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
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