Related references
Note: Only part of the references are listed.The delivery challenge: fulfilling the promise of therapeutic genome editing
Joost van Haasteren et al.
NATURE BIOTECHNOLOGY (2020)
Cas9 gene therapy for Angelman syndrome trapsUbe3a-ATSlong non-coding RNA
Justin M. Wolter et al.
NATURE (2020)
Genome editing in the mammalian brain using the CRISPR-Cas system
Jun Nishiyama
NEUROSCIENCE RESEARCH (2019)
CRISPR-Cas9-Mediated Genome Editing Increases Lifespan and Improves Motor Deficits in a Huntington's Disease Mouse Model
Freja K. Ekman et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2019)
CRISPR/Cas9 Mediated Disruption of the Swedish APP Allele as a Therapeutic Approach for Early-Onset Alzheimer's Disease
Bence Gyorgy et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2018)
A behavioral test battery for mouse models of Angelman syndrome: a powerful tool for testing drugs and novel Ube3a mutants
Monica Sonzogni et al.
MOLECULAR AUTISM (2018)
In vivo genome editing improves motor function and extends survival in a mouse model of ALS
Thomas Gaj et al.
SCIENCE ADVANCES (2017)
Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain
Benjamin E. Deverman et al.
NATURE BIOTECHNOLOGY (2016)
Angelman syndrome - insights into a rare neurogenetic disorder
Karin Buiting et al.
NATURE REVIEWS NEUROLOGY (2016)
Ube3a reinstatement identifies distinct developmental windows in a murine Angelman syndrome model
Sara Silva-Santos et al.
JOURNAL OF CLINICAL INVESTIGATION (2015)
Therapeutic genome editing: prospects and challenges
David Benjamin Turitz Cox et al.
NATURE MEDICINE (2015)
Towards a therapy for Angelman syndrome by targeting a long non-coding RNA
Linyan Meng et al.
NATURE (2015)
Truncation of Ube3a-ATS Unsilences Paternal Ube3a and Ameliorates Behavioral Defects in the Angelman Syndrome Mouse Model
Linyan Meng et al.
PLOS GENETICS (2013)
Topoisomerase inhibitors unsilence the dormant allele of Ube3a in neurons
Hsien-Sung Huang et al.
NATURE (2012)
Molecular and Clinical Aspects of Angelman Syndrome
A. Dagli et al.
MOLECULAR SYNDROMOLOGY (2011)
The Angelman syndrome ubiquitin ligase localizes to the synapse and nucleus, and maternal deficiency results in abnormal dendritic spine morphology
Scott V. Dindot et al.
HUMAN MOLECULAR GENETICS (2008)
Human synapsin 1 gene promoter confers highly neuron-specific long-term transgene expression from an adenoviral vector in the adult rat brain depending on the transduced area
S Kügler et al.
GENE THERAPY (2003)
The Prader-Willi syndrome imprinting center activates the paternally expressed murine Ube3a antisense transcript but represses paternal Ube3a
SJ Chamberlain et al.
GENOMICS (2001)
Share Paper
