Combination of stem cell and gene therapy ameliorates symptoms in Huntington's disease mice

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Combination of stem cell and gene therapy ameliorates symptoms in Huntington's disease mice is …
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scholarly articleQ13442814

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P356DOI10.1038/S41536-019-0066-7
P932PMC publication ID6435637
P698PubMed publication ID30937182

P50authorIn Ki ChoQ90333413
P2093author name stringAlvince Learnz Pongos
Anthony Wing Sang Chan
Carissa Emerson Hunter
Sarah Ye
P2860cites workHuntington's disease: a clinical reviewQ21202876
Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's diseaseQ24626259
Grafted human-induced pluripotent stem-cell-derived neurospheres promote motor functional recovery after spinal cord injury in miceQ24635187
Towards a transgenic model of Huntington's disease in a non-human primateQ24644013
Allele-specific silencing of mutant huntingtin in rodent brain and human stem cellsQ27324212
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Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factorsQ27860937
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Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patientsQ28542911
Dopaminergic neurons from midbrain-specified human embryonic stem cell-derived neural stem cells engrafted in a monkey model of Parkinson's diseaseQ28729121
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Singles engage the RNA interference pathwayQ84913461
Single-stranded siRNAs activate RNAi in animalsQ84913472
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Induced Pluripotent HD Monkey Stem Cells Derived Neural Cells for Drug DiscoveryQ89353427
Silencing mutant huntingtin by adeno-associated virus-mediated RNA interference ameliorates disease manifestations in the YAC128 mouse model of Huntington's diseaseQ39795092
Elimination of huntingtin in the adult mouse leads to progressive behavioral deficits, bilateral thalamic calcification, and altered brain iron homeostasis.Q41187089
Functional maturation of hPSC-derived forebrain interneurons requires an extended timeline and mimics human neural development.Q41325951
CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington's diseaseQ41661649
Taking a break from huntingtinQ41834318
Direct comparison of autologous and allogeneic transplantation of iPSC-derived neural cells in the brain of a non-human primateQ42072961
CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In VivoQ42504823
Differential fate and functional outcome of lithium chloride primed adult neural progenitor cell transplants in a rat model of Huntington diseaseQ42578017
Huntington's disease intranuclear inclusions contain truncated, ubiquitinated huntingtin proteinQ42598739
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Relationship between trinucleotide repeat expansion and phenotypic variation in Huntington's diseaseQ45290871
Preconditioning mesenchymal stem cells with the mood stabilizers lithium and valproic acid enhances therapeutic efficacy in a mouse model of Huntington's diseaseQ45294512
Genetically engineered mesenchymal stem cells reduce behavioral deficits in the YAC 128 mouse model of Huntington's diseaseQ45298203
Inhibition of Huntington synthesis by antisense oligodeoxynucleotidesQ45301678
MicroRNA-27a reduces mutant hutingtin aggregation in an in vitro model of Huntington's diseaseQ45302123
Speech and language delay are early manifestations of juvenile-onset Huntington diseaseQ45302788
Therapies targeting DNA and RNA in Huntington's diseaseQ45304275
The Self-Inactivating KamiCas9 System for the Editing of CNS Disease GenesQ47772508
Huntington diseaseQ48465200
Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR/Cas9.Q48840079
Aggregation of huntingtin in neuronal intranuclear inclusions and dystrophic neurites in brainQ29617982
Ablation of huntingtin in adult neurons is nondeleterious but its depletion in young mice causes acute pancreatitis.Q30356472
A two years longitudinal study of a transgenic Huntington disease monkeyQ30577737
Huntington disease: natural history, biomarkers and prospects for therapeuticsQ30774172
Human multipotent stromal cells (MSCs) increase neurogenesis and decrease atrophy of the striatum in a transgenic mouse model for Huntington's diseaseQ33533774
Gametic but not somatic instability of CAG repeat length in Huntington's disease.Q33596051
Developmental Whole Brain White Matter Alterations in Transgenic Huntington's Disease MonkeyQ33671108
Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice.Q33713689
microRNA-128a dysregulation in transgenic Huntington's disease monkeysQ33786296
Cell-based therapies for Huntington's diseaseQ33849242
RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse modelQ33936969
Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene / allele-specific silencing of mutant huntingtinQ34221351
Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeatQ34348297
Reprogramming Huntington Monkey Skin Cells into Pluripotent Stem CellsQ34356830
A common gene expression signature in Huntington's disease patient brain regionsQ34446889
Reversal of cellular phenotypes in neural cells derived from Huntington's disease monkey-induced pluripotent stem cellsQ34469493
Progressive cognitive deficit, motor impairment and striatal pathology in a transgenic Huntington disease monkey model from infancy to adulthoodQ35597258
Successful function of autologous iPSC-derived dopamine neurons following transplantation in a non-human primate model of Parkinson's diseaseQ35711351
RNA Sequence Analysis of Human Huntington Disease Brain Reveals an Extensive Increase in Inflammatory and Developmental Gene ExpressionQ35860947
miR-196a Ameliorates Cytotoxicity and Cellular Phenotype in Transgenic Huntington's Disease Monkey Neural CellsQ36133682
Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression.Q36239309
Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 miceQ36389503
Multiple pathways contribute to the pathogenesis of Huntington diseaseQ36686156
Refining the diagnosis of Huntington disease: the PREDICT-HD studyQ36732557
Induced pluripotent stem cell-derived neural cells survive and mature in the nonhuman primate brainQ36787847
Improved cell therapy protocols for Parkinson's disease based on differentiation efficiency and safety of hESC-, hiPSC-, and non-human primate iPSC-derived dopaminergic neuronsQ37179338
Mechanisms of neurodegeneration in Huntington's disease.Q37202865
Intracerebral transplantation of neural stem cells combined with trehalose ingestion alleviates pathology in a mouse model of Huntington's disease.Q37494307
Allele-selective inhibition of mutant huntingtin by peptide nucleic acid-peptide conjugates, locked nucleic acid, and small interfering RNA.Q37536671
Surveying the landscape of Huntington's disease mechanisms, measurements, and medicines.Q38234003
Men and mice: Relating their agesQ38643990
AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington's disease.Q38644645
Regenerative medicine in Huntington's disease: Strengths and weaknesses of preclinical studies.Q39145677
Transplants of adult mesenchymal and neural stem cells provide neuroprotection and behavioral sparing in a transgenic rat model of Huntington's diseaseQ39368017
P275copyright licenseCreative Commons Attribution 4.0 InternationalQ20007257
P6216copyright statuscopyrightedQ50423863
P433issue1
P407language of work or nameEnglishQ1860
P921main subjectHuntington's diseaseQ190564
P304page(s)7
P577publication date2019-03-26
P1433published inNPG Regenerative MedicineQ50815323
P1476titleCombination of stem cell and gene therapy ameliorates symptoms in Huntington's disease mice
P478volume4

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cites work (P2860)
Q90662510Recent Overview of the Use of iPSCs Huntington's Disease Modeling and Therapy
Q94560788Species Classification for Neuroscience Literature Based on Span of Interest Using Sequence-to-Sequence Learning Model
Q92662205Therapeutic Advances for Huntington's Disease