Featured
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Article |
MiR-21 derived from the exosomes of MSCs regulates the death and differentiation of neurons in patients with spinal cord injury
- Jian Kang
- , Zhenhuan Li
- & Guanghui Xu
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Article
| Open AccessDownregulation of the human peripheral myelin protein 22 gene by miR-29a in cellular models of Charcot–Marie–Tooth disease
- Jacquelyn Serfecz
- , Hannah Bazick
- & Lucia Notterpek
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Review Article |
AAV9 Vector: a Novel modality in gene therapy for spinal muscular atrophy
- Rithu Pattali
- , Yongchao Mou
- & Xue-Jun Li
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Brief Communication |
Safe and neuroprotective vectors for long-term traumatic brain injury gene therapy
- Daniela Blanco-Ocampo
- , Fabio Andrés Cawen
- & Hugo Peluffo
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Article |
Safety and efficacy evaluations of an adeno-associated virus variant for preparing IL10-secreting human neural stem cell-based therapeutics
- Mira Cho
- , Kwangsoo Jung
- & Jae-Hyung Jang
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Brief Communication
| Open AccessGDNF-mediated rescue of the nigrostriatal system depends on the degree of degeneration
- Luis Quintino
- , Martino Avallone
- & Cecilia Lundberg
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Article |
Unilateral ex vivo gene therapy by GDNF in epileptic rats
- Avtandil Nanobashvili
- , Esbjörn Melin
- & Merab Kokaia
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Brief Communication |
MR-guided delivery of AAV2-BDNF into the entorhinal cortex of non-human primates
- Alan H. Nagahara
- , Bayard R. Wilson
- & Mark H. Tuszynski
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Original Article
| Open AccessAAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington’s disease
- J Miniarikova
- , V Zimmer
- & P Konstantinova
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Review |
Management of neuromuscular diseases and spinal muscular atrophy in Latin America
- S Monges
- & A L Rosa
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Short Communication
| Open AccessAAV9-based gene therapy partially ameliorates the clinical phenotype of a mouse model of Leigh syndrome
- I Di Meo
- , S Marchet
- & C Viscomi
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Original Article |
Nasal application of HSV encoding human preproenkephalin blocks craniofacial pain in a rat model of traumatic brain injury
- A C Meidahl
- , M Klukinov
- & D C Yeomans
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Short Communication |
Gene therapy for spinomuscular atrophy: a biomedical advance, a missed opportunity for more equitable drug pricing
- T Friedmann
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Review |
The SMA Trust: the role of a disease-focused research charity in developing treatments for SMA
- V Christie-Brown
- , J Mitchell
- & K Talbot
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Original Article |
IL-4 mediated by HSV vector suppresses morphine withdrawal response and decreases TNFα, NR2B, and pC/EBPβ in the periaqueductal gray in rats
- H Yi
- , T Iida
- & S Hao
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Original Article
| Open AccessExtraneuronal pathology in a canine model of CLN2 neuronal ceroid lipofuscinosis after intracerebroventricular gene therapy that delays neurological disease progression
- M L Katz
- , G C Johnson
- & M S Whitney
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News and Commentary |
Gene therapy approaches for prevention of retinal degeneration in Usher syndrome
- D S Williams
- , A Chadha
- & D Gibbs
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Short Communication
| Open AccessDevelopment of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65
- A Georgiadis
- , Y Duran
- & R R Ali
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Short Communication |
Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism
- S K Powell
- , N Khan
- & T J McCown
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Original Article
| Open AccessLentivirus-meditated frataxin gene delivery reverses genome instability in Friedreich ataxia patient and mouse model fibroblasts
- H Khonsari
- , M Schneider
- & M Themis
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Original Article |
DREADDs suppress seizure-like activity in a mouse model of pharmacoresistant epileptic brain tissue
- N Avaliani
- , M Andersson
- & M Kokaia
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Original Article
| Open AccessDevelopment of an AAV9 coding for a 3XFLAG-TALEfrat#8-VP64 able to increase in vivo the human frataxin in YG8R mice
- P Chapdelaine
- , C Gérard
- & J P Tremblay
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Original Article |
Hair cell stereociliary bundle regeneration by espin gene transduction after aminoglycoside damage and hair cell induction by Notch inhibition
- A Taura
- , K Taura
- & A F Ryan
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Original Article |
Mifepristone-inducible transgene expression in neural progenitor cells in vitro and in vivo
- B E Hjelm
- , C Grunseich
- & T M Pierson
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Original Article |
Intracerebroventricular delivery of self-complementary adeno-associated virus serotype 9 to the adult rat brain
- A Donsante
- , Z McEachin
- & N M Boulis
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Original Article |
HSV vector-mediated GAD67 suppresses neuropathic pain induced by perineural HIV gp120 in rats through inhibition of ROS and Wnt5a
- H Kanda
- , M Kanao
- & S Hao
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Original Article |
AAV1/2-mediated BDNF gene therapy in a transgenic rat model of Huntington’s disease
- B Connor
- , Y Sun
- & C Maucksch
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Original Article
| Open AccessMucopolysaccharidosis IIIB confers enhanced neonatal intracranial transduction by AAV8 but not by 5, 9 or rh10
- J A Gilkes
- , M D Bloom
- & C D Heldermon
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Editorial |
Moving toward a gene therapy for Huntington’s disease
- J C Glorioso
- , J B Cohen
- & R M Friedlander
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Original Article |
Local and systemic responses following intravitreous injection of AAV2-encoded modified Volvox channelrhodopsin-1 in a genetically blind rat model
- E Sugano
- , K Tabata
- & H Tomita
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Original Article |
Hyaluronic acid pretreatment for Sendai virus-mediated cochlear gene transfer
- T Kurioka
- , K Mizutari
- & A Shiotani
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Original Article |
Photoreceptor rescue by an abbreviated human RPGR gene in a murine model of X-linked retinitis pigmentosa
- B S Pawlyk
- , O V Bulgakov
- & T Li
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Short Communication |
Slow AAV2 clearance from the brain of nonhuman primates and anti-capsid immune response
- L Samaranch
- , P Hadaczek
- & K S Bankiewicz
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Original Article |
β-Sarcoglycan gene transfer decreases fibrosis and restores force in LGMD2E mice
- E R Pozsgai
- , D A Griffin
- & L R Rodino-Klapac
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Original Article |
Evaluation of helper-dependent canine adenovirus vectors in a 3D human CNS model
- D Simão
- , C Pinto
- & P M Alves
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Original Article - Enabling Technologies |
Noninvasive tracking of gene transcript and neuroprotection after gene therapy
- J Ren
- , Y I Chen
- & P K Liu
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Original Article |
The spread of adenoviral vectors to central nervous system through pathway of cochlea in mimetic aging and young rats
- X Chen
- , X Zhao
- & W Kong
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Original Article
| Open AccessAAV1.NT-3 gene therapy attenuates spontaneous autoimmune peripheral polyneuropathy
- M E Yalvac
- , W D Arnold
- & Z Sahenk
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Original Article |
Gene delivery to rat and human Schwann cells and nerve segments: a comparison of AAV 1–9 and lentiviral vectors
- S A Hoyng
- , F De Winter
- & M J A Malessy
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Original Article |
Gene delivery to the spinal cord using MRI-guided focused ultrasound
- D Weber-Adrian
- , E Thévenot
- & I Aubert
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Original Article |
Cochlear protection against cisplatin by viral transfection of X-linked inhibitor of apoptosis protein across round window membrane
- H Jie
- , S Tao
- & J Wang
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Review |
Potential treatments for genetic hearing loss in humans: current conundrums
- R Minoda
- , T Miwa
- & H Takeda
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Original Article |
Minimal ureagenesis is necessary for survival in the murine model of hyperargininemia treated by AAV-based gene therapy
- C Hu
- , D S Tai
- & G S Lipshutz
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Original Article |
Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease
- V J McCurdy
- , H E Rockwell
- & D R Martin
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