.
Iscience
|
September 2, 2025
Elovl1 inhibition reduced very long chain fatty acids in a mouse model of adrenoleukodystrophy
Jeremy Y Huang, Brian Freed, Martin Hanus, et al.
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Search research articles
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Showing results (31-40 of 36) with videos related to
Sort By:
Page
of 4
You have reached the last page of results.
This site can display upto 36 results.
Molecular Therapy. Methods & Clinical Development
|
September 2, 2025
Novel transferrin receptor-mediated enzyme replacement therapy efficiently treats myogenic and neurogenic aspects of Pompe disease in mice
Kelly George, Raquel Riley, Shan Zhou, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
December 11, 2025
AAV gene therapy for GBA1-related diseases
Swathi Ayloo, Jae Cheon Ryu, Shih-Ching Chou, et al.
Nature Neuroscience
|
December 19, 2022
Microglia ferroptosis is regulated by SEC24B and contributes to neurodegeneration
Sean K Ryan, Matija Zelic, Yingnan Han, et al.
Communications Biology
|
August 2, 2024
Glucosylceramide synthase modulation ameliorates murine renal pathologies and promotes macrophage effector function in vitro
Agnes Cheong, Florin Craciun, Hervé Husson, et al.
The Journal of Clinical Investigation
|
June 19, 2025
Cross-species efficacy of AAV-mediated ARSA replacement for metachromatic leukodystrophy
Shyam Ramachandran, Jeffery Ardinger, Jie Bu, et al.
Iscience
|
September 2, 2025
Elovl1 inhibition reduced very long chain fatty acids in a mouse model of adrenoleukodystrophy
Jeremy Y Huang, Brian Freed, Martin Hanus, et al.
Page
of 4