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Showing 1–6 of 6 results

Advanced filters: Author: Justin M. Wolter Clear advanced filters

Cas9 gene therapy for Angelman syndrome traps Ube3a-ATS long non-coding RNA

Genomic integration of an adeno-associated virus vector in a mouse model of Angelman syndrome unsilences paternal Ube3a and rescues anatomical and behavioural phenotypes, suggesting a pathway towards the treatment of this neurodevelopmental disorder.

Justin M. Wolter
Hanqian Mao
Mark J. Zylka
Research21 Oct 2020
Nature

Volume: 587, P: 281-284
Multi-targeting zinc finger nuclease vector unsilences paternal UBE3A in a mouse model of Angelman syndrome

Hannah O. Bazick
Lucas M. James
Mark J. Zylka
ResearchOpen Access25 Nov 2025
Gene Therapy

P: 1-11
Stimulating Wnt signaling reveals context-dependent genetic effects on gene regulation in primary human neural progenitors

Matoba, Le, Valone et al. characterized context-dependent genetic effects on gene regulatory activity during Wnt stimulation, finding that genetic variant function during neurodevelopment patterning can lead to differences in adult brain traits.

Nana Matoba
Brandon D. Le
Jason L. Stein
Research30 Sept 2024
Nature Neuroscience

Volume: 27, P: 2430-2442
AAV-dCas9 vector unsilences paternal Ube3a in neurons by impeding Ube3a-ATS transcription

Snord115-targeted AAV-dCas9 can dose dependently disrupt Ube3a-ATS in the absence of DNA double-strand breaks or a chromatin repression domain, highlighting utility of dCas9 for transcriptional collision-based applications.

Justin M. Wolter
Lucas M. James
Mark J. Zylka
ResearchOpen Access02 Sept 2025
Communications Biology

Volume: 8, P: 1-14
Cell-type-specific effects of genetic variation on chromatin accessibility during human neuronal differentiation

Cell-type-specific chromatin accessibility QTL during neurogenesis allow fine mapping of causal variants and more complete underlying of regulatory mechanisms underlying noncoding loci associated with gene expression and neuropsychiatric disorders.

Dan Liang
Angela L. Elwell
Jason L. Stein
Research20 May 2021
Nature Neuroscience

Volume: 24, P: 941-953
High-throughput screening and classification of chemicals and their effects on neuronal gene expression using RASL-seq

Jeremy M. Simon
Smita R. Paranjape
Mark J. Zylka
ResearchOpen Access14 Mar 2019
Scientific Reports

Volume: 9, P: 1-9