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Human Gene Therapy
|
December 29, 2025
Brain-Directed AAV Gene Therapy Rescues a Mouse Model of the CLN5 Form of Neuronal Ceroid Lipofuscinosis Disease and Normalizes a Blood Plasma Biomarker of Neurodegeneration
Wenfei Liu, Amy F Geard, Giulia Massaro, et al.
Plos One
|
March 29, 2016
Defective Cytochrome P450-Catalysed Drug Metabolism in Niemann-Pick Type C Disease
Elena-Raluca Nicoli, Nada Al Eisa, Celine V M Cluzeau, et al.
Iscience
|
November 9, 2020
c-Abl Inhibition Activates TFEB and Promotes Cellular Clearance in a Lysosomal Disorder
Pablo S Contreras, Pablo J Tapia, Lila González-Hódar, et al.
Nature
|
July 23, 2020
MeCP2 links heterochromatin condensates and neurodevelopmental disease
Charles H Li, Eliot L Coffey, Alessandra Dall'Agnese, et al.
Iscience
|
June 3, 2024
Compartmentalization proteomics revealed endolysosomal protein network changes in a goat model of atrial fibrillation
Thamali Ayagama, Philip D Charles, Samuel J Bose, et al.
Clinical Ophthalmology (Auckland, N.Z.)
|
November 29, 2023
One-Year and 18-Month Outcomes in nAMD Patient Eyes Switched to Brolucizumab Alone versus to Brolucizumab Alternating with Other Anti-VEGF Agents
Joseph M Coney, Jasmyne E McCoy, Samriddhi Buxy Sinha, et al.
Frontiers in Genetics
|
March 29, 2021
Precision Population Medicine in Primary Care: The Sanford Chip Experience
Kurt D Christensen, Megan Bell, Carrie L B Zawatsky, et al.
JAMA Dermatology
|
October 23, 2024
Upadacitinib in Adolescents With Moderate to Severe Atopic Dermatitis: Analysis of 3 Phase 3 Randomized Clinical Trials Through 76 Weeks
Amy S Paller, Pedro Mendes-Bastos, Elaine Siegfried, et al.
Scientific Reports
|
July 15, 2017
Gasoline cars produce more carbonaceous particulate matter than modern filter-equipped diesel cars
S M Platt, I El Haddad, S M Pieber, et al.
European Journal of Immunology
|
May 16, 2012
Invariant natural killer T cells are not affected by lysosomal storage in patients with Niemann-Pick disease type C
Anneliese O Speak, Nicholas Platt, Mariolina Salio, et al.
Page
of 51
Search research articles
Search
Showing results (471-480 of 505) with videos related to
Sort By:
Page
of 51
Human Gene Therapy
|
December 29, 2025
Brain-Directed AAV Gene Therapy Rescues a Mouse Model of the CLN5 Form of Neuronal Ceroid Lipofuscinosis Disease and Normalizes a Blood Plasma Biomarker of Neurodegeneration
Wenfei Liu, Amy F Geard, Giulia Massaro, et al.
Plos One
|
March 29, 2016
Defective Cytochrome P450-Catalysed Drug Metabolism in Niemann-Pick Type C Disease
Elena-Raluca Nicoli, Nada Al Eisa, Celine V M Cluzeau, et al.
Iscience
|
November 9, 2020
c-Abl Inhibition Activates TFEB and Promotes Cellular Clearance in a Lysosomal Disorder
Pablo S Contreras, Pablo J Tapia, Lila González-Hódar, et al.
Nature
|
July 23, 2020
MeCP2 links heterochromatin condensates and neurodevelopmental disease
Charles H Li, Eliot L Coffey, Alessandra Dall'Agnese, et al.
Iscience
|
June 3, 2024
Compartmentalization proteomics revealed endolysosomal protein network changes in a goat model of atrial fibrillation
Thamali Ayagama, Philip D Charles, Samuel J Bose, et al.
Clinical Ophthalmology (Auckland, N.Z.)
|
November 29, 2023
One-Year and 18-Month Outcomes in nAMD Patient Eyes Switched to Brolucizumab Alone versus to Brolucizumab Alternating with Other Anti-VEGF Agents
Joseph M Coney, Jasmyne E McCoy, Samriddhi Buxy Sinha, et al.
Frontiers in Genetics
|
March 29, 2021
Precision Population Medicine in Primary Care: The Sanford Chip Experience
Kurt D Christensen, Megan Bell, Carrie L B Zawatsky, et al.
JAMA Dermatology
|
October 23, 2024
Upadacitinib in Adolescents With Moderate to Severe Atopic Dermatitis: Analysis of 3 Phase 3 Randomized Clinical Trials Through 76 Weeks
Amy S Paller, Pedro Mendes-Bastos, Elaine Siegfried, et al.
Scientific Reports
|
July 15, 2017
Gasoline cars produce more carbonaceous particulate matter than modern filter-equipped diesel cars
S M Platt, I El Haddad, S M Pieber, et al.
European Journal of Immunology
|
May 16, 2012
Invariant natural killer T cells are not affected by lysosomal storage in patients with Niemann-Pick disease type C
Anneliese O Speak, Nicholas Platt, Mariolina Salio, et al.
Page
of 51