Search Results - "Zhang, H. Steve"
-
1
Generation of Isogenic Pluripotent Stem Cells Differing Exclusively at Two Early Onset Parkinson Point Mutations
Published in Cell (22-07-2011)“…Patient-specific induced pluripotent stem cells (iPSCs) derived from somatic cells provide a unique tool for the study of human disease, as well as a promising…”
Get full text
Journal Article -
2
LRRK2 mutations cause mitochondrial DNA damage in iPSC-derived neural cells from Parkinson's disease patients: Reversal by gene correction
Published in Neurobiology of disease (01-02-2014)“…Abstract Parkinson's disease associated mutations in leucine rich repeat kinase 2 ( LRRK2 ) impair mitochondrial function and increase the vulnerability of…”
Get full text
Journal Article -
3
An engineered zinc finger protein activator of the endogenous glial cell line-derived neurotrophic factor gene provides functional neuroprotection in a rat model of Parkinson's disease
Published in The Journal of neuroscience (08-12-2010)“…Loss of dopaminergic neurons is primarily responsible for the onset and progression of Parkinson's disease (PD); thus, neuroprotective and/or neuroregenerative…”
Get full text
Journal Article -
4
A Designed Zinc-finger Transcriptional Repressor of Phospholamban Improves Function of the Failing Heart
Published in Molecular therapy (01-08-2012)“…Selective inhibition of disease-related proteins underpins the majority of successful drug–target interactions. However, development of effective antagonists…”
Get full text
Journal Article -
5
Genome editing with engineered zinc finger nucleases
Published in Nature reviews. Genetics (01-09-2010)“…Key Points Targeted genetic engineering in many important model systems and in human tissue culture cells has historically been challenging. This has changed…”
Get full text
Journal Article -
6
A TALE nuclease architecture for efficient genome editing
Published in Nature biotechnology (01-02-2011)“…TALEs (transcription activator-like effectors) are transcription factors from the plant pathogen Xanthomonas that can be readily engineered to bind new DNA…”
Get full text
Journal Article -
7
Transduction of striatum and cortex tissues by adeno-associated viral vectors produced by herpes simplex virus- and baculovirus-based methods
Published in Journal of virological methods (2012)“…► We examined the in vivo performances of rAAV2 packaged with different methods. ► Different packaging methods caused no changes in in vivo performances of…”
Get full text
Journal Article -
8
Locus-specific epigenetic remodeling controls addiction- and depression-related behaviors
Published in Nature neuroscience (01-12-2014)“…The authors selectively modify chromatin in a specific gene in vivo to examine the link between chromatin dynamics and drug- and stress-evoked responses. They…”
Get full text
Journal Article -
9
Persistent repression of tau in the brain using engineered zinc finger protein transcription factors
Published in Science advances (01-03-2021)“…Neuronal tau reduction confers resilience against β-amyloid and tau-related neurotoxicity in vitro and in vivo. Here, we introduce a novel translational…”
Get full text
Journal Article -
10
Ocular Gene Transfer with Self-Complementary AAV Vectors
Published in Investigative ophthalmology & visual science (01-07-2007)“…Self-complementary AAV (scAAV) vectors have been developed to circumvent rate-limiting second-strand synthesis in single-stranded AAV vector genomes and to…”
Get full text
Journal Article -
11
Generation of Isogenic Pluripotent Stem Cells Differing Exclusively at Two Early Onset Parkinson Point Mutations
Published in Cell (19-08-2011)Get full text
Journal Article -
12
Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington’s disease
Published in Nature medicine (01-07-2019)“…Huntington’s disease (HD) is a dominantly inherited neurodegenerative disorder caused by a CAG trinucleotide expansion in the huntingtin gene ( HTT ), which…”
Get full text
Journal Article -
13
Axonal transport of AAV9 in nonhuman primate brain
Published in Gene therapy (01-06-2016)“…A pilot study in nonhuman primates was conducted, in which two Rhesus macaques received bilateral parenchymal infusions of adeno-associated virus serotype 9…”
Get full text
Journal Article -
14
A7 Drug discovery approach for rare neurological diseases: using novel zinc finger protein technology to develop potential therapy for huntington’s disease
Published in Journal of neurology, neurosurgery and psychiatry (01-09-2016)“…There are approximately 7000 known rare and orphan diseases, over a third of which affect the central nervous system, virtually all do not have adequate…”
Get full text
Journal Article -
15
A Designed Zinc-finger Transcriptional Repressor of Phospholamban Improves Function of the Failing Heart
Published in Molecular therapy (01-12-2012)Get full text
Journal Article -
16
649. Repression of Vascular Endothelial Growth Factor-A Over-Expression in Glioblastoma Cells Using Engineered Zinc-Finger Transcription Factors
Published in Molecular therapy (01-05-2004)“…Angiogenesis is of critical importance to tumor progression and therefore is an attractive target in the treatment of cancer. The pro-angiogenic Vascular…”
Get full text
Journal Article