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Center for Gene Therapy
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Astrocytes from familial and sporadic ALS patients are toxic to motor neurons.
Nature biotechnology Sep, 2011 | Pubmed ID: 21832997
Transplantation of gene-corrected motor neurons as a therapeutic strategy for spinal muscular atrophy.
Molecular therapy : the journal of the American Society of Gene Therapy Mar, 2013 | Pubmed ID: 23449105
Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS.
Molecular therapy : the journal of the American Society of Gene Therapy Dec, 2013 | Pubmed ID: 24008656
Making sense of pain: are pluripotent stem cell-derived sensory neurons a new tool for studying pain mechanisms?
Molecular therapy : the journal of the American Society of Gene Therapy Aug, 2014 | Pubmed ID: 25082088
Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA - a dose response study in mice and nonhuman primates.
Molecular therapy : the journal of the American Society of Gene Therapy Oct, 2014 | Pubmed ID: 25358252
Glia-neuron interactions in neurological diseases: Testing non-cell autonomy in a dish.
Brain research 02, 2017 | Pubmed ID: 26778174
Major histocompatibility complex class I molecules protect motor neurons from astrocyte-induced toxicity in amyotrophic lateral sclerosis.
Nature medicine Apr, 2016 | Pubmed ID: 26928464
The C9orf72 protein interacts with Rab1a and the ULK1 complex to regulate initiation of autophagy.
The EMBO journal 08, 2016 | Pubmed ID: 27334615
AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD.
JCI insight 11, 2018 | Pubmed ID: 30429376
Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease.
Molecular therapy : the journal of the American Society of Gene Therapy 10, 2019 | Pubmed ID: 31331814
AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease.
Molecular therapy : the journal of the American Society of Gene Therapy 01, 2021 | Pubmed ID: 33010819
Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice.
Molecular therapy. Methods & clinical development Mar, 2021 | Pubmed ID: 33665223
The Research Institute at Nationwide Children’s Hospital
College of Medicine, The Ohio State University
Cassandra N. Dennys1,
Julieth A. Sierra-Delgado1,
Shrestha Sinha Ray1,
Annalisa M. Hartlaub1,
Florence S. Roussel1,
Yacidzohara Rodriguez1,
Kathrin Meyer1,2
1Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital,
2, College of Medicine, The Ohio State University
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