Intrathecal delivery of frataxin mRNA encapsulated in lipid nanoparticles to dorsal root ganglia as a potential therapeutic for Friedreich's ataxia

scientific article published on 17 February 2016

Intrathecal delivery of frataxin mRNA encapsulated in lipid nanoparticles to dorsal root ganglia as a potential therapeutic for Friedreich's ataxia is …
instance of (P31):
scholarly articleQ13442814

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P356DOI10.1038/SREP20019
P932PMC publication ID4756688
P698PubMed publication ID26883577
P5875ResearchGate publication ID295173666

P50authorJeffrey MorinQ61142628
Joseph F NabhanQ81346655
Renea GoochQ83272755
P2093author name stringChristine E Bulawa
Timothy P LaBranche
Kristy M Wood
Surya Bhamidipaty
Varada P Rao
Braydon C Guild
Fazli Bozal
P2860cites workPathology and pathogenesis of sensory neuropathy in Friedreich's ataxiaQ83912301
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Mammalian iron-sulphur proteins: novel insights into biogenesis and functionQ38271496
Spectrum of neuropathophysiology in spinal muscular atrophy type IQ39084725
Transcription activator-like effector proteins induce the expression of the frataxin geneQ39348028
A Potential New Therapeutic Approach for Friedreich Ataxia: Induction of Frataxin Expression With TALE ProteinsQ39793086
Comparative Immunogenicity of a Cytotoxic T Cell Epitope Delivered by Penetratin and TAT Cell Penetrating Peptides.Q40654151
Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich's ataxiaQ42226636
Expression of therapeutic proteins after delivery of chemically modified mRNA in miceQ45866149
Human BAC-mediated rescue of the Friedreich ataxia knockout mutation in transgenic miceQ45875661
Iron-dependent regulation of frataxin expression: implications for treatment of Friedreich ataxiaQ24323371
Cardiomyopathy in Friedreich ataxia: clinical findings and researchQ26827933
Frataxin is reduced in Friedreich ataxia patients and is associated with mitochondrial membranesQ28249379
Friedreich's ataxia: autosomal recessive disease caused by an intronic GAA triplet repeat expansionQ28275699
Mouse models for Friedreich ataxia exhibit cardiomyopathy, sensory nerve defect and Fe-S enzyme deficiency followed by intramitochondrial iron depositsQ28505173
Modified mRNA directs the fate of heart progenitor cells and induces vascular regeneration after myocardial infarctionQ30580723
The in vivo mitochondrial two-step maturation of human frataxin.Q33363024
Mammalian frataxin: an essential function for cellular viability through an interaction with a preformed ISCU/NFS1/ISD11 iron-sulfur assembly complex.Q33813547
Cellular, molecular and functional characterisation of YAC transgenic mouse models of Friedreich ataxiaQ34150158
A rapid, noninvasive immunoassay for frataxin: Utility in assessment of Friedreich ataxiaQ34376985
Epigenetic and neurological effects and safety of high-dose nicotinamide in patients with Friedreich's ataxia: an exploratory, open-label, dose-escalation study.Q34418695
Human frataxin activates Fe-S cluster biosynthesis by facilitating sulfur transfer chemistryQ34434217
Repeat expansion affects both transcription initiation and elongation in friedreich ataxia cellsQ34575655
R-loops associated with triplet repeat expansions promote gene silencing in Friedreich ataxia and fragile X syndrome.Q35160930
Epigenetic therapy for Friedreich ataxiaQ35180607
An AAV9 coding for frataxin clearly improved the symptoms and prolonged the life of Friedreich ataxia mouse modelsQ35184184
Biodistribution of small interfering RNA at the organ and cellular levels after lipid nanoparticle-mediated deliveryQ35681765
A TAT-frataxin fusion protein increases lifespan and cardiac function in a conditional Friedreich's ataxia mouse modelQ35770976
Frataxin levels in peripheral tissue in Friedreich ataxiaQ36010435
Expression kinetics of nucleoside-modified mRNA delivered in lipid nanoparticles to mice by various routesQ36218294
Nikolaus Friedreich and degenerative atrophy of the dorsal columns of the spinal cordQ37040757
Improving the endosomal escape of cell-penetrating peptides and their cargos: strategies and challengesQ37277330
The pathogenesis of Friedreich ataxia and the structure and function of frataxinQ37414028
Friedreich ataxia: metal dysmetabolism in dorsal root gangliaQ37485501
The dorsal root ganglion under attack: the acquired sensory ganglionopathiesQ37811641
Design of multifunctional non-viral gene vectors to overcome physiological barriers: dilemmas and strategiesQ37907456
Animal and cellular models of Friedreich ataxia.Q38121944
Cardiomyopathy of Friedreich ataxiaQ38121946
P275copyright licenseCreative Commons Attribution 4.0 InternationalQ20007257
P6216copyright statuscopyrightedQ50423863
P407language of work or nameEnglishQ1860
P921main subjectnanoparticleQ61231
Friedreich ataxiaQ913856
P304page(s)20019
P577publication date2016-02-17
P1433published inScientific ReportsQ2261792
P1476titleIntrathecal delivery of frataxin mRNA encapsulated in lipid nanoparticles to dorsal root ganglia as a potential therapeutic for Friedreich's ataxia
P478volume6

Reverse relations

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