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Perspective
| Open AccessPrecision ophthalmology: a call for Africa not to be left in the dark
- Lisa Roberts
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Article |
AAV2 vector optimization for retinal ganglion cell-targeted delivery of therapeutic genes
- Brahim Chaqour
- , Thu T. Duong
- & Ahmara G. Ross
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Brief Communication
| Open Access
Engineered compact pan-neuronal promoter from Alphaherpesvirus LAP2 enhances target gene expression in the mouse brain and reduces tropism in the liver
- Carola J. Maturana
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Article |
Overexpression of KCNN4 channels in principal neurons produces an anti-seizure effect without reducing their coding ability
- Evgeny S. Nikitin
- , Tatiana Y. Postnikova
- & Aleksey V. Zaitsev
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Article |
Acoustically targeted noninvasive gene therapy in large brain volumes
- Shirin Nouraein
- , Sangsin Lee
- & Jerzy O. Szablowski
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Article
| Open Access
CRISPR/sgRNA-directed synergistic activation mediator (SAM) as a therapeutic tool for Parkinson´s disease
- Luis Fernando Narváez-Pérez
- , Francisco Paz-Bermúdez
- & José Segovia
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Article
| Open Access
rAAV-PHP.B escapes the mouse eye and causes lethality whereas rAAV9 can transduce aniridic corneal limbal stem cells without lethality
- Seyedeh Zeinab Mirjalili Mohanna
- , Andrea J. Korecki
- & Elizabeth M. Simpson
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Article
| Open Access
Combinatorial gene therapy for epilepsy: Gene sequence positioning and AAV serotype influence expression and inhibitory effect on seizures
- Esbjörn Melin
- , My Andersson
- & Merab Kokaia
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Article |
Novel rAAV vector mediated intrathecal HGF delivery has an impact on neuroimmune modulation in the ALS motor cortex with TDP-43 pathology
- Barış Genç
- , Boram Nho
- & Hande Özdinler
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Brief Communication |
Sonoselective delivery using ultrasound and microbubbles combined with intravenous rAAV9 CLDN5-GFP does not increase endothelial gene expression
- Rikke Hahn Kofoed
- , Elizabeth M. Simpson
- & Isabelle Aubert
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Brief Communication |
Ptbp1 knockdown failed to induce astrocytes to neurons in vivo
- Guixiang Yang
- , Zixiang Yan
- & Kailun Fang
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Article
| Open Access
Improving adeno-associated viral (AAV) vector-mediated transgene expression in retinal ganglion cells: comparison of five promoters
- Bart Nieuwenhuis
- , Elise Laperrousaz
- & Andrew Osborne
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Article |
A new protocol for whole-brain biodistribution analysis of AAVs by tissue clearing, light-sheet microscopy and semi-automated spatial quantification
- Miguel M. Lopes
- , Jacques Paysan
- & Rui Jorge Nobre
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Article |
Characterization of a novel Pde6b-deficient rat model of retinal degeneration and treatment with adeno-associated virus (AAV) gene therapy
- Ian C. Han
- , Luke A. Wiley
- & Budd A. Tucker
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Article |
rAAV-mediated over-expression of acid ceramidase prevents retinopathy in a mouse model of Farber lipogranulomatosis
- Hanmeng Zhang
- , Murtaza S. Nagree
- & Daniel M. Lipinski
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Review Article |
Cerebral organoids as an in vitro model to study autism spectrum disorders
- Alexa Rabeling
- & Mubeen Goolam
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Article |
Assessment of AAV9 distribution and transduction in rats after administration through Intrastriatal, Intracisterna magna and Lumbar Intrathecal routes
- Jayanth Chandran
- , Ekram Ahmed Chowdhury
- & Guy M. L. Meno-Tetang
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Article |
Adeno-associated virus serotype 9 antibody titers in patients with SMA pre-screened for treatment with onasemnogene abeparvovec –routine care evidence
- Sharon Aharoni
- , Jacob Bistritzer
- & Yoram Nevo
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Article |
Gene therapy restores mitochondrial function and protects retinal ganglion cells in optic neuropathy induced by a mito-targeted mutant ND1 gene
- Yuan Liu
- , Jeremy D. Eastwood
- & Hong Yu
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Article |
Overexpressed ski efficiently promotes neurorestoration, increases neuronal regeneration, and reduces astrogliosis after traumatic brain injury
- Yu Zhai
- , Shi-Yang Ye
- & Yuan-Guo Zhou
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Article |
Upper motor neurons are a target for gene therapy and UCHL1 is necessary and sufficient to improve cellular integrity of diseased upper motor neurons
- Barış Genç
- , Javier H. Jara
- & P. Hande Özdinler
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Article |
Outer retinal transduction by AAV2-7m8 following intravitreal injection in a sheep model of CNGA3 achromatopsia
- M. Ross
- , A. Obolensky
- & R. Ofri
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Review Article |
Progress on siRNA-based gene therapy targeting secondary injury after intracerebral hemorrhage
- Daniyah A. Almarghalani
- & Zahoor A. Shah
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Article
| Open Access
Rs1h−/y exon 3-del rat model of X-linked retinoschisis with early onset and rapid phenotype is rescued by RS1 supplementation
- Yong Zeng
- , Haohua Qian
- & Paul A. Sieving
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Article |
CCR1 enhances SUMOylation of DGCR8 by up-regulating ERK phosphorylation to promote spinal nerve ligation-induced neuropathic pain
- Cunxian Shi
- , Jin Jin
- & Zhen Li
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Article |
XIAP gene therapy effects on retinal ganglion cell structure and function in a mouse model of glaucoma
- Shagana Visuvanathan
- , Adam N. Baker
- & Catherine Tsilfidis
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Brief Communication
| Open Access
Viral vector gene delivery of the novel chaperone protein SRCP1 to modify insoluble protein in in vitro and in vivo models of ALS
- Ian W. Luecke
- , Gloria Lin
- & Allison D. Ebert
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Article
| Open Access
RNAi and chemogenetic reporter co-regulation in primate striatal interneurons
- Walter Lerchner
- , Abdullah A. Adil
- & Barry J. Richmond
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Brief Communication
| Open Access
Enhanced CNS transduction from AAV.PHP.eB infusion into the cisterna magna of older adult rats compared to AAV9
- Diptaman Chatterjee
- , David J. Marmion
- & Jeffrey H. Kordower
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Article |
Striatal ΔFosB gene suppression inhibits the development of abnormal involuntary movements induced by L-Dopa in rats
- Goichi Beck
- , Jie Zhang
- & Stella M. Papa
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Article
| Open Access
AAV9-mediated Schwann cell-targeted gene therapy rescues a model of demyelinating neuropathy
- Alexia Kagiava
- , Christos Karaiskos
- & Kleopas A. Kleopa
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Article
| Open Access
Rescue of retinal ganglion cells in optic nerve injury using cell-selective AAV mediated delivery of SIRT1
- Ahmara G. Ross
- , Devin S. McDougald
- & Kenneth S. Shindler
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Review Article
| Open Access
Gene delivery to the hypoglossal motor system: preclinical studies and translational potential
- Brendan M. Doyle
- , Michele L. Singer
- & David D. Fuller
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Article
| Open Access
Highly efficient neuronal gene knockout in vivo by CRISPR-Cas9 via neonatal intracerebroventricular injection of AAV in mice
- Sam Hana
- , Michael Peterson
- & Shih-Ching Lo
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Brief Communication
| Open Access
Single-cell mapping of focused ultrasound-transfected brain
- A. S. Mathew
- , C. M. Gorick
- & R. J. Price
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Article
| Open Access
Double viral vector technology for selective manipulation of neural pathways with higher level of efficiency and safety
- Yoshinori Koshimizu
- , Kaoru Isa
- & Tadashi Isa
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Article |
Sensitization of ON-bipolar cells with ambient light activatable multi-characteristic opsin rescues vision in mice
- Subrata Batabyal
- , Sivakumar Gajjeraman
- & Samarendra Mohanty
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Article
| Open Access
Maximizing lentiviral vector gene transfer in the CNS
- Morgane Humbel
- , Mergim Ramosaj
- & Nicole Déglon
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Article
| Open Access
Optimization of adeno-associated viral vector-mediated transduction of the corticospinal tract: comparison of four promoters
- Bart Nieuwenhuis
- , Barbara Haenzi
- & James W. Fawcett
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Brief Communication |
Effect of connective tissue growth factor gene editing using adeno-associated virus–mediated CRISPR–Cas9 on rabbit glaucoma filtering surgery outcomes
- Eun Jung Lee
- , Jong Chul Han
- & Changwon Kee
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Article
| Open Access
Muscle overexpression of Klf15 via an AAV8-Spc5-12 construct does not provide benefits in spinal muscular atrophy mice
- Nina Ahlskog
- , Daniel Hayler
- & Melissa Bowerman
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Comment |
Mesenchymal stem cells as new perspective for the treatment of alcohol use disorder
- Rosana Camarini
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Article |
Therapeutic potential of adenovirus-encoding brain-derived neurotrophic factor for spina bifida aperta by intra-amniotic delivery in a rat model
- Wei Ma
- , Xiaowei Wei
- & Zhengwei Yuan
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Article |
Retina transduction by rAAV2 after intravitreal injection: comparison between mouse and rat
- Mariana S. Dias
- , Victor G. Araujo
- & Hilda Petrs-Silva
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Article
| Open Access
Downregulation 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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Article |
Effects of a combinatorial treatment with gene and cell therapy on retinal ganglion cell survival and axonal outgrowth after optic nerve injury
- Gabriel Nascimento-dos-Santos
- , Leandro Coelho Teixeira-Pinheiro
- & Hilda Petrs-Silva
Long-term benefits of hematopoietic stem cell-based macrophage/microglia delivery of GDNF to the CNS in a mouse model of Parkinson’s diseaseBrowse broader subjects
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