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1 . Academic Journal
Expression of ALS-PFN1 impairs vesicular degradation in iPSC-derived microglia
저자
by
Salome Funes
;
Jonathan Jung
;
Del Hayden Gadd
;
Michelle Mosqueda
;
Jianjun Zhong
, et al.
소스
Nature Communications, Vol 15, Iss 1, Pp 1-25 (2024)
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7 . Academic Journal
EZH2 inhibition reactivates epigenetically silenced FMR1 and normalizes molecular and electrophysiological abnormalities in fragile X syndrome neurons
저자
by
Minggang Fang
;
Sara K. Deibler
;
Pranathi Meda Krishnamurthy
;
Feng Wang
;
Paola Rodriguez
, et al.
소스
Frontiers in Neuroscience, Vol 18 (2024)
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7 . Academic Journal
Focused Ultrasound-Mediated Disruption of the Blood–Brain Barrier for AAV9 Delivery in a Mouse Model of Huntington’s Disease
저자
by
Bernie S. Owusu-Yaw
;
Yongzhi Zhang
;
Lilyan Garrett
;
Alvin Yao
;
Kai Shing
, et al.
소스
Pharmaceutics, Vol 16, Iss 6, p 710 (2024)
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7 . Academic Journal
Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model
저자
by
Hector Ribeiro Benatti
;
Rachel D. Prestigiacomo
;
Toloo Taghian
;
Rachael Miller
;
Robert King
, et al.
소스
Molecular Therapy: Methods & Clinical Development, Vol 31, Iss , Pp 101122- (2023)
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7 . Academic Journal
AAVrh10 vector corrects pathology in animal models of GM1 gangliosidosis and achieves widespread distribution in the CNS of nonhuman primates
저자
by
Michaël Hocquemiller
;
Laura Giersch
;
Xin Mei
;
Amanda L. Gross
;
Ashley N. Randle
, et al.
소스
Molecular Therapy: Methods & Clinical Development, Vol 27, Iss , Pp 281-292 (2022)
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7 . Academic Journal
Real-time MR tracking of AAV gene therapy with βgal-responsive MR probe in a murine model of GM1-gangliosidosis
저자
by
Toloo Taghian
;
Ana Rita Batista
;
Sarah Kamper
;
Michael Caldwell
;
Laura Lilley
, et al.
소스
Molecular Therapy: Methods & Clinical Development, Vol 23, Iss , Pp 128-134 (2021)
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7 . Academic Journal
Upregulating β-hexosaminidase activity in rodents prevents α-synuclein lipid associations and protects dopaminergic neurons from α-synuclein-mediated neurotoxicity
저자
by
Oeystein R. Brekk
;
Joanna A. Korecka
;
Cecile C. Crapart
;
Mylene Huebecker
;
Zachary K. MacBain
, et al.
소스
Acta Neuropathologica Communications, Vol 8, Iss 1, Pp 1-14 (2020)
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7 . Academic Journal
7T MRI Predicts Amelioration of Neurodegeneration in the Brain after AAV Gene Therapy
저자
by
Heather L. Gray-Edwards
;
Anne S. Maguire
;
Nouha Salibi
;
Lauren E. Ellis
;
Taylor L. Voss
, et al.
소스
Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 258-270 (2020)
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7 . Academic Journal
Long-Term Therapeutic Efficacy of Intravenous AAV-Mediated Hamartin Replacement in Mouse Model of Tuberous Sclerosis Type 1
저자
by
Shilpa Prabhakar
;
Pike See Cheah
;
Xuan Zhang
;
Max Zinter
;
Maria Gianatasio
, et al.
소스
Molecular Therapy: Methods & Clinical Development, Vol 15, Iss , Pp 18-26 (2019)
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7 . Academic Journal
Selection of an Efficient AAV Vector for Robust CNS Transgene Expression
저자
by
Killian S. Hanlon
;
Jonah C. Meltzer
;
Tetyana Buzhdygan
;
Ming J. Cheng
;
Miguel Sena-Esteves
, et al.
소스
Molecular Therapy: Methods & Clinical Development, Vol 15, Iss , Pp 320-332 (2019)
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